Juul, Tina; Søgaard, Karen; Roos, Ewa M.;
OBJECTIVE: To develop a patient-reported outcome evaluating the impact of neck pain. The results of item generation and reduction and subscale structure in support of the content and construct validity of the measure are reported. METHODS: Items were generated from the literature and through focus....... CONCLUSION: The Neck OutcOme Score has excellent content validity and preliminary results support a 5-subscale structure. Additional work is needed to assess the reliability, further construct validity and responsiveness....
Sørensen, Tina Juul; Søgaard, Karen; Roos, Ewa M;
OBJECTIVE: To develop a patient-reported outcome evaluating the impact of neck pain. The results of item generation and reduction and subscale structure in support of the content and construct validity of the measure are reported. METHODS: Items were generated from the literature and through focus...... deviation (SD) 15.9) years, range 24-85 years); 19 women) and 12 healthcare professionals were conducted before data saturation was achieved. A total of 196 patients with neck pain (mean age 47.8 (SD 13.7) years), range 18-89 years; 146 women) completed the preliminary questionnaire. Overall 35 items were....... CONCLUSION: The Neck OutcOme Score has excellent content validity and preliminary results support a 5-subscale structure. Additional work is needed to assess the reliability, further construct validity and responsiveness....
Basch, Ethan; Spertus, John; Dudley, R Adams; Wu, Albert; Chuahan, Cynthia; Cohen, Perry; Smith, Mary Lou; Black, Nick; Crawford, Amaris; Christensen, Keri; Blake, Kathleen; Goertz, Christine
To recommend methods for assessing quality of care via patient-reported outcome-based performance measures (PRO-PMs) of symptoms, functional status, and quality of life. A Technical Expert Panel was assembled by the American Medical Association-convened Physician Consortium for Performance Improvement. An environmental scan and structured literature review were conducted to identify quality programs that integrate PRO-PMs. Key methodological considerations in the design, implementation, and analysis of these PRO-PM data were systematically identified. Recommended methods for addressing each identified consideration were developed on the basis of published patient-reported outcome (PRO) standards and refined through public comment. Literature review focused on programs using PROs to assess performance and on PRO guidance documents. Thirteen PRO programs and 10 guidance documents were identified. Nine best practices were developed, including the following: provide a rationale for measuring the outcome and for using a PRO-PM; describe the context of use; select a measure that is meaningful to patients with adequate psychometric properties; provide evidence of the measure's sensitivity to differences in care; address missing data and risk adjustment; and provide a framework for implementation, interpretation, dissemination, and continuous refinement. Methods for integrating PROs into performance measurement are available. Copyright © 2015 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.
Jonathan C Hill
Full Text Available To develop and validate a patient report outcome measure (PROM for clinical practice that can monitor health status of patients with a range of musculoskeletal (MSK disorders.Constructs for inclusion in the MSK-PROM were identified from a consensus process involving patients with musculoskeletal conditions, clinicians, purchasers of healthcare services, and primary care researchers. Psychometric properties of the brief tool, including face and construct validity, repeatability and responsiveness were assessed in a sample of patients with musculoskeletal pain consulting physiotherapy services in the United Kingdom (n=425.The consensus process identified 10 prioritised domains for monitoring musculoskeletal health status: pain intensity, quality of life, physical capacity, interference with social/leisure activities, emotional well-being, severity of most difficult thing, activities and roles, understanding independence, and overall impact. As the EuroQol (EQ-5D-5L is a widely adopted PROMs tool and covers the first four domains listed, to reduce patient burden to a minimum the MSK-PROM was designed to capture the remaining six prioritised domains which are not measured by the EQ-5D-5L. The tool demonstrated excellent reliability, construct validity, responsiveness and acceptability to patients and clinicians for use in clinical practice.We have validated a brief patient reported outcome measure (MSK-PROM for use in clinical practice to measure musculoskeletal health status and monitor outcomes over time using domains that are meaningful to patients and sensitive to change. Further work will establish whether the MSK-PROM is useful in other musculoskeletal healthcare settings.
Watt, Torquil; Rasmussen, Ase Krogh; Groenvold, Mogens;
was within the set of problems involving attribution. Conclusion The cognitive interview methodology was effective in identifying and reducing problems within the questionnaire responding process. Patients tended to selectively report problems they considered to be caused by their thyroid disease even when......Objective To improve a newly developed patient-reported outcome measure for thyroid patients using cognitive interviewing. Methods Thirty-one interviews using immediate retrospective and expansive probing were conducted among patients with non-toxic goiter (n = 4), nodular toxic goiter (n = 5.......e. whether or not to report only issues considered of thyroid causality. Within each round of interviews, the number of problems declined from an initial average of six per interview to two, mainly due to a reduction in the number of problems associated with comprehension. The least amount of reduction...
Francis, David O.; Daniero, James J.; Hovis, Kristen L.; Sathe, Nila; Jacobson, Barbara; Penson, David F.; Feurer, Irene D.; McPheeters, Melissa L.
Purpose: The purpose of this study was to perform a comprehensive systematic review of the literature on voice-related patient-reported outcome (PRO) measures in adults and to evaluate each instrument for the presence of important measurement properties. Method: MEDLINE, the Cumulative Index of Nursing and Allied Health Literature, and the Health…
Lebwohl, Mark; Swensen, Andrine R; Nyirady, Judit; Kim, Edward; Gwaltney, Chad J; Strober, Bruce E
Chronic plaque psoriasis has a profound impact on a patient's daily life. To understand the effects of psoriasis treatments, it is essential to assess the patient's experience of symptoms and how they impact their daily life. The goal of this study was to develop and establish the content validity of a new patient reported outcome (PRO) psoriasis measure. The Psoriasis Symptom Diary was developed by (i) identifying key plaque psoriasis-related symptoms and impacts through qualitative patient interviews (n = 29); (ii) developing an initial set of items that captured the key patient experiences; and (iii) conducting cognitive interviews to test patient understanding of items selected for inclusion in the new psoriasis symptom measure (n = 16). Patients noted a variety of symptoms, with plaque-related pain (including related concepts of burning and stinging), changes in skin appearance, and itching reported by all patients. Patients also expressed notable embarrassment and avoidance of social situations, due to the appearance of plaques, and limited mobility. The Psoriasis Symptom Diary assesses the severity and impact of symptoms using a 24-hour recall period to reduce recall bias and error. The Psoriasis Symptom Diary was developed to assess important symptoms and disease-related impacts in a manner consistent with guidelines for establishing the content validity of new PRO instruments. Following quantitative psychometric testing, the Psoriasis Symptom Diary may support efficacy endpoints in clinical trials. © 2013 The International Society of Dermatology.
Evans, Christopher J; Chiou, Chiun-Fang; Fitzgerald, Kristina A; Evans, William J; Ferrell, Betty R; Dale, William; Fried, Linda P; Gandra, Shravanthi R; Dennee-Sommers, Brooke; Patrick, Donald L
The objective of this study was to develop a patient-reported outcome (PRO) to assess reduced muscle strength in sarcopenia. Qualitative research study. University of Arkansas for Medical Sciences. Subjects with sarcopenia. Adults aged 55 years and older with sarcopenia (n = 12) attended open-ended, concept elicitation interviews to characterize the functional effects of reduced muscle strength on their lives. The resulting qualitative data were analyzed using a qualitative analysis software program (Atlas.ti [Atlas.ti GmbH, Berlin, Germany]) and a common set of codes was developed to summarize the data. Subsequently, the initial PRO measure was drafted. Cognitive interviews were then conducted with additional sarcopenia subjects (n = 12) to refine the measure. Qualitative interviews identified key concepts (eg, impacts) in the areas of activities of daily living, emotions, social activities, energy, balance, coordination, sleep, and strength. Based on data from the cognitive debriefing interviews (eg, understandability, relevance, suggestions to reword items), the PRO measure development team came to consensus on which items or parts of the instructions to retain, revise, or delete. The final measure included 14 items. The final PRO measure, the Age-Related Muscle Loss Questionnaire, can be used in both clinical practice and clinical trial settings to assess functional impacts of reduced muscle strength in sarcopenia. Copyright © 2011 American Medical Directors Association. Published by Elsevier Inc. All rights reserved.
Arbuckle, Rob; Abetz-Webb, Linda
The US FDA and the European Medicines Agency (EMA) have issued incentives and laws mandating clinical research in pediatrics. While guidances for the development and validation of patient-reported outcomes (PROs) or health-related quality of life (HRQL) measures have been issued by these agencies, little attention has focused on pediatric PRO development methods. With reference to the literature, this article provides an overview of specific considerations that should be made with regard to the development of pediatric PRO measures, with a focus on performing qualitative research to ensure content validity. Throughout the questionnaire development process it is critical to use developmentally appropriate language and techniques to ensure outcomes have content validity, and will be reliable and valid within narrow age bands (0-2, 3-5, 6-8, 9-11, 12-14, 15-17 years). For qualitative research, sample sizes within those age bands must be adequate to demonstrate saturation while taking into account children's rapid growth and development. Interview methods, interview guides, and length of interview must all take developmental stage into account. Drawings, play-doh, or props can be used to engage the child. Care needs to be taken during cognitive debriefing, where repeated questioning can lead a child to change their answers, due to thinking their answer is incorrect. For the PROs themselves, the greatest challenge is in measuring outcomes in children aged 5-8 years. In this age range, while self-report is generally more valid, parent reports of observable behaviors are generally more reliable. As such, 'team completion' or a parent-administered child report is often the best option for children aged 5-8 years. For infants and very young children (aged 0-4 years), patient rating of observable behaviors is necessary, and, for adolescents and children aged 9 years and older, self-reported outcomes are generally valid and reliable. In conclusion, the development of PRO
Willke, Richard J
Patient-reported outcomes (PROs) can be important measures of the impact and value of new drug treatments to patients. Recently, both multisector stakeholder groups and the U.S. Food and Drug Administration have carefully considered and issued guidance on best practices for the use of PROs in measuring treatment impact. When best practices are followed and PRO data are appropriately included in drug development strategy and clinical trials, these data can be part of the evidence submitted for drug approval and included in drug labeling. One study showed that PRO data were included in 30% of a sample of new drug labels and were more concentrated in certain therapeutic areas, such as anti-inflammatory agents, vaccines, gastrointestinal agents, and respiratory and urologic agents. PRO data included in labeling, or generated in a similar scientific manner, may often then be used in other communication vehicles, such as formulary submission dossiers, journal or direct-to-consumer advertisements, publications, or continuing medical education. Meaningful and reliable PRO results regarding the effects of new treatments on how patients feel and function provide useful information to those who must make decisions about the availability and utilization of such treatments.
Watt, Torquil; Bjorner, Jakob Bue; Groenvold, Mogens
BACKGROUND: Thyroid diseases affect quality of life (QoL). The Thyroid-Related Patient-Reported Outcome (ThyPRO) is an international comprehensive well-validated patient-reported outcome, measuring thyroid-related QoL. The current version is rather long-85 items. The purpose of the present study...... was to develop an abbreviated version of the ThyPRO, with conserved good measurement properties. METHODS: A cross-sectional (N = 907) and a longitudinal sample (N = 435) of thyroid patients were analyzed. A graded item response theory (IRT) model was fitted to the cross-sectional data. Short-form scales...
I have a particular interest in the FDA's guidance on patient-reported outcome (PRO measures as I specialize in the design, development and use of such measures and license them to pharmaceutical companies, research organizations, academics and clinicians for use in clinical trials, other research and routine clinical practice. My measures include: the Diabetes Treatment Satisfaction Questionnaire (DTSQ) in it's status (DTSQs) and change (DTSQc) forms and related measures for other conditions...
Sadiqi, Said; Lehr, A. Mechteld; Post, Marcel W.; Dvorak, Marcel F.; Kandziora, Frank; Rajasekaran, S.; Schnake, Klaus J.; Vaccaro, Alexander R.; Oner, F. Cumhur
To report on the multi-phase process used in developing the AOSpine Patient Reported Outcome Spine Trauma (AOSpine PROST), as well as the results of its application in a pilot study. The International Classification of Functioning, Disability and Health (ICF) methodology was used as the basis for th
Petkovic, Jennifer; Barton, Jennifer L; Flurey, Caroline
and how to address equity issues within the core outcome sets of domains and instruments. METHODS: We surveyed current and previous OMERACT meeting attendees and members of the Campbell and Cochrane Equity Group regarding whether to address equity issues within the OMERACT Filter 2.0 Core Outcome Sets...
Castillo Mayret M
Full Text Available Abstract Background Measures that reflect patients' assessment of their health are of increasing importance as outcome measures in randomised controlled trials. The methodological approach used in the pre-validation development of new instruments (item generation, item reduction and question formatting should be robust and transparent. The totality of the content of existing PRO instruments for a specific condition provides a valuable resource (pool of items that can be utilised to develop new instruments. Such 'top down' approaches are common, but the explicit pre-validation methods are often poorly reported. This paper presents a systematic and generalisable 5-step pre-validation PRO instrument methodology. Methods The method is illustrated using the example of the Aberdeen Glaucoma Questionnaire (AGQ. The five steps are: 1 Generation of a pool of items; 2 Item de-duplication (three phases; 3 Item reduction (two phases; 4 Assessment of the remaining items' content coverage against a pre-existing theoretical framework appropriate to the objectives of the instrument and the target population (e.g. ICF; and 5 qualitative exploration of the target populations' views of the new instrument and the items it contains. Results The AGQ 'item pool' contained 725 items. Three de-duplication phases resulted in reduction of 91, 225 and 48 items respectively. The item reduction phases discarded 70 items and 208 items respectively. The draft AGQ contained 83 items with good content coverage. The qualitative exploration ('think aloud' study resulted in removal of a further 15 items and refinement to the wording of others. The resultant draft AGQ contained 68 items. Conclusions This study presents a novel methodology for developing a PRO instrument, based on three sources: literature reporting what is important to patient; theoretically coherent framework; and patients' experience of completing the instrument. By systematically accounting for all items dropped
Khanna, Puja; Agarwal, Nikhil; Khanna, Dinesh; Hays, Ron D; Chang, Lin; Bolus, Roger; Melmed, Gil; Whitman, Cynthia B; Kaplan, Robert M; Ogawa, Rikke; Snyder, Bradley; Spiegel, Brennan Mr
Because gastrointestinal (GI) illnesses can cause physical, emotional, and social distress, patient-reported outcomes (PROs) are used to guide clinical decision making, conduct research, and seek drug approval. It is important to develop a mechanism for identifying, categorizing, and evaluating the over 100 GI PROs that exist. Here we describe a new, National Institutes of Health (NIH)-supported, online PRO clearinghouse-the GI-PRO database. Using a protocol developed by the NIH Patient-Reported Outcome Measurement Information System (PROMIS(®)), we performed a systematic review to identify English-language GI PROs. We abstracted PRO items and developed an online searchable item database. We categorized symptoms into content "bins" to evaluate a framework for GI symptom reporting. Finally, we assigned a score for the methodological quality of each PRO represented in the published literature (0-20 range; higher indicates better). We reviewed 15,697 titles (κ>0.6 for title and abstract selection), from which we identified 126 PROs. Review of the PROs revealed eight GI symptom "bins": (i) abdominal pain, (ii) bloat/gas, (iii) diarrhea, (iv) constipation, (v) bowel incontinence/soilage, (vi) heartburn/reflux, (vii) swallowing, and (viii) nausea/vomiting. In addition to these symptoms, the PROs covered four psychosocial domains: (i) behaviors, (ii) cognitions, (iii) emotions, and (iv) psychosocial impact. The quality scores were generally low (mean 8.88 ± 4.19; 0 (min)-20 (max). In addition, 51% did not include patient input in developing the PRO, and 41% provided no information on score interpretation. GI PROs cover a wide range of biopsychosocial symptoms. Although plentiful, GI PROs are limited by low methodological quality. Our online PRO library (www.researchcore.org/gipro/) can help in selecting PROs for clinical and research purposes.
DeWalt Darren A
Full Text Available Abstract Background The evaluation of patient-reported outcomes (PROs in health care has seen greater use in recent years, and methods to improve the reliability and validity of PRO instruments are advancing. This paper discusses the cognitive interviewing procedures employed by the Patient Reported Outcomes Measurement Information System (PROMIS pediatrics group for the purpose of developing a dynamic, electronic item bank for field testing with children and adolescents using novel computer technology. The primary objective of this study was to conduct cognitive interviews with children and adolescents to gain feedback on items measuring physical functioning, emotional health, social health, fatigue, pain, and asthma-specific symptoms. Methods A total of 88 cognitive interviews were conducted with 77 children and adolescents across two sites on 318 items. From this initial item bank, 25 items were deleted and 35 were revised and underwent a second round of cognitive interviews. A total of 293 items were retained for field testing. Results Children as young as 8 years of age were able to comprehend the majority of items, response options, directions, recall period, and identify problems with language that was difficult for them to understand. Cognitive interviews indicated issues with item comprehension on several items which led to alternative wording for these items. Conclusion Children ages 8–17 years were able to comprehend most item stems and response options in the present study. Field testing with the resulting items and response options is presently being conducted as part of the PROMIS Pediatric Item Bank development process.
Neale, Joanne; Vitoratou, Silia; Finch, Emily; Lennon, Paul; Mitcheson, Luke; Panebianco, Daria; Rose, Diana; Strang, John; Wykes, Til; Marsden, John
Patient Reported Outcome Measures (PROMs) assess health status and health-related quality of life from the patient/service user perspective. Our study aimed to: i. develop a PROM for recovery from drug and alcohol dependence that has good face and content validity, acceptability and usability for people in recovery; ii. evaluate the psychometric properties and factorial structure of the new PROM ('SURE'). Item development included Delphi groups, focus groups, and service user feedback on draft versions of the new measure. A 30-item beta version was completed by 575 service users (461 in person [IP] and 114 online [OL]). Analyses comprised rating scale evaluation, assessment of psychometric properties, factorial structure, and differential item functioning. The beta measure had good face and content validity. Nine items were removed due to low stability, low factor loading, low construct validity or high complexity. The remaining 21 items were re-scaled (Rasch model analyses). Exploratory and confirmatory factor analyses revealed 5 factors: substance use, material resources, outlook on life, self-care, and relationships. The MIMIC model indicated 95% metric invariance across the IP and OL samples, and 100% metric invariance for gender. Internal consistency and test-retest reliability were granted. The 5 factors correlated positively with the corresponding WHOQOL-BREF and ARC subscales and score differences between participant sub-groups confirmed discriminative validity. 'SURE' is a psychometrically valid, quick and easy-to-complete outcome measure, developed with unprecedented input from people in recovery. It can be used alongside, or instead of, existing outcome tools. Copyright © 2016 The Authors. Published by Elsevier Ireland Ltd.. All rights reserved.
Bhatia Kailash P
Full Text Available Abstract Background The United States Food and Drug Administration (FDA are currently producing guidelines for the scientific adequacy of patient reported outcome measures (PROMs in clinical trials, which will have implications for the selection of scales used in future clinical trials. In this study, we examine how the Cervical Dystonia Impact Profile (CDIP-58, a rigorous Rasch measurement developed neurologic PROM, stands up to traditional psychometric criteria for three reasons: 1 provide traditional psychometric evidence for the CDIP-58 in line with proposed FDA guidelines; 2 enable researchers and clinicians to compare it with existing dystonia PROMs; and 3 help researchers and clinicians bridge the knowledge gap between old and new methods of reliability and validity testing. Methods We evaluated traditional psychometric properties of data quality, scaling assumptions, targeting, reliability and validity in a group of 391 people with CD. The main outcome measures used were the CDIP-58, Medical Outcome Study Short Form-36, the 28-item General Health Questionnaire, and Hospital and Anxiety and Depression Scale. Results A total of 391 people returned completed questionnaires (corrected response rate 87%. Analyses showed: 1 data quality was high (low missing data ≤ 4%, subscale scores could be computed for > 96% of the sample; 2 item groupings passed tests for scaling assumptions; 3 good targeting (except for the Sleep subscale, ceiling effect = 27%; 4 good reliability (Cronbach's alpha ≥ 0.92, test-retest intraclass correlations ≥ 0.83; and 5 validity was supported. Conclusion This study has shown that new psychometric methods can produce a PROM that stands up to traditional criteria and supports the clinical advantages of Rasch analysis.
Kimball, Alexa B; Sundaram, Murali; Banderas, Benjamin; Foley, Catherine; Shields, Alan L
Two patient-reported outcome (PRO) questionnaires, the Hidradenitis Suppurativa Symptom Assessment (HSSA) and Hidradenitis Suppurativa Impact Assessment (HSIA), were developed to measure signs, symptoms and impacts of HS in treatment efficacy studies. In accordance with FDA guidelines and published best practices, four stages of research were conducted to create the questionnaires: concept elicitation, questionnaire construction, content evaluation and psychometric evaluation. Subjects (N = 20) who participated in the concept elicitation stage reported 15 unique HS-related signs and symptoms and 51 impacts. Following this, eight sign and symptom concepts and 21 impacts were selected for construction of the HSSA and HSIA, respectively. During content evaluation, cognitive debriefing interviews with HS subjects (N = 20) confirmed subjects could read, comprehend and meaningfully respond to both questionnaires. Modifications made after this stage of work resulted in a nine-item HSSA and a 17-item HSIA. The HSSA and HSIA were subsequently entered into a US-based observational study (N = 40), and the scores produced by each were found to be reliable, construct valid, and able to distinguish among clinically distinct groups. The HSSA and HSIA are content-valid, HS-specific, PRO questionnaires with demonstrated ability to generate reliable, valid scores when administered to patients with HS in a research setting.
Alexis, Andrew; Daniels, Selena R; Johnson, Nathan; Pompilus, Farrah; Burgess, Somali Misra; Harper, Julie C
Facial acne has been associated with impaired health-related quality of life, which is an essential patient outcome for evaluating the success of acne treatment. In consideration of the US Food and Drug Administration's (FDA) new recommendations on patient reported outcome (PRO) measures, the objectives of this study were to (1) establish the need for a new PRO measure that assesses facial acne outcomes and satisfies the criteria set forth by the FDA and (2) develop the content of a new facial acne PRO measure appropriate for use in both adolescents and adults as well as adherent to the FDA PRO Guidance. A literature and PRO review, patient interviews (concept elicitation), and input from clinical experts were used to develop a conceptual framework for the outcomes deemed important to facial acne patients, and to construct items for a preliminary PRO measure: the Acne Symptom and Impact Scale (ASIS). Cognitive interviews were conducted to pilot test the ASIS. A review of the literature and PROs revealed that, of the 34 measures identified, no suitable PRO measure for the population of interest was available. The conceptual framework comprised two main themes: symptoms and psychosocial impacts. Concept elicitation interviews included a diverse set of patients (n=48) with facial acne, of various ages: 12-17 years (n=15), 18-25 years (n=20), and 26-50 years (n=13). The most frequently reported symptoms were: pimples, oily skin, scabs/scars/marks, blackheads, acne, and whiteheads. The most frequently reported impacts were impacts on appearance, self-consciousness, annoyance, bothersomeness, mood, social criticism, embarrassment, confidence, and social withdrawal. These reported symptoms and impacts constituted the 15-item draft ASIS. The draft ASIS was modified following the analysis of 20 cognitive interviews, resulting in the current 17-item ASIS. Results from both the concept elicitation and cognitive interviews demonstrated that the ASIS is content valid in both
Chua, Alicia S; Glanz, Bonnie I; Guarino, Anthony J; Cook, Sandra L; Greeke, Emily E; Little, Grace E; Chitnis, Tanuja; Healy, Brian C
Several patient-reported outcome (PRO) measures are commonly used in multiple sclerosis (MS) research, but the relationship among items across measures is uncertain. We proposed to evaluate the associations between items from a standard battery of PRO measures used in MS research and to develop a brief, reliable and valid instrument measure by combining these items into a single measure. Subjects (N = 537) enrolled in CLIMB complete a PRO battery that includes the Center for Epidemiologic Studies Depression Scale, Medical Outcomes Study Modified Social Support Survey, Modified Fatigue Impact Scale and Multiple Sclerosis Quality of Life-54. Subjects were randomly divided into two samples: calibration (n = 269) and validation (n = 268). In the calibration sample, an Exploratory Factor Analysis (EFA) was used to identify latent constructs within the battery. The model constructed based on the EFA was evaluated in the validation sample using Confirmatory Factor Analysis (CFA), and reliability and validity were assessed for the final measure. The EFA in the calibration sample revealed an eight factor solution, and a final model with one second-order factor along with the eight first-order factors provided the best fit. The model combined items from each of the four parent measures, showing important relationships among the parent measures. When the model was fit using the validation sample, the results confirmed the validity and reliability of the model. A brief PRO for MS (BPRO-MS) that combines MS-related psychosocial and quality of life domains can be used to assess overall functioning in mildly disabled MS patients.
Full Text Available Abstract Objective To develop the content for two new patient reported outcome (PRO measures to: a assess the severity of symptoms; and b the impact of facial skin oiliness on emotional wellbeing using qualitative data from face to face, and internet focus groups in Germany and the US. Methods Using input from initial treatment satisfaction focus groups (n = 42, a review of relevant literature and expert clinicians (n = 3, a discussion guide was developed to guide qualitative inquiry using Internet focus groups (IFGs. IFGs were conducted with German (n = 26 and US (n = 28 sufferers of oily skin. Questionnaire items were generated using coded transcript data from the focus groups. Cognitive debriefing was conducted online with 42 participants and face to face with an additional five participants to assess the comprehension of the items. Results There were equal numbers of male and female participants; mean age was 35.4 (SD 9.3 years. On average, participants had had oily skin for 15.2 years, and 74% (n = 40 reported having mild-moderate acne. Participants reported using visual, tactile and sensory (feel without touching their face methods to evaluate the severity of facial oiliness. Oily facial skin had both an emotional and social impact, and was associated with feelings of unattractiveness, self-consciousness, embarrassment, irritation and frustration. Items were generated for a measure of oily skin severity (Oily Skin Self-Assessment Scale and a measure of the impact of oily skin on emotional well-being (Oily Skin Impact Scale. Cognitive debriefing resulted in minor changes to the draft items and confirmed their face and content validity. Conclusion The research provides insight into the experience of having oily skin and illustrates significant difficulties associated with the condition. Item content was developed for early versions of two PRO measures of the symptoms and emotional impact of oily facial skin. The psychometric validation of
Marquis, P; Lasch, K E; Delgado-Herrera, L; Kothari, S; Lembo, A; Lademacher, C; Spears, G; Nishida, A; Tesler, Waldman L; Piault, E; Rosa, K; Zeiher, B
Despite a documented clinical need, no patient reported outcome (PRO) symptom measure meeting current regulatory requirements for clinically relevant end points is available for the evaluation of treatment benefit in diarrhea-predominant IBS (IBS-D). Patients (N=113) with IBS-D participated in five study phases: (1) eight concept elicitation focus groups (N=34), from which a 17-item IBS-D Daily Symptom Diary and four-item IBS-D Symptom Event Log (Diary and Event Log) were developed; (2) one-on-one cognitive interviews (N=11) to assess the instrument's comprehensiveness, understandability, appropriateness, and readability; (3) four data triangulation focus groups (N=32) to confirm the concepts elicited; (4) two hybrid (concept elicitation and cognitive interview) focus groups (N=16); and (5) two iterative sets of one-on-one cognitive interviews (N=20) to further clarify the symptoms of IBS-D and debrief a revised seven-item Diary and four-item Event Log. Of thirty-six concepts initially identified, 22 were excluded because they were not saturated, not clinically relevant, not critical symptoms of IBS-D, considered upper GI symptoms, or too broad or vaguely defined. The remaining concepts were diarrhea, immediate need (urgency), bloating/pressure, frequency of bowel movements, cramps, abdominal/stomach pain, gas, completely emptied bowels/incomplete evacuation, accidents, bubbling in intestines (bowel sounds), rectal burning, stool consistency, rectal spasm, and pain while wiping. The final instrument included a daily diary with separate items for abdominal and stomach pain and an event log with four items completed after each bowel movement as follows: (1) a record of the bowel movement/event and an assessment of (2) severity of immediacy of need/bowel urgency, (3) incomplete evacuation, and (4) stool consistency (evaluated using the newly developed Astellas Stool Form Scale). Based on rounds of interviews and clinical input, items considered secondary or
Pusic, Andrea L; Klassen, Anne F; Scott, Amie M; Cano, Stefan J
Satisfaction with appearance and improved quality of life are key outcomes for patients undergoing facial aesthetic procedures. The FACE-Q is a new patient-reported outcome (PRO) instrument encompassing a suite of independently functioning scales designed to measure a range of important outcomes for facial aesthetics patients. FACE-Q scales were developed with strict adherence to international guidelines for PRO instrument development. This article describes the development and psychometric evaluation of the core FACE-Q scale, the Satisfaction with Facial Appearance scale. Both modern and traditional psychometric methods were used to confirm that this new 10-item scale is a reliable, valid, and responsive measure.
Botturi, Davide; Rodella, Stefania
The concept of "patient-reported outcomes" have been proposed by the Food and Drug Administration in the year 2000, in order to describe one of the different and potential sources of information on the drug's safety and effectiveness. It represents an "umbrella" term, which covers a multiplicity of meanings and primarily identifies a conceptual approach and a methodology specifically oriented to the patients' point of view on outcomes, instead of the traditional clinical and professional perspective. The patient-reported outcomes measures are frequently self-completed questionnaires. The measures can be classified in general and specific. The first one, general, relates to the assessment of the quality of life or the health status, in the general population or in subgroups with particular health problems (eg. SF-36 Health Survey, EQ-5D). The second one, specific, mainly relates to the assessment of particular types of symptoms (eg. pain, anxiety, fear, depression) and functions (eg. daily living activities), in population's subgroups with definite health problems, undergoing or not to a healthcare procedure (eg. Adult Asthma Quality of Life Questionnaire, Kidney Disease Quality of Life Instrument, Oxford Hip Score, Oxford Knee Score). For the selection of an instrument a series of criteria needs to be taken into account, among which the psychometric properties, the expert judgement, the interpretability, the acceptability, and the feasibility of the entire process.
Naegeli, A. N.; Nixon, A; Burge, R.; Gold, D. T.; Silverman, S
Summary We have developed a short, patient-reported outcome questionnaire—the Osteoporosis Assessment Questionnaire—Physical Function (OPAQ-PF)—that assesses the impact of osteoporosis on physical function. OPAQ-PF contains 15 items in three domains (mobility, physical positions, and transfers) and has content validity in osteoporosis patients with and without a history of fracture. Introduction This paper describes the development of the Osteoporosis Assessment Questionnaire—Physical Functio...
Doward Lynda C
Full Text Available Abstract The use of patient reported outcome scales in clinical trials conducted by the pharmaceutical industry has become more widespread in recent years. The use of such outcomes is particularly common for products developed to treat chronic, disabling conditions where the intention is not to cure but to ameliorate symptoms, facilitate functioning or, ultimately, to improve quality of life. In such cases, patient reported evidence is increasingly viewed as an essential complement to traditional clinical evidence for establishing a product's competitive advantage in the marketplace. In a commercial setting, the value of patient reported outcomes is viewed largely in terms of their potential for securing a labelling claim in the USA or inclusion in the summary of product characteristics in Europe. Although, the publication of the recent US Food and Drug Administration guidance makes it difficult for companies to make claims in the USA beyond symptom improvements, the value of these outcomes goes beyond satisfying requirements for a label claim. The European regulatory authorities, payers both in the US and Europe, clinicians and patients all play a part in determining both the availability and the pricing of medicinal products and all have an interest in patient-reported data that go beyond just symptoms. The purpose of the current paper is to highlight the potential added value of patient reported outcome data currently collected and held by the industry for these groups.
Full Text Available Abstract This guidance describes how the FDA evaluates patient-reported outcome (PRO instruments used as effectiveness endpoints in clinical trials. It also describes our current thinking on how sponsors can develop and use study results measured by PRO instruments to support claims in approved product labeling (see appendix point 1. It does not address the use of PRO instruments for purposes beyond evaluation of claims made about a drug or medical product in its labeling. By explicitly addressing the review issues identified in this guidance, sponsors can increase the efficiency of their endpoint discussions with the FDA during the product development process, streamline the FDA's review of PRO endpoint adequacy, and provide optimal information about the patient's perspective of treatment benefit at the time of product approval. A PRO is a measurement of any aspect of a patient's health status that comes directly from the patient (i.e., without the interpretation of the patient's responses by a physician or anyone else. In clinical trials, a PRO instrument can be used to measure the impact of an intervention on one or more aspects of patients' health status, hereafter referred to as PRO concepts, ranging from the purely symptomatic (response of a headache to more complex concepts (e.g., ability to carry out activities of daily living, to extremely complex concepts such as quality of life, which is widely understood to be a multidomain concept with physical, psychological, and social components. Data generated by a PRO instrument can provide evidence of a treatment benefit from the patient perspective. For this data to be meaningful, however, there should be evidence that the PRO instrument effectively measures the particular concept that is studied. Generally, findings measured by PRO instruments may be used to support claims in approved product labeling if the claims are derived from adequate and well-controlled investigations that use PRO
Rothman, Margaret; Gnanaskathy, Ari; Wicks, Paul; Papadopoulos, Elektra J
We report a panel designed to open a dialog between pharmaceutical sponsors, regulatory reviewers, and other stakeholders regarding the use of social media to collect data to support the content validity of patient-reported outcome instruments in the context of medical product labeling. Multiple stakeholder perspectives were brought together to better understand the issues encountered in pursuing social media as a form of data collection to support content validity. Presenters represented a pharmaceutical sponsor of clinical trials, a regulatory reviewer from the Food and Drug Administration, and an online data platform provider. Each presenter shared its perspective on the advantages and disadvantages of using social media to collect this type of information. There was consensus that there is great potential for using social media for this purpose. There remain, however, unanswered questions that need to be addressed such as identifying which type of social media is most appropriate for data collection and ensuring that participants are representative of the target population while maintaining the advantages of anonymity provided by online platforms. The use of social media to collect evidence of content validity holds much promise. Clarification of issues that need to be addressed and accumulation of empirical evidence to address these questions are essential to moving forward.
Hviid, Line E; Healy, Brian C; Rintell, David J; Chitnis, Tanuja; Weiner, Howard L; Glanz, Bonnie I
Benign MS patients have a mild course of disease and show no or minimal accumulation of disability over time. Little is known about the patient reported outcomes (PROs) in benign MS. The objective of the study was to compare PROs in benign MS patients and patients with similar disease duration or disability status, and to investigate how the definition of benign MS affected this outcome. Two groups of Benign MS patients (disease duration ≥15 years, Expanded Disability Status Scale [EDSS] score ≤1.5 [Benign-1.5], or ≤3.0 [Benign-3]) were compared with four other MS groups: disease duration ≥15 years, EDSS score >3.0 (Late-MS); disease duration MS). PROs included measures of QOL, fatigue, depression, and social support. Cognitive function was also assessed. Both benign groups had better PROs than Late MS patients on all measures (p QOL, depression, and fatigue were significantly different between Benign-1.5 and Early-MS groups (p QOL than Low EDSS-1.5, but was otherwise similar. Benign-3 patients had worse depression than Early-MS (p QOL and lower fatigue (p MS had better PROs than other groups of MS patients, suggesting that both disease duration and disability influence PROs. The study also showed a difference in PROs based on the way benign MS was defined.
McGowan, Shane M.; Audley, Brittany N.; Sokunbi, Gbolabo; Puccio, Steven T.
Study Design Retrospective, case series. Purpose The purpose of this study is to determine morbidity, complications, and patient reported outcomes from minimally invasive sacroiliac joint (SIJ) fusion. Overview of Literature Lumbar back pain emanating from the SIJ can be surgically treated via a percutaneous approach in the appropriately selected patient with minimal morbidity and acceptable functional outcomes. Methods Patients diagnosed by >2 physical examination maneuvers and subjective relief from a computed tomography–guided lidocaine-bupivacaine-steroid injection underwent SIJ fusion after failing conservative management with a combination of oral anti-inflammatory medications, physical therapy, and pelvic belt stabilization. Perioperative data collected include estimated blood loss (EBL) and operative time. Oswestry disability index, 12-item short form health survey (SF-12), visual analogue score, and functional status were analyzed. All complications were noted. Results The study cohort of 45 cases (69% female) achieved postoperative survey follow-up at 9.9 and 32.3 months. SF-12 physical component summary statistically improved while all other scores were equivalent. Mean EBL and operative time were 22 mL and 36 minutes, respectively. Initial survey showed that 64% of patients discontinued narcotics (29/45), 71% do not use an assistive device (32/45), and 15.6% do not work due to pain (7/45). 73% of patients stated they would have the surgery again (33/45). For the second survey, 65% of patients discontinued narcotics (26/40), 70% did not use an assistive device (28/40), and 17.5% did not work due to pain (7/40). A history of thoracolumbar instrumentation (16/45) did not significantly affect outcomes. Three complications described by screw malposition with neurologic deficit (6.7%) were treated with screw repositioning (1 case) and removal of a single superior implant (2 cases) with time to revision of 2.2 months. All three ultimately had resolution of
Many interventions for neurogenic bladder patients are directed towards improving quality of life (QOL). Patient reported outcome measures (PROMs) are the primary method of evaluating QOL, and they provide an important quantification of symptoms which can’t be measured objectively. Our goal was to review general measurement principles, and identify and discuss PROMs relevant to neurogenic bladder patients. We identify two recent reviews of the state of the literature and updated the results with an additional Medline search up to September 1, 2015. Using the previous identified reviews, and our updated literature review, we identified 16 PROMs which are used for the assessment of QOL and symptoms in neurogenic bladder patients. Several are specifically designed for neurogenic bladder patients, such as the Qualiveen (for neurogenic bladder related QOL), and the Neurogenic Bladder Symptom Score (NBSS) (for neurogenic bladder symptoms). We also highlight general QOL measures for patients with multiple sclerosis (MS) and spinal cord injury (SCI) which include questions about bladder symptoms, and incontinence PROMs which are commonly used, but not specifically designed for neurogenic bladder patients. It is essential for clinicians and researchers with an interest in neurogenic bladder to be aware of the current PROMs, and to have a basic understanding of the principals of measurement in order to select the most appropriate one for their purpose. PMID:26904409
Clark, Roderick; Welk, Blayne
Many interventions for neurogenic bladder patients are directed towards improving quality of life (QOL). Patient reported outcome measures (PROMs) are the primary method of evaluating QOL, and they provide an important quantification of symptoms which can't be measured objectively. Our goal was to review general measurement principles, and identify and discuss PROMs relevant to neurogenic bladder patients. We identify two recent reviews of the state of the literature and updated the results with an additional Medline search up to September 1, 2015. Using the previous identified reviews, and our updated literature review, we identified 16 PROMs which are used for the assessment of QOL and symptoms in neurogenic bladder patients. Several are specifically designed for neurogenic bladder patients, such as the Qualiveen (for neurogenic bladder related QOL), and the Neurogenic Bladder Symptom Score (NBSS) (for neurogenic bladder symptoms). We also highlight general QOL measures for patients with multiple sclerosis (MS) and spinal cord injury (SCI) which include questions about bladder symptoms, and incontinence PROMs which are commonly used, but not specifically designed for neurogenic bladder patients. It is essential for clinicians and researchers with an interest in neurogenic bladder to be aware of the current PROMs, and to have a basic understanding of the principals of measurement in order to select the most appropriate one for their purpose.
Subramaniam, Mythily; Soh, Pauline; Ong, Clarissa; Esmond Seow, Lee Seng; Picco, Louisa; Vaingankar, Janhavi Ajit; Chong, Siow Ann
The purpose of the article was to provide an overview of patient-reported outcomes (PROs) and related measures that have been examined in the context of obsessive-compulsive disorder (OCD). The current review focused on patient-reported outcome measures (PROMs) that evaluated three broad outcome domains: functioning, health-related quality of life (HRQoL), and OCD-related symptoms. The present review ultimately included a total of 155 unique articles and 22 PROMs. An examination of the PROs revealed that OCD patients tend to suffer from significant functional disability, and report lower HRQoL than controls. OCD patients report greater symptom severity than patients with other mental disorders and evidence indicates that PROMs are sensitive to change and may be even better than clinician-rated measures at predicting treatment outcomes. Nonetheless, it should be noted that the measures reviewed lacked patient input in their development. Future research on PROMs must involve patient perspectives and include rigorous psychometric evaluation of these measures. PMID:25152661
Pellar, Russell E; Tingey, Theresa M; Pope, Janet Elizabeth
Scleroderma (systemic sclerosis) is a rare autoimmune connective tissue disease that can damage multiple organs and reduce quality of life. Patient-reported outcome measures capture the patient's perspective. Some measures are specific to systemic sclerosis and others are general. Patient-reported outcomes in systemic sclerosis are important to aid in understanding the impact of systemic sclerosis on patients.
Juhl, Carsten Bogh; Lund, Hans; Guyatt, GH
Title A hierarchy of patient-reported outcome measures for meta-analysis of knee osteoarthritis trials: empirical evidence from a survey of high impact journals Objective To develop a prioritized list for extracting patient-reported outcomes (PROs) measuring pain and disability for meta-analyses ......Title A hierarchy of patient-reported outcome measures for meta-analysis of knee osteoarthritis trials: empirical evidence from a survey of high impact journals Objective To develop a prioritized list for extracting patient-reported outcomes (PROs) measuring pain and disability for meta...
Full Text Available Table of contents S1 Using computerized adaptive testing Tim Croudace S2 Well-being: what is it, how does it compare to health and what are the implications of using it to inform health policy John Brazier O1 “Am I going to get better?”—Using PROMs to inform patients about the likely benefit of surgery Nils Gutacker, Andrew Street O2 Identifying Patient Reported Outcome Measures for an electronic Personal Health Record Dan Robotham, Samantha Waterman, Diana Rose, Safarina Satkunanathan, Til Wykes O3 Examining the change process over time qualitatively: transformative learning and response shift Nasrin Nasr, Pamela Enderby O4 Developing a PROM to evaluate self-management in diabetes (HASMID: giving patients a voice Jill Carlton, Donna Rowen, Jackie Elliott, John Brazier, Katherine Stevens, Hasan Basarir, Alex Labeit O5 Development of the Primary Care Outcomes Questionnaire (PCOQ Mairead Murphy, Sandra Hollinghurst, Chris Salisbury O6 Developing the PKEX score- a multimodal assessment tool for patients with shoulder problems Dominic Marley, James Wilson, Amy Barrat, Bibhas Roy O7 Applying multiple imputation to multi-item patient reported outcome measures: advantages and disadvantages of imputing at the item, sub-scale or score level Ines Rombach, Órlaith Burke, Crispin Jenkinson, Alastair Gray, Oliver Rivero-Arias O8 Integrating Patient Reported Outcome Measures (PROMs into routine primary care for patients with multimorbidity: a feasibility study Ian Porter, Jaheeda Gangannagaripalli, Charlotte Bramwell, Jose M. Valderas O9 eRAPID: electronic self-report and management of adverse-events for pelvic radiotherapy (RT patients Patricia Holch, Susan Davidson, Jacki Routledge, Ann Henry, Kevin Franks, Alex Gilbert, Kate Absolom & Galina Velikova O10 Patient reported outcomes (PROMs based recommendation in clinical guidance for the management of chronic conditions in the United Kingdom Ian Porter, Jose M.Valderas O11 Cross-sectional and
Copyright laws are intended to protect the rights of authors in their literary, scientific, and artistic works. The recent controversy about the standardized version of the Asthma Quality of Life Questionnaire between Elizabeth Juniper and Eirini Grammatopoulou et al. is an example of the difficulties inherent to copyright faced by developers and users of patient-reported outcome (PRO) instruments. This brief communication presents the basics of international copyright laws (i.e., the Berne Convention), the facts behind the controversy, and our analysis of the case based on our experience as a distributing center of PRO instruments. We conclude that better communication between developers and users would prevent most unfortunate complications and misunderstandings.
The development and validation of a multidimensional sum-scaling questionnaire to measure patient-reported outcomes in acute respiratory tract infections in primary care: the Acute Respiratory Tract Infection Questionnaire: ARTIQ
Aabenhus, R.; Thorsen, H.; Siersma, V.
OBJECTIVE: Patient-reported outcomes are seldom validated measures in clinical trials of acute respiratory tract infections (ARTIs) in primary care. We developed and validated a patient-reported outcome sum-scaling measure to assess the severity and functional impacts of ARTIs. METHODS: Qualitati......, sum-scaling questionnaire with high face and content validity and adequate psychometric properties for assessing severity and functional impacts from ARTIs in adults is available to clinical trials and audits in primary care....
Bonner, Nicola; Abetz-Webb, Linda; Renault, Lydie; Caballero, Teresa; Longhurst, Hilary; Maurer, Marcus; Christiansen, Sandra; Zuraw, Bruce
Hereditary Angioedema (HAE), a rare genetic disease, manifests as intermittent, painful attacks of angioedema. Attacks vary in frequency and severity and include skin, abdominal and life-threatening laryngeal swellings. This study aimed to develop a patient reported outcome (PRO) tool for the assessment of HAE attacks, including their management and impact on patients' lives, for use in clinical studies, or by physicians in general practice. The results of open-ended face to face concept elicitation interviews with HAE patients in Argentina (n = 10) and the US (n = 33) were used to develop the first draft questionnaire of the HAE patient reported outcomes questionnaire (HAE PRO). Subsequently, in-depth cognitive debriefing interviews were performed with HAE patients in the UK (n = 10), Brazil (n = 10), Germany (n = 11) and France (n = 12). Following input from eight multinational clinical experts further cognitive interviews were conducted in the US (n = 12) and Germany (n = 12). Patients who experienced abdominal, cutaneous or laryngeal attacks of varying severity levels were included in all rounds of interviews. Across the rounds of interviews patients discussed their HAE attack symptoms, impacts and treatments. Cognitive debriefing interviews explored patient understanding and relevance of questionnaire items. All interviews were conducted face to face following a pre-defined semi-structured interview guide in the patient's native language. Patients reported a variety of HAE symptoms, attack triggers, warning signs, attack impacts and treatment options which were used to develop the HAE PRO. The HAE PRO was revised and refined following input from patients and clinical experts. The final 18-item HAE PRO provides an assessment of the HAE attack experience including symptoms, impacts, treatment requirements, healthcare resource use and loss of productivity caused by HAE attacks. Patient and expert input has contributed to the
Moore, P; Jackson, C; Mutch, K; Methley, A; Pollard, C; Hamid, S; Jacob, A
Objective This study outlines the development of a patient-reported outcome measure (PROM), an instrument to obtain self-reported health status for neuromyeltis optica (NMO), a disabling neurological condition. Design Development was conducted in accordance with international guidance for PROMs including systematic review of existing literature, item generation guided by qualitative interviews, health-related quality of life conceptual framework and clinical expert panel and cognitive interviews with NMO patients. Setting Participants were identified through a national NMO clinic in a tertiary NHS neurosciences service. Participants 15 individuals with NMO participated in cognitive interviews requiring review and ranking of proposed PROM items and qualitative feedback on content, layout and response options. Results Participants endorsed the draft instrument as reflecting their experience of the condition and as being easy to understand. Rating and ranking of item relevance and importance reduced the draft instrument from 106 to 48 items. Participant feedback on overlapping items eliminated a further 2 items and resulted in a preliminary instrument of 46 items. As a direct result of participant feedback ordering of the 10 domains was revised, a 4 option Likert scale was employed and a 4-week recall period for impact of symptoms was selected. Conclusions A 46-item instrument developed in accordance with international PROM development guidelines through literature review, developed by subject matter experts and refined through pretesting examining content validity provides a preliminary measure for assessing patient-report of health status in NMO. Further evaluation is proposed including sensitivity to clinical change, and international contributions to evaluating the measure are encouraged. PMID:27694484
Gibbons Chris J
Full Text Available Abstract Background The objective of this research was to develop a disease-specific measure for fatigue in patients with motor neurone disease (MND by generating data that would fit the Rasch measurement model. Fatigue was defined as reversible motor weakness and whole-body tiredness that was predominantly brought on by muscular exertion and was partially relieved by rest. Methods Qualitative interviews were undertaken to confirm the suitability of a previously identified set of 52 neurological fatigue items as relevant to patients with MND. Patients were recruited from five U.K. MND clinics. Questionnaires were administered during clinic or by post. A sub-sample of patients completed the questionnaire again after 2-4 weeks to assess test-retest validity. Exploratory factor analyses and Rasch analysis were conducted on the item set. Results Qualitative interviews with ten MND patients confirmed the suitability of 52 previously identified neurological fatigue items as relevant to patients with MND. 298 patients consented to completing the initial questionnaire including this item set, with an additional 78 patients completing the questionnaire a second time after 4-6 weeks. Exploratory Factor Analysis identified five potential subscales that could be conceptualised as representing: 'Energy', 'Reversible muscular weakness' (shortened to 'Weakness', 'Concentration', 'Effects of heat' and 'Rest'. Of the original five factors, two factors 'Energy' and 'Weakness' met the expectations of the Rasch model. A higher order fatigue summary scale, consisting of items from the 'Energy' and 'Weakness' subscales, was found to fit the Rasch model and have acceptable unidimensionality. The two scales and the higher order summary scale were shown to fulfil model expectations, including assumptions of unidimensionality, local independency and an absence of differential item functioning. Conclusions The Neurological Fatigue Index for MND (NFI-MND is a simple, easy
Donath, Carolin; Dorscht, Lisa; Graessel, Elmar; Sittl, Reinhard; Schoen, Christoph
There is a need for a way to measure success in multi-modal pain therapy that researchers and clinicians can agree upon. According to developments in health services research, operationalizing success should take patient-reported outcomes into account. We will present a success criterion for pain therapy that combines different patient-reported variables and includes validity measures. The usable criterion should be part of a statistically significant and satisfactory model identifying predictors of successful pain therapy. Routine data from 375 patients treated with multi-modal pain therapy from 2008 to 2013 were used. The change scores of five constructs were used for the combined success criterion: pain severity, disability due to pain, depressiveness, and physical- and mental-health-related quality of life. According to the literature, an improvement of at least ½ standard deviation was required on at least four of the five constructs to count as successful. A three-step analytical approach including multiple binary logistic regression analysis was chosen to identify the predictors of therapy success with the success criterion as the dependent variable. A total of 58.1% of the patients were classified as successful. Convergent and predictive validity data show significant correlations between the criterion and established instruments, while discriminative validity could also be shown. A multiple binary logistic regression analysis confirmed the feasibility; a significant model (Chi(2) (8) = 52.585; p multi-modal pain therapy. The criterion was based on basic constructs used in pain therapy and used widespread validated self-rating instruments. Thus, it should be easy to transfer this criterion to other institutions.
Basch, Ethan; Geoghegan, Cindy; Coons, Stephen Joel; Gnanasakthy, Ari; Slagle, Ashley F; Papadopoulos, Elektra J; Kluetz, Paul G
Data reported directly by patients about how they feel and function are rarely included in oncology drug labeling in the United States, in contrast to Europe and to nononcology labeling in the United States, where this practice is more common. Multiple barriers exist, including challenges unique to oncology trials, and industry's concerns regarding cost, logistical complexities, and the Food and Drug Administration's (FDA's) rigorous application of its 2009 guidance on the use of patient-reported outcome (PRO) measures. A panel consisting of representatives of industry, FDA, the PRO Consortium, clinicians, and patients was assembled at a 2014 workshop cosponsored by FDA to identify practical recommendations for overcoming these barriers. Key recommendations included increasing proactive encouragement by FDA to clinical trial sponsors for including PROs in drug development programs; provision of comprehensive PRO plans by sponsors to FDA early in drug development; promotion of an oncology-specific PRO research agenda; development of an approach to existing ("legacy") PRO measures, when appropriate (focused initially on symptoms and functional status); and increased FDA and industry training in PRO methodology. FDA has begun implementing several of these recommendations.
Worth, Allison; Hammersley, Victoria; Knibb, Rebecca; Flokstra-de-Blok, Bertine; DunnGalvin, Audrey; Walker, Samantha; Dubois, Anthony EJ; Sheikh, Aziz
BACKGROUND: Patient-reported outcome measures (PROMs) are measures of the outcome of treatment(s) reported directly by the patient or carer. There is increasing international policy interest in using these to assess the impact of clinical care. AIMS: To identify suitably validated PROMs for asthma a
Gater, Adam; Kitchen, Helen; Heron, Louise; Pollard, Catherine; Håkan-Bloch, Jonas; Højbjerre, Lise; Hansen, Brian Bekker; Strandberg-Larsen, Martin
The primary objective of this review is to develop a conceptual model for Crohn's disease (CD) outlining the disease burden for patients, healthcare systems and wider society, as reported in the scientific literature. A search was conducted using MEDLINE, PsycINFO, EconLit, Health Economic Evaluation Database and Centre for Reviews and Dissemination databases. Patient-reported outcome (PRO) measures widely used in CD were reviewed according to the US FDA PRO Guidance for Industry. The resulting conceptual model highlights the characterization of CD by gastrointestinal disturbances, extra-intestinal and systemic symptoms. These symptoms impact physical functioning, ability to complete daily activities, emotional wellbeing, social functioning, sexual functioning and ability to work. Gaps in conceptual coverage and evidence of reliability and validity for some PRO measures were noted. Review findings also highlight the substantial direct and indirect costs associated with CD. Evidence from the literature confirms the substantial burden of CD to patients and wider society; however, future research is still needed to further understand burden from the perspective of patients and to accurately understand the economic burden of disease. Challenges with existing PRO measures also suggest the need for future research to refine or develop new measures.
Full Text Available Abstract Background Outcome measurement in shoulder surgery is essential to evaluate the patient safety and treatment efficiency. Currently this is jeopardized by the fact that most patient-reported self-assessment instruments are not comparable. Hence, the aim was to develop a reliable self-assessment questionnaire which allows an easy follow-up of patients. The questionnaire also allows the calculation of 3 well established scoring systems, i.e. the Shoulder Pain and Disability Index (SPADI, the Constant-Murley Score (CMS, and the Disabilities of the Arm, Shoulder and Hand (DASH Score. The subjective and objective items of these three systems were condensed into a single 30-questions form and validated against the original questionnaires. Methods A representative collective of patients of our shoulder clinic was asked to fill in the newly designed self-assessment Munich Shoulder Questionnaire (MSQ. At the same time, the established questionnaires for self-assessment of CONSTANT, SPADI and DASH scores were handed out. The obtained results were compared by linear regression analysis. Results Fifty one patients completed all questionnaires. The correlation coefficients of the results were r = 0.91 for the SPADI, r = -0.93 for the DASH and r = 0.94 for the CMS scoring system, respectively. Conclusions We developed an instrument which allows a quantitative self-assessment of shoulder function. It provides compatible data sets for the three most popular shoulder function scoring systems by one single, short 30-item. This instrument can be used by shoulder surgeons to effectively monitor the outcome, safety and quality of their treatment and also compare the results to published data in the literature.
Johnson, Colin D; Arbuckle, Rob; Bonner, Nicola; Connett, Gary; Dominguez-Munoz, Enrique; Levy, Philippe; Staab, Doris; Williamson, Nicola; Lerch, Markus M
Pancreatic exocrine insufficiency (PEI) affects patients with chronic pancreatitis (CP) and cystic fibrosis (CF) who produce insufficient digestive pancreatic enzymes. Common symptoms include steatorrhoea, diarrhea, and abdominal pain. The objective of the study was to develop and test the content validity of a patient-reported outcome (PRO) instrument assessing PEI symptoms and their impact on health-related quality of life. Instrument development was supported by a literature review, expert physician interviews (n = 10: Germany 4, UK 3, France 3), and exploratory, qualitative, concept-elicitation interviews with patients with CF and CP with PEI (n = 61: UK 29, Germany 18, France 14) and expert physicians (n = 10). Cognitive debriefing of the draft instrument was then performed with patients with PEI (n = 37: UK 24, Germany 8, France 5), and feasibility was assessed with physicians (n = 3). For all interviews, verbatim transcripts were qualitatively analysed using thematic analysis methods and Atlas.ti computerized qualitative software. All themes were data driven rather than a priori. Patient interviews elicited symptoms and impacts not reported in the literature. Six symptom concepts emerged: pain, bloating, bowel symptoms, nausea/vomiting, eating problems, and tiredness/fatigue. Six impact domains were also identified. A 45-item instrument was developed in English, French, and German for testing in cognitive debriefing patient interviews. Following cognitive debriefing, 18 items were deleted. Rigorous qualitative patient research and expert clinical input supported development of a PEI-specific PRO with the potential to aid management and monitoring of unmet needs among patients with PEI. The next step is to perform psychometric evaluation of the resulting instrument.
Full Text Available Abstract Background Capturing dimensions of physical activity relevant to patients may provide a unique perspective for clinical studies of chronically ill patients. However, the quality of the development of existing instruments is uncertain. The aim of this systematic review was to assess the development process of patient-reported outcome (PRO instruments including their initial validation to measure physical activity in chronically ill or elderly patient populations. Methods We conducted a systematic literature search of electronic databases (Medline, Embase, Psychinfo, Cinahl and hand searches. We included studies describing the original development of fully structured instruments measuring dimensions of physical activity or related constructs in chronically ills or elderly. We broadened the population to elderly because they are likely to share physical activity limitations. At least two reviewers independently conducted title and abstract screening and full text assessment. We evaluated instruments in terms of their aim, items identification and selection, domain development, test-retest reliability, internal consistency, validity and responsiveness. Results Of the 2542 references from the database search and 89 from the hand search, 103 full texts which covered 104 instruments met our inclusion criteria. For almost half of the instruments the authors clearly described the aim of the instruments before the scales were developed. For item identification, patient input was used in 38% of the instruments and in 32% adaptation of existing scales and/or unsystematic literature searches were the only sources for the generation of items. For item reduction, in 56% of the instruments patient input was used and in 33% the item reduction process was not clearly described. Test-retest reliability was assessed for 61%, validity for 85% and responsiveness to change for 19% of the instruments. Conclusions Many PRO instruments exist to measure
Rolfson, Ola; Eresian Chenok, Kate; Bohm, Eric
The International Society of Arthroplasty Registries (ISAR) Steering Committee established the Patient-Reported Outcome Measures (PROMs) Working Group to convene, evaluate, and advise on best practices in the selection, administration, and interpretation of PROMs and to support the adoption and u...
Brouwers, Corline; Denollet, Johan; de Jonge, Nicolaas;
Technological advancements of left ventricular assist devices (LVAD) have created today's potential for extending the lives of patients with end-stage heart failure. Few studies have examined the effect of LVAD therapy on patient-reported outcomes (PROs), such as health status, quality of life...
Juhl, Carsten Bogh; Lund, Hans; Guyatt, GH
Title A hierarchy of patient-reported outcome measures for meta-analysis of knee osteoarthritis trials: empirical evidence from a survey of high impact journals Objective To develop a prioritized list for extracting patient-reported outcomes (PROs) measuring pain and disability for meta-analyses ......Title A hierarchy of patient-reported outcome measures for meta-analysis of knee osteoarthritis trials: empirical evidence from a survey of high impact journals Objective To develop a prioritized list for extracting patient-reported outcomes (PROs) measuring pain and disability for meta......-analyses in knee osteoarthritis (OA). Methods A systematic literature search was conducted in high impact factor journals. Eligible were randomized controlled trials, using two or more PROs measuring pain or disability. A prioritized list was developed based on the capacity to discriminate between intervention...
Reeve, Bryce B.; Mitchell, Sandra A.; Clauser, Steven B.; Minasian, Lori M.; Dueck, Amylou C.; Mendoza, Tito R.; Hay, Jennifer; Atkinson, Thomas M.; Abernethy, Amy P.; Bruner, Deborah W.; Cleeland, Charles S.; Sloan, Jeff A.; Chilukuri, Ram; Baumgartner, Paul; Denicoff, Andrea; St. Germain, Diane; O’Mara, Ann M.; Chen, Alice; Kelaghan, Joseph; Bennett, Antonia V.; Sit, Laura; Rogak, Lauren; Barz, Allison; Paul, Diane B.; Schrag, Deborah
The standard approach for documenting symptomatic adverse events (AEs) in cancer clinical trials involves investigator reporting using the National Cancer Institute’s (NCI’s) Common Terminology Criteria for Adverse Events (CTCAE). Because this approach underdetects symptomatic AEs, the NCI issued two contracts to create a patient-reported outcome (PRO) measurement system as a companion to the CTCAE, called the PRO-CTCAE. This Commentary describes development of the PRO-CTCAE by a group of multidisciplinary investigators and patient representatives and provides an overview of qualitative and quantitative studies of its measurement properties. A systematic evaluation of all 790 AEs listed in the CTCAE identified 78 appropriate for patient self-reporting. For each of these, a PRO-CTCAE plain language term in English and one to three items characterizing the frequency, severity, and/or activity interference of the AE were created, rendering a library of 124 PRO-CTCAE items. These items were refined in a cognitive interviewing study among patients on active cancer treatment with diverse educational, racial, and geographic backgrounds. Favorable measurement properties of the items, including construct validity, reliability, responsiveness, and between-mode equivalence, were determined prospectively in a demographically diverse population of patients receiving treatments for many different tumor types. A software platform was built to administer PRO-CTCAE items to clinical trial participants via the internet or telephone interactive voice response and was refined through usability testing. Work is ongoing to translate the PRO-CTCAE into multiple languages and to determine the optimal approach for integrating the PRO-CTCAE into clinical trial workflow and AE analyses. It is envisioned that the PRO-CTCAE will enhance the precision and patient-centeredness of adverse event reporting in cancer clinical research. PMID:25265940
Basch, Ethan; Reeve, Bryce B; Mitchell, Sandra A; Clauser, Steven B; Minasian, Lori M; Dueck, Amylou C; Mendoza, Tito R; Hay, Jennifer; Atkinson, Thomas M; Abernethy, Amy P; Bruner, Deborah W; Cleeland, Charles S; Sloan, Jeff A; Chilukuri, Ram; Baumgartner, Paul; Denicoff, Andrea; St Germain, Diane; O'Mara, Ann M; Chen, Alice; Kelaghan, Joseph; Bennett, Antonia V; Sit, Laura; Rogak, Lauren; Barz, Allison; Paul, Diane B; Schrag, Deborah
The standard approach for documenting symptomatic adverse events (AEs) in cancer clinical trials involves investigator reporting using the National Cancer Institute's (NCI's) Common Terminology Criteria for Adverse Events (CTCAE). Because this approach underdetects symptomatic AEs, the NCI issued two contracts to create a patient-reported outcome (PRO) measurement system as a companion to the CTCAE, called the PRO-CTCAE. This Commentary describes development of the PRO-CTCAE by a group of multidisciplinary investigators and patient representatives and provides an overview of qualitative and quantitative studies of its measurement properties. A systematic evaluation of all 790 AEs listed in the CTCAE identified 78 appropriate for patient self-reporting. For each of these, a PRO-CTCAE plain language term in English and one to three items characterizing the frequency, severity, and/or activity interference of the AE were created, rendering a library of 124 PRO-CTCAE items. These items were refined in a cognitive interviewing study among patients on active cancer treatment with diverse educational, racial, and geographic backgrounds. Favorable measurement properties of the items, including construct validity, reliability, responsiveness, and between-mode equivalence, were determined prospectively in a demographically diverse population of patients receiving treatments for many different tumor types. A software platform was built to administer PRO-CTCAE items to clinical trial participants via the internet or telephone interactive voice response and was refined through usability testing. Work is ongoing to translate the PRO-CTCAE into multiple languages and to determine the optimal approach for integrating the PRO-CTCAE into clinical trial workflow and AE analyses. It is envisioned that the PRO-CTCAE will enhance the precision and patient-centeredness of adverse event reporting in cancer clinical research. © The Author 2014. Published by Oxford University Press. All
Althof, Stanley E
The term 'Patient Reported Outcome', abbreviated as PRO, was introduced by the US Food and Drug Administration (FDA) which proposed guidance on the development and validation of PROs. Previously PROs were known as self-report diaries, event-logs, self-administered questionnaires, and clinician administered rating scales. PROs seek to capture the subjective perceptions of patients and/or partner's related to their specific symptoms, degree of bother, efficacy of a medication or psychotherapy intervention, and quality of life issues related to a specific condition. This article reviews the essential psychometric and regulatory agency requirements in the development of PROs. The constructs of reliability, various forms of validity, sensitivity, and specificity as well as concerns with translating a PRO into a different language are reviewed. Three PROs, the Premature Ejaculation Profile (PEP), the Index of Premature Ejaculation (IPE) and the Premature Ejaculation Diagnostic Tool (PEDT) all used in the assessment of premature ejaculation (PE) are discussed. These questionnaires meet or exceed all the psychometric requirements and have been employed in clinical trials and observational studies of men with PE. The article concludes on discussing some of the limitations of PRO use and recommendations for the future.
Hasler, Gregor; Hopwood, Christopher J; Jacob, Gitta A; Brändle, Laura S; Schulte-Vels, Thomas
Patient-reported outcome (PRO) refers to measures that emphasize the subjective view of patients about their health-related conditions and behaviors. Typically, PROs include self-report questionnaires and clinical interviews. Defining PROs for borderline personality disorder (BPD) is particularly challenging given the disorder's high symptomatic heterogeneity, high comorbidity with other psychiatric conditions, highly fluctuating symptoms, weak correlations between symptoms and functional outcomes, and lack of valid and reliable experimental measures to complement self-report data. Here, we provide an overview of currently used BPD outcome measures and discuss them from clinical, psychometric, experimental, and patient perspectives. In addition, we review the most promising leads to improve BPD PROs, including the DSM-5 Section III, the Recovery Approach, Ecological Momentary Assessments, and novel experimental measures of social functioning that are associated with functional and social outcomes.
Jennifer L Ridgeway
Full Text Available Jeff Sloan and colleagues describe the development of the Patient-Reported Outcomes Quality of Life (PROQOL instrument, which captures and stores patient-recorded outcomes in the medical record for patients with diabetes. Please see later in the article for the Editors' Summary.
Collins, Natalie J; Roos, Ewa M.
, and high personal and financial cost associated with THA and TKA, patient-reported outcomes are required to ensure optimal selection of patients, and that postoperative outcomes outweigh the burden associated with surgical procedures. It is clear from the information presented that clinicians need...... to consider a number of factors when selecting a "good" patientreported outcome for use in their TJA patients. Not only does the instrument need to measure dimensions appropriate for THA and TKA patients, but it also needs to have minimal administrative burden, accessibility to a variety of clinicians...... and patients, reliability, validity, and responsiveness to change. Furthermore, knowledge regarding the minimal score that patients deem to be meaningful is useful in interpreting whether a patient has experienced real improvement in their condition after surgery. It is clear that further studies are required...
Stanley E. Althof
The term ‘Patient Reported Outcome’, abbreviated as PRO, was introduced by the US Food and Drug Administration (FDA) which proposed guidance on the development and validation of PROs. Previously PROs were known as self-report diaries, event-logs, self-administered questionnaires, and clinician administered rating scales. PROs seek to capture the subjective perceptions of patients and/or partner’s related to their specific symptoms, degree of bother, efficacy of a medication or psychotherapy i...
Vojinovic, Jelena; Tincani, Angela; Sulli, Alberto; Soldano, Stefano; Andreoli, Laura; Dall'Ara, Francesca; Ionescu, Ruxandra; Pasalic, Katarina Simic; Balcune, Inete; Ferraz-Amaro, Ivan; Tlustochowicz, Małgorzata; Butrimiene, Irena; Punceviciene, Egle; Toroptsova, Natalia; Grazio, Simeon; Morovic-Vergles, Jadranka; Masaryk, Pavol; Otsa, Kati; Bernardes, Miguel; Boyadzhieva, Vladimira; Salaffi, Fausto; Cutolo, Maurizio
To collect data on vitamin D (25(OH)D) serum levels in a large number of rheumatoid arthritis (RA) patients from different European countries, to investigate their relation with disease activity, disability, quality of life, and possibly to construct a new Patient Reported Outcome (PRO) questionnaire in order to self-estimate if they are at risk for vitamin D insufficiency/deficiency-related clinical implications (D-PRO). This was a European League Against Rheumatism (EULAR) supported cross-sectional study (project No CLI064) which involved 625 RA patients (mean age 55±11years, mean disease duration 11±9years), 276 age and sex matched healthy subjects, and rheumatologists working in academic institutions or hospital centres, as well as PARE organizations (patient representatives) from 13 European countries. Serum samples for 25(OH)D level measurement were collected during winter time and analyzed in a central laboratory using chemiluminescence immunoassay (DiaSorin). Patient past medical history was recorded. RA patients were provided with three questionnaires: the Rheumatoid Arthritis Impact Diseases score (RAID), the Health Assessment Questionnaire (HAQ), and the new D-PRO questionnaire at the time of 25(OH)D serum sampling. D-PRO questionnaire consisted of three domains, Symptom Risk Score (SRS), Habitus Risk Score (HRS) and Global Risk Score (SRS+HRS=GRS), constructed with items possibly related to vitamin D deficiency. D-PRO was correlated with both clinical and PRO scores. DAS28-CRP was also evaluated. Statistical analysis was performed by non parametric tests. Mean serum concentration of 25(OH)D in RA patients (17.62±9.76ng/ml) was found significantly lower if compared to the levels obtained in matched controls (18.95±9.45ng/ml) (p=0.01), with statistically significant differences among several European countries. Negative correlations were found between 25(OH)D serum levels and DAS28-CRP (pPRO questionnaire. This first multicentre European survey add
Juhl, Carsten; Lund, Hans; Roos, Ewa M
Objectives. To develop a prioritised list based on responsiveness for extracting patient-reported outcomes (PROs) measuring pain and disability for performing meta-analyses in knee osteoarthritis (OA). Methods. A systematic search was conducted in 20 highest impact factor general and rheumatology...
Full Text Available International and national health policy seeks to increase service user and carer involvement in mental health care planning, but suitable user-centred tools to assess the success of these initiatives are not yet available. The current study describes the development of a new reliable and valid, interval-scaled service-user and carer reported outcome measure for quantifying user/carer involvement in mental health care planning. Psychometric development reduced a 70-item item bank to a short form questionnaire using a combination of Classical Test, Mokken and Rasch Analyses. Test-retest reliability was calculated using t-tests of interval level scores between baseline and 2-4 week follow-up. Items were worded to be relevant to both service users and carers. Nine items were removed following cognitive debriefing with a service user and carer advisory group. An iterative process of item removal reduced the remaining 61 items to a final 14-item scale. The final scale has acceptable scalability (Ho = .69, reliability (alpha = .92, fit to the Rasch model (χ2(70 = 97.25, p = .02, and no differential item functioning or locally dependent items. Scores remained stable over the 4 week follow-up period, indicating good test-retest reliability. The 'Evaluating the Quality of User and Carer Involvement in Care Planning (EQUIP' scale displays excellent psychometric properties and is capable of unidimensional linear measurement. The scale is short, user and carer-centred and will be of direct benefit to clinicians, services, auditors and researchers wishing to quantify levels of user and carer involvement in care planning.
Santana, MJ; Haverman, L; Absolom, K; Takeuchi, E.; Feeny, D; Grootenhuis, M; Velikova, G
Introduction: Patient-reported outcome measures (PROs) were originally developed for comparing groups of people in clinical trials and population studies, and the results were used to support treatment recommendations or inform health policy, but there was not direct benefit for the participants providing PROs data. However, as the experience in using those measures increased, it became obvious the clinical value in using individual patient PROs profiles in daily practice to identify/monitor ...
Thompson, E. A.; Mathie, R. T.; Baitson, E.S.; Barron, S J; Berkovitz, S.R.; Brands, M.; Fisher, P.; Kirby, T.M.; Leckridge, R.W.; Mercer, S.W.; Nielsen, H J; Ratsey, D.H.K.; Reilly, D.; Roniger, H.; Whitmarsh, T.E.
We report findings from a pilot data collection study within a programme of quality assurance, improvement and development across all five homeopathic hospitals in the UK National Health Service (NHS).\\ud \\ud Aims (1) To pilot the collection of clinical data in the homeopathic hospital outpatient setting, recording patient-reported outcome since first appointment; (2) to sample the range of medical complaints that secondary-care doctors treat using homeopathy, and thus identify the nature and...
Magasi, Susan; Ryan, Gery; Revicki, Dennis; Lenderking, William; Hays, Ron D; Brod, Meryl; Snyder, Claire; Boers, Maarten; Cella, David
Content validity of patient-reported outcome measures (PROs) has been a focus of debate since the 2006 publication of the U.S. FDA Draft Guidance for Industry in Patient Reported Outcome Measurement. Under the auspices of the Patient Reported Outcomes Measurement Information System (PROMIS) initiative, a working meeting on content validity was convened with leading PRO measurement experts. Platform presentations and participant discussion highlighted key issues in the content validity debate, including inconsistency in the definition and evaluation of content validity, the need for empirical research to support methodological approaches to the evaluation of content validity, and concerns that continual re-evaluation of content validity slows the pace of science and leads to the proliferation of study-specific PROs. We advocate an approach to the evaluation of content validity, which includes meticulously documented qualitative and advanced quantitative methods. To advance the science of content validity in PROs, we recommend (1) development of a consensus definition of content validity; (2) development of content validity guidelines that delineate the role of qualitative and quantitative methods and the integration of multiple perspectives; (3) empirical evaluation of generalizability of content validity across applications; and (4) use of generic measures as the foundation for PROs assessment.
Watt, Torquil; Bjorner, Jakob Bue; Groenvold, Mogens;
To establish a reliable and valid scale structure of a patient-reported outcome measuring thyroid-specific quality of life.......To establish a reliable and valid scale structure of a patient-reported outcome measuring thyroid-specific quality of life....
Steven R. Feldman
Conclusion: The PSSD, developed according to the Food and Drug Administration PRO Guidance, assesses severity of symptoms and signs commonly associated with plaque PsO. Its measurement properties are currently being evaluated.
Kemp, Joanne L; Collins, Natalie J; Roos, Ewa M.
Patient-reported outcomes (PROs) are considered the gold standard when evaluating outcomes in a surgical population. While the psychometric properties of some PROs have been tested, the properties of newer PROs in patients undergoing hip arthroscopic surgery remain somewhat unknown.......Patient-reported outcomes (PROs) are considered the gold standard when evaluating outcomes in a surgical population. While the psychometric properties of some PROs have been tested, the properties of newer PROs in patients undergoing hip arthroscopic surgery remain somewhat unknown....
Anne M. Skalicky
Full Text Available Objective. The objective of this research was to develop a disease-specific symptom inventory for soft tissue sarcoma. Methods. Literature review and clinical expert and patient interviews were conducted to determine disease-specific symptoms important to patients with one of the four STS subtypes. Clinical experts identified the most relevant STS symptom items from the item pool developed from literature review. Concept elicitation interviews were conducted with patients to elicit their STS symptom experiences followed by a completion of the draft symptom list via web survey. A cognitive interview was conducted on the comprehension and importance of the symptom items. Results. Eighty-three symptom items were compiled and discussed with three clinical experts who identified 26 symptoms specific to the four STS subtypes. A total sample of 27 STS participants with self-reported leiomyosarcoma (74%, undifferentiated sarcoma (15%, synovial sarcoma (7%, or liposarcoma (4% diagnosis completed the web survey and 10 were interviewed. The draft 12-item STS-specific symptom inventory includes abdominal pain, pressure in abdomen, early satiety, bloating, gastrointestinal pain, muscle pain, bone pain, heavy menstrual flow, shortness of breath, chest pain, cough, and painful menstruation. Conclusion. A number of symptoms are common across STS subtypes and may form a single STS symptom inventory.
Bullinger, Monika; Quitmann, Julia
Assessing quality of life (QoL) as a patient-reported outcome in adult psychiatry poses challenges in terms of concepts, methods, and applications in research and practice. This review will outline conceptually the construct of QoL, its dimensionality, and its representation across patient groups. Methodological challenges are examined, along with principles of QoL instrument development and testing, as well as across cultures. Application of instruments in epidemiological, clinical health economics, and health services research is reviewed based on pertinent literature. Validated measures for depression, psychosis, and anxiety disorders are available in adult psychiatry, and are increasingly used in research. Still, targeted measures are lacking for many mental health conditions and only rarely are tools applied in the practice context. Progress has been made in the development of instruments that are now ready for implementation. The information to be gained is valuable for identifying patient-reported needs for and benefits of treatment.
Girgis, Afaf; Delaney, Geoff P; Arnold, Anthony; Miller, Alexis Andrew; Levesque, Janelle V; Kaadan, Nasreen; Carolan, Martin G; Cook, Nicole; Masters, Kenneth; Tran, Thomas T; Sandell, Tiffany; Durcinoska, Ivana; Gerges, Martha; Avery, Sandra; Ng, Weng; Della-Fiorentina, Stephen; Dhillon, Haryana M; Maher, Ashley
Patient-reported outcome (PRO) measures have been used widely to screen for depression, anxiety, and symptoms in cancer patients. Computer-based applications that collect patients' responses and transfer them to the treating health professional in real time have the potential to improve patient well-being and cancer outcomes. This study will test the feasibility and acceptability of a newly developed eHealth system which facilitates PRO data capture from cancer patients, data linkage and retrieval to support clinical decisions and patient self-management, and data retrieval to support ongoing evaluation and innovative research. The eHealth system is being developed in consultation with 3 overarching content-specific expert advisory groups convened for this project: the clinical advisory group, technical advisory group, and evaluation advisory group. The following work has already been completed during this phase of the study: the Patient-Reported Outcome Measures for Personalized Treatment and Care (PROMPT-Care) eHealth system was developed, patient-reported outcomes were selected (distress, symptoms, unmet needs), algorithms to inform intervention thresholds for clinical and self-management were determined, clinician PRO feedback summary and longitudinal reports were designed, and patient self-management resources were collated. PROsaiq, a custom information technology system, will transfer PRO data in real time into the hospital-based oncology information system to support clinical decision making. The PROMPT-Care system feasibility and acceptability will be assessed through patients completing PROMPT-Care assessments, participating in face-to-face cognitive interviews, and completing evaluation surveys and telephone interviews and oncology staff participating in telephone interviews. Over the course of 3 months, the system will be pilot-tested with up to 50 patients receiving treatment or follow-up care and 6 oncology staff at 2 hospitals in New South Wales
Wong Riff, Karen W Y; Tsangaris, Elena; Goodacre, Tim; Forrest, Christopher R; Pusic, Andrea L; Cano, Stefan J; Klassen, Anne F
Introduction Patient-reported outcome (PRO) instruments should be developed according to rigorous guidelines in order to provide clinically meaningful, scientifically sound measurement. Understanding the methodology behind instrument development informs the selection of the most appropriate tool. This mixed methods protocol describes the development of an internationally applicable PRO instrument, the CLEFT-Q, for evaluating outcomes of treatment for cleft lip and/or palate (CL/P). Methods and analysis The study includes three main phases that occur iteratively and interactively. In phase I, we determine what concepts are important to patients regarding their outcome. A conceptual framework for the CLEFT-Q is formed through a systematic review and an extensive international qualitative study. The systematic review ascertains what concepts have previously been measured in patients with CL/P. The qualitative study employs interpretive description and involves in-depth interviews with patients in high-income and lower-middle income countries. Preliminary items are generated from the qualitative data. Preliminary scales are then created for each theme in the framework. Cognitive debriefing interviews and expert clinician input are used to refine the scales in an iterative process. In phase II, the preliminary scales are administered to a large international group of patients with CL/P. The modern psychometric method of Rasch Measurement Theory analysis is employed to define the measurement characteristics. The preliminary scales are shortened based on these results. In phase III, further tests assess reliability, validity and responsiveness of the instrument. Ethics and dissemination The study is approved by Research Ethics Boards for each participating site. Findings from this study will be published in open access peer-reviewed journals and presented at national and international conferences. Integrated knowledge translation is employed to engage stakeholders from
Makhni, Eric C; Meadows, Molly; Hamamoto, Jason T; Higgins, John D; Romeo, Anthony A; Verma, Nikhil N
Patient reported outcomes (PROs) serve an integral role in clinical research by helping to determine the impact of clinical care as experienced by the patient. With recent initiatives in health care policy and pay for performance, outcome reporting is now recognized as a policy-driven requirement in addition to a clinical research tool. For outcome measures to satisfy these regulatory requirements and provide value in understanding disease outcomes, they must be responsive and efficient. Recent research has uncovered certain concerns regarding traditional PROs in patients with upper extremity disability and injury. These include lack of consensus regarding selection of PROs for a given diagnoses, inconsistent techniques of administration of the same PROs, and the administrative burden to patients and providers of completing these forms. To address these limitations, emphasis has been placed on streamlining the outcomes reporting process, and, as a result, the National Institutes of Health (NIH) created the Patient Reported Outcomes Measurement Information System (PROMIS). PROMIS forms were created to comprehensively and efficiently measure outcomes across multiple disease states, including orthopedics. These tools exist in computer adaptive testing and short forms with the intention of more efficiently measuring outcomes compared with legacy PROs. The goals of this review are to highlight the main components of PROMIS reporting tools and identify recent use of the scores in the upper extremity literature. The review will also highlight the research and health policy potentials and limitations of implementing PROMIS into everyday orthopedic practice. Copyright © 2017 Journal of Shoulder and Elbow Surgery Board of Trustees. Published by Elsevier Inc. All rights reserved.
Patel, Darshan P; Elliott, Sean P; Stoffel, John T; Brant, William O; Hotaling, James M; Myers, Jeremy B
To describe existing bladder and bowel specific quality of life (QoL) measurement tools, QoL in patients with multiple sclerosis (MS), spinal cord injury (SCI), Parkinson's Disease (PD), stroke, or spina bifida (SB) affected by bladder or bowel dysfunction, and the impact of specific bladder and bowel management on QoL. We performed a systematic review in PubMed/Medline databases in accordance with the PRISMA statement for English publications between January 1, 2000 and January 1, 2014. Articles were first screened based on their abstract and select full-text articles were then reviewed for eligibility. Articles with no QoL or PROM assessing urinary or bowel dysfunction were excluded. Risk of bias assessment included randomization, incomplete outcomes data, selective outcomes reporting, and other biases. All articles were graded using the Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) system as per the Cochrane Handbook for Systematic Reviews of Interventions. The most common QoL measurement tool for urinary and bowel dysfunction was the Medical Outcomes Study SF-36. Twelve (24%) studies used only non-validated QoL questionnaires. Only three urinary or bowel specific QoL measures were found: the Qualiveen questionnaire, the FICQoL, and the QoL-BM. Several studies identified instances were clinical and patient-reported outcomes were inconsistent particularly with indwelling urinary catheter usage and reconstructive surgery. Additionally, certain clinical outcomes surrogates commonly used as primary outcomes measures may not correlate with the patient reported outcomes (PRO). Current PRO measures (PROM) and QoL assessments are heterogeneous and several inconsistencies in clinical and PRO for various management options exist. Standardized PROM will help identify optimal bladder and bowel management for patients with neurologic conditions. © 2014 Wiley Periodicals, Inc.
Rasmussen, Jeppe V; Polk, Anne; Brorson, Stig;
PURPOSE: We used patient-reported outcome and risk of revision to compare hemiarthroplasty (HA) with total shoulder arthroplasty (TSA) and stemmed hemiarthroplasty (SHA) with resurfacing hemiarthroplasty (RHA) in patients with glenohumeral osteoarthritis. PATIENTS AND METHODS: We included all pat...
Jolly, Meenakshi; Pickard, A Simon; Block, Joel A; Kumar, Rajan B; Mikolaitis, Rachel A; Wilke, Caitlyn T; Rodby, Roger A; Fogg, Louis; Sequeira, Winston; Utset, Tammy O; Cash, Thomas F; Moldovan, Iona; Katsaros, Emmanuel; Nicassio, Perry; Ishimori, Mariko L; Kosinsky, Mark; Merrill, Joan T; Weisman, Michael H; Wallace, Daniel J
Systemic lupus erythematosus (SLE) can significantly affect both health and non-health-related quality of life (HRQOL and non-HRQOL). However, of the existent published patient-reported outcome (PRO) tools, none were developed from US patients, an ethnically diverse population. Furthermore, these tools do not address men with SLE or assess non-HRQOL issues. Herein, we present the development and validation of the Lupus Patient-Reported Outcome tool (LupusPRO) and discuss its clinical utility and research value compared with other PRO tools currently available for SLE. Beginning with a conceptual framework, items for LupusPRO were generated using feedback from women and men with SLE. The tool underwent iterations based on patient feedback and clinimetric and psychometric analyses. Validity (content, construct, and criterion) and reliability (internal consistency and test-retest) for the 44-item LupusPRO tool are presented. Consistent with the conceptual framework, items were identified that were related to HRQOL and non-HRQOL constructs. HRQOL domains included (1) lupus symptoms; (2) physical health (physical function, role physical); (3) pain-vitality; (4) emotional health (emotional function and role emotional); (5) body image; (6) cognition; (7) procreation; and (8) lupus medications. Non-HRQOL domains were (1) available social support and coping; (2) desires-goals; and (3) satisfaction with medical care. Internal consistency reliability (0.68-0.94), test-retest reliability (0.55-0.92), content, construct (r > 0.50 with SF-36), and criterion (r > -0.35 with disease activity) validity were fair to good. LupusPRO is a valid and reliable disease-targeted patient-reported health outcome tool that is generalizable to SLE patients in the United States of varied ethnic backgrounds and either gender. Copyright © 2012. Published by Elsevier Inc.
Larsen, Kristian; Hansen, Torben B; Søballe, Kjeld;
PURPOSE: The purpose of this study was to describe patient-related functional outcomes after fast-track total knee arthroplasty and unicompartmental knee arthroplasty. Furthermore, we wanted to assess physical areas where an additional need for rehabilitation could be identified, and finally, we...
Leffler, Daniel A; Acaster, Sarah; Gallop, Katy; Dennis, Melinda; Kelly, Ciarán P; Adelman, Daniel C
Celiac disease is a chronic inflammatory condition with wide ranging effects on individual's lives caused by a combination of symptoms and the burden of adhering to a gluten-free diet (GFD). To further understand patients' experience of celiac disease, the impact it has on health-related quality of life (HRQOL), and to develop a conceptual model describing this impact. Adults with celiac disease on a GFD reporting symptoms within the previous 3 months were included; patients with refractory celiac disease and confounding medical conditions were excluded. A semistructured discussion guide was developed exploring celiac disease symptoms and impact on patients' HRQOL. An experienced interviewer conducted in-depth interviews. The data set was coded and analyzed using thematic analysis to identify concepts, themes, and the inter-relationships between them. Data saturation was monitored and concepts identified formed the basis of the conceptual model. Twenty-one participants were recruited, and 32 distinct gluten-related symptoms were reported and data saturation was reached. Analysis identified several themes impacting patients' HRQOL: fears and anxiety, day-to-day management of celiac disease, physical functioning, sleep, daily activities, social activities, emotional functioning, and relationships. The conceptual model highlights the main areas of impact and the relationships between concepts. Both symptoms and maintaining a GFD have a substantial impact on patient functioning and HRQOL in adults with celiac disease. The conceptual model derived from these data may help to design future patient-reported outcomes as well as interventions to improve the quality of life in an individual with celiac disease. Copyright © 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.
Li, Fuzhong; Harmer, Peter; Liu, Yu; Eckstrom, Elizabeth; Fitzgerald, Kathleen; Stock, Ronald; Chou, Li-Shan
A previous randomized, controlled trial of tai chi showed improvements in objectively measured balance and other motor-related outcomes in patients with Parkinson's disease. This study evaluated whether patient-reported outcomes could be improved through exercise interventions and whether improvements were associated with clinical outcomes and exercise adherence. In a secondary analysis of the tai chi trial, patient-reported and clinical outcomes and exercise adherence measures were compared between tai chi and resistance training and between tai chi and stretching exercise. Patient-reported outcome measures were perceptions of health-related benefits resulting from participation, assessed by the Parkinson's Disease Questionnaire (PDQ-8) and Vitality Plus Scale (VPS). Clinical outcome measures included motor symptoms, assessed by a modified Unified Parkinson's Disease Rating Scale-Motor Examination (UPDRS-ME) and a 50-foot speed walk. Information on continuing exercise after the structured interventions were terminated was obtained at a 3-month postintervention follow-up. Tai chi participants reported significantly better improvement in the PDQ-8 (-5.77 points, P = 0.014) than did resistance training participants and in PDQ-8 (-9.56 points, P tai chi, patient-reported improvement in the PDQ-8 and VPS was significantly correlated with their clinical outcomes of UPDRS-ME and a 50-foot walk, but these correlations were not statistically different from those shown for resistance training or stretching. However, patient-reported outcomes from tai chi training were associated with greater probability of continued exercise behavior than were either clinical outcomes or patient-reported outcomes from resistance training or stretching. Tai chi improved patient-reported perceptions of health-related benefits, which were found to be associated with a greater probability of exercise adherence. The findings indicate the potential of patient perceptions to drive exercise
Zraick, Richard I; Atcherson, Samuel R; Brown, Angela M
The purpose of this study was to examine the readability of several published patient-reported outcome (PRO) questionnaires for use with persons who stutter, and to compare the readability results to existing data about average reading levels for English-speaking adults living in the United States. Published PRO questionnaires were identified that are traditionally completed by persons who stutter in a self-administered format. Reading grade levels were analyzed using the Flesch Reading Ease, FOG, and FORCAST formulas as computed by a readability calculations software package. Descriptive statistics were computed across the questionnaires. The results of this study demonstrate that many of the PRO questionnaires exceeded the fifth to sixth grade reading levels recommended by health literacy experts. The clinician should consider the average reading level needed to understand a particular PRO questionnaire when administering it to a patient or their proxy. Likewise, developers of PRO questionnaires should consider reading level of respondents and include information about this when reporting psychometric data. The reader will get an overview over the literature on patient-reported outcome (PRO) questionnaires and their use with persons who stutter and will be able to: (1) define readability, (2) describe how reading levels are determined for a given PRO questionnaire, (3) list the strengths and limitations of readability assessment in the evaluation of persons who stutter and (4) analyze the role of readability assessment in future PRO questionnaire development. Copyright © 2011 Elsevier Inc. All rights reserved.
Christina Lampe MD
Full Text Available This cross-sectional analysis assessed the correlation between patient-reported outcomes (PROs and clinical outcomes in 24 German patients with Morquio A. Clinical outcomes included 6-minute walk test (6MWT, 3-minute stair climb (3MSC test, and joint range of motion as measures for endurance/mobility, forced vital capacity (FVC and maximum voluntary ventilation (MVV as measures for respiratory function, and height as an important manifestation. The PROs included the EuroQoL (EQ 5D-5L (EQ5D-5L, to measure health-related QoL (HRQoL, and patients’ rating of their ability to walk, climb, or breathe. In adults, endurance and pulmonary function measures and height showed strong and statistically significant correlation with the patients’ EQ5D-5L (6MWT: R = .884, 3MSC test: R = .852, FVC: R = .815, MVV: R = .825, height: R = .842. The adult patients’ rating of their ability to walk and climb also correlated strongly with 6MWT (R = .839 and 3MSC test (R = .700 results. Improvements in these clinical outcomes may be robust surrogate parameters of a better EQ5D-5L/HRQoL in patients with Morquio A.
Full Text Available Abstract Background Two Phase III randomized controlled clinical trials were conducted to assess the efficacy, safety, and tolerability of weekly subcutaneous administration of efalizumab for the treatment of psoriasis. Patient reported measures of psoriasis-related functionality and health-related quality of life and of psoriasis-related symptom assessments were included as part of the trials. Objective To assess the reliability, validity, and responsiveness of the patient reported outcome measures that were used in the trials – the Dermatology Life Quality Index (DLQI, the Psoriasis Symptom Assessment (PSA Scale, and two itch measures, a Visual Analog Scale (VAS and the National Psoriasis Foundation (NPF itch measure. Methods Subjects aged 18 to 70 years with moderate to severe psoriasis for at least 6 months were recruited into the two clinical trials (n = 1095. Internal consistency reliability was evaluated for all patient reported outcomes at baseline and at 12 weeks. Construct validity was evaluated by relations among the different patient reported outcomes and between the patient reported outcomes and the clinical assessments (Psoriasis Area and Severity Index; Overall Lesion Severity Scale; Physician's Global Assessment of Change assessed at baseline and at 12 weeks, as was the change over the course of the 12 week portion of the trial. Results Internal consistency reliability ranged from 0.86 to 0.95 for the patient reported outcome measures. The patient reported outcome measures were all shown to have significant construct validity with respect to each other and with respect to the clinical assessments. The four measures also demonstrated significant responsiveness to change in underlying clinical status of the patients over the course of the trial, as measured by the independently assessed clinical outcomes. Conclusions The DLQI, the PSA, VAS, and the NPF are considered useful tools for the measurement of dermatology
S. Nicole Culos-Reed
Full Text Available Limited research suggests yoga may be a viable gentle physical activity option with a variety of health-related quality of life, psychosocial and symptom management benefits. The purpose of this review was to determine the clinical significance of patient-reported outcomes from yoga interventions conducted with cancer survivors. A total of 25 published yoga intervention studies for cancer survivors from 2004–2011 had patient-reported outcomes, including quality of life, psychosocial or symptom measures. Thirteen of these studies met the necessary criteria to assess clinical significance. Clinical significance for each of the outcomes of interest was examined based on 1 standard error of the measurement, 0.5 standard deviation, and relative comparative effect sizes and their respective confidence intervals. This review describes in detail these patient-reported outcomes, how they were obtained, their relative clinical significance and implications for both clinical and research settings. Overall, clinically significant changes in patient-reported outcomes suggest that yoga interventions hold promise for improving cancer survivors' well-being. This research overview provides new directions for examining how clinical significance can provide a unique context for describing changes in patient-reported outcomes from yoga interventions. Researchers are encouraged to employ indices of clinical significance in the interpretation and discussion of results from yoga studies.
Rosenlund, Signe; Broeng, Leif; Larsen, Anders Holsgaard
. In this randomized controlled trial, we tested the hypothesis that patient-reported outcomes are better in patients who have undergone total hip arthroplasty (THA) with PA than in those who have undergone THA with LA, 12 months postoperatively. Patients and methods — 80 patients with hip osteoarthritis (mean age 61......Background and purpose — Criticism of the lateral approach (LA) for hip arthroplasty is mainly based on the risk of poor patient-reported outcomes compared to the posterior approach (PA). However, there have been no controlled studies comparing patient-reported outcomes between them......-Pain, HOOS-Quality-Of-Life, EQ-5D, UCLA Activity Score, and limping. Results — We found no statistically signifi cant difference in the improvements in HOOS-PS between the treatment groups at 12-month follow-up. All secondary outcomes showed similar results except for limping, where PA patients improved...
Larsen, Michael Due; Lose, Gunnar; Guldberg, Rikke
INTRODUCTION AND HYPOTHESIS: In order to assess the outcome following surgery for urinary incontinence (UI) and pelvic organ prolapse (POP) the importance of patient-reported outcome measures, in addition to the clinical objective measures, has been recognised. The International Consultation...
Koo John; Thompson Christine; Stone Stephen P; Bresnahan Brian W; Shikiar Richard; Revicki Dennis A
Abstract Background Two Phase III randomized controlled clinical trials were conducted to assess the efficacy, safety, and tolerability of weekly subcutaneous administration of efalizumab for the treatment of psoriasis. Patient reported measures of psoriasis-related functionality and health-related quality of life and of psoriasis-related symptom assessments were included as part of the trials. Objective To assess the reliability, validity, and responsiveness of the patient reported outcome m...
Full Text Available Paul Jones,1 Marc Miravitlles,2 Thys van der Molen,3 Karoly Kulich41Division of Clinical Science, University of London, London, UK; 2Institut d'Investigacions Biomèdiques August Pi i Sunyer, Hospital Clínic, Ciber de Enfermedades Respiratorias, Barcelona, Spain; 3Department of Primary Care, University Medical Centre Groningen, Groningen, The Netherlands; 4Novartis Pharma AG, Basel, SwitzerlandAbstract: Patients with chronic obstructive pulmonary disease (COPD present with a variety of symptoms and pathological consequences. Although primarily viewed as a respiratory disease, COPD has both pulmonary and extrapulmonary effects, which have an impact on many aspects of physical, emotional, and mental well-being. Traditional assessment of COPD relies heavily on measuring lung function, specifically forced expiratory volume in 1 second (FEV1. However, the evidence suggests that FEV1 is a relatively poor correlate of symptoms such as breathlessness and the impact of COPD on daily life. Furthermore, many consequences of the disease, including anxiety and depression and the ability to perform daily activities, can only be described and reported reliably by the patient. Thus, in order to provide a comprehensive view of the effects of interventions in clinical trials, it is essential that spirometry is accompanied by assessments using patient-reported outcome (PRO instruments. We provide an overview of patient-reported outcome concepts in COPD, such as breathlessness, physical functioning, and health status, and evaluate the tools used for measuring these concepts. Particular attention is given to the newly developed instruments emerging in response to recent regulatory guidelines for the development and use of PROs in clinical trials. We conclude that although data from the development and validation of these new PRO instruments are emerging, to build the body of evidence that supports the use of a new instrument takes many years. Furthermore, new
Fehnel, Sheri; DeMuro, Carla; McLeod, Lori; Coon, Cheryl; Gnanasakthy, Ari
Release of the US FDA patient-reported outcome (PRO) guidance raised expectations within the pharmaceutical industry for the use of PRO measures in support of labeling claims. The FDA developed the guidance with admirable intent, and the recommendations within this document are based on sound scientific principles. However, implementation of the guidance has been somewhat inconsistent within the Study Endpoints and Label Development (SEALD) and across the various FDA-reviewing divisions. Industry and regulatory bodies need to work toward gaining common ground to best support registration of treatments that could extend patients' lives, reduce symptoms, and/or improve health-related quality of life. PROs are valuable tools in communicating these messages, and realistic implementation of the FDA PRO Guidance may truly facilitate this process.
Full Text Available INTRODUCTION: The ability of patients to finalise their affairs at the end of life is an often neglected aspect of quality of life (QOL measurement in palliative care effectiveness research despite compelling evidence of the high value patients place on this domain. OBJECTIVE: This paper describes the preliminary development and evaluation of a new, single-item, end-of-life patient-reported outcome measure (EOLPRO designed to capture changes in the ability of patients to finalise their affairs at the end of life. METHODS: Cognitive interviews with purposively sampled Australian palliative care patients (N = 9 were analysed thematically to explore content validity. Simultaneously, secondary analysis of data from a randomised controlled trial comparing ketamine and placebo for the management of cancer pain (N = 185 evaluated: construct validity; test-retest reliability; and responsiveness. RESULTS: Preliminary findings suggest patients interpret the new measure consistently. The EOLPRO captures the ability to complete physical tasks and finalise practical matters although it is unclear whether emotional tasks or resolution of relationship issues are considered. Personal and financial affairs should be separated to allow for differences in ability for these two types of affairs. The significant correlation between performance status and EOLPRO scores (r = 0.41, p<0.01, n = 137 and expected relationships between EOLPRO and proximity to death and constipation demonstrated construct validity. Pre- and post-treatment EOLPRO scores moderately agreed (n = 14, κ = 0.52 [95% CI 0.19, 0.84] supporting reliability. The measure's apparent lack of sensitivity to discriminate between treatment responders and non-responders may be confounded. CONCLUSION: Based on the preliminary findings, the EOLPRO should be separated into 'personal' and 'financial' affairs with further testing suggested, particularly to verify coverage and
Strasburger, Christian J; Karavitaki, Niki; Störmann, Sylvère; Trainer, Peter J; Kreitschmann-Andermahr, Ilonka; Droste, Michael; Korbonits, Márta; Feldmann, Berit; Zopf, Kathrin; Sanderson, Violet Fazal; Schwicker, David; Gelbaum, Dana; Haviv, Asi; Bidlingmaier, Martin; Biermasz, Nienke R
Background Long-acting somatostatin analogues delivered parenterally are the most widely used medical treatment in acromegaly. This patient-reported outcomes survey was designed to assess the impact of chronic injections on subjects with acromegaly. Methods The survey was conducted in nine pituitary centres in Germany, UK and The Netherlands. The questionnaire was developed by endocrinologists and covered aspects of acromegaly symptoms, injection-related manifestations, emotional and daily life impact, treatment satisfaction and unmet medical needs. Results In total, 195 patients participated, of which 112 (57%) were on octreotide (Sandostatin LAR) and 83 (43%) on lanreotide (Somatuline Depot). The majority (>70%) of patients reported acromegaly symptoms despite treatment. A total of 52% of patients reported that their symptoms worsen towards the end of the dosing interval. Administration site pain lasting up to a week following injection was the most frequently reported injection-related symptom (70% of patients). Other injection site reactions included nodules (38%), swelling (28%), bruising (16%), scar tissue (8%) and inflammation (7%). Injection burden was similar between octreotide and lanreotide. Only a minority of patients received injections at home (17%) and 5% were self-injecting. Over a third of patients indicated a feeling of loss of independence due to the injections, and 16% reported repeated work loss days. Despite the physical, emotional and daily life impact of injections, patients were satisfied with their treatment, yet reported that modifications that would offer major improvement over current care would be ‘avoiding injections’ and ‘better symptom control’. Conclusion Lifelong injections of long-acting somatostatin analogues have significant burden on the functioning, well-being and daily lives of patients with acromegaly. PMID:26744896
Sung, Lillian; Stark, Daniel; Frazier, A. Lindsay; Rosenberg, Abby R.
Purpose Peripheral neuropathy is an important, yet poorly studied, side effect of pediatric cancer treatment. There are many measures of patient-reported peripheral neuropathy in adults but very few in children. We aimed to systematically review and summarize reliable and valid patient-reported peripheral neuropathy scales used in pediatrics. Methods Four major electronic databases (Medline, Embase, EBSCO Host in Cumulative Index to Nursing and Allied Health Literature, and PsycINFO) were reviewed for studies that measured peripheral neuropathy in pediatric patients. Studies eligible for inclusion were those that described use of any patient-reported scale of peripheral neuropathy among children, adolescents, and young adults with any underlying diagnosis (not limited to cancer). Results From a total of 765 articles retrieved, 5 met eligibility criteria and were included. One was a neuropathy symptom score used in patients with diabetes, and the remaining four were in oncology patients and all were based on the total neuropathy score. All involved objective assessments conducted by trained professionals; none relied purely on patient report. Conclusions There are no validated instruments that consist solely of a patient-reported outcome measure of neuropathy in pediatrics and adolescents. Because the clinical evaluation of neuropathy requires specialized training, it is not generalizable in large studies conducted in many diverse institutions. Future studies should validate adult patient-reported neuropathy scales in pediatric and adolescent populations, or develop novel instruments designed for this population. PMID:27037813
Johnston, Donna L; Sung, Lillian; Stark, Daniel; Frazier, A Lindsay; Rosenberg, Abby R
Peripheral neuropathy is an important, yet poorly studied, side effect of pediatric cancer treatment. There are many measures of patient-reported peripheral neuropathy in adults but very few in children. We aimed to systematically review and summarize reliable and valid patient-reported peripheral neuropathy scales used in pediatrics. Four major electronic databases (Medline, Embase, EBSCO Host in Cumulative Index to Nursing and Allied Health Literature, and PsycINFO) were reviewed for studies that measured peripheral neuropathy in pediatric patients. Studies eligible for inclusion were those that described use of any patient-reported scale of peripheral neuropathy among children, adolescents, and young adults with any underlying diagnosis (not limited to cancer). From a total of 765 articles retrieved, 5 met eligibility criteria and were included. One was a neuropathy symptom score used in patients with diabetes, and the remaining four were in oncology patients and all were based on the total neuropathy score. All involved objective assessments conducted by trained professionals; none relied purely on patient report. There are no validated instruments that consist solely of a patient-reported outcome measure of neuropathy in pediatrics and adolescents. Because the clinical evaluation of neuropathy requires specialized training, it is not generalizable in large studies conducted in many diverse institutions. Future studies should validate adult patient-reported neuropathy scales in pediatric and adolescent populations, or develop novel instruments designed for this population.
Kyte, D G; Calvert, M; van der Wees, P J; ten Hove, R; Tolan, S; Hill, J C
The use of patient-reported outcome measures (PROMs) is set to rise in physiotherapy. PROMs provide additional 'patient-centred' data which is unique in capturing the patient's own opinion on the impact of their disease or disorder, and its treatment, on their life. Thus, PROMs are increasingly used by clinicians to guide routine patient care, or for the purposes of audit, and are already firmly embedded in clinical research. This article seeks to summarise the key aspects of PROM use for physiotherapists, both in routine clinical practice and in the research setting, and highlights recent developments in the field. Generic and condition-specific PROMs are defined and examples of commonly used measures are provided. The selection of appropriate PROMs, and their effective use in the clinical and research settings is discussed. Finally, existing barriers to PROM use in practice are identified and recent physiotherapy PROM initiatives, led by the Royal Dutch Society for Physical Therapy are explored.
Witter, James P
PROMIS, the Patient-Reported Outcomes Measurement Information System, is opening new possibilities to explore and learn how patient (or proxy) self-report of core symptoms and health-related quality of life can meaningfully advance clinical research and patient care. PROMIS leverages Item Response Theory to agnostically assess, across diseases and conditions or clinical settings, numerous universally applicable core "domains" of health (symptoms and functioning) from the patient perspective. Importantly, PROMIS is enabling the testing and adoption of computerized adaptive testing, which holds great potential to minimize patient burden while maximizing accuracy.
Full Text Available Abstract The exponential development of Patient-Reported Outcomes (PRO measures in clinical research has led to the creation of the Patient-Reported Outcome and Quality of Life Instruments Database (PROQOLID to facilitate the selection process of PRO measures in clinical research. The project was initiated by Mapi Research Trust in Lyon, France. Initially called QOLID (Quality of Life Instruments Database, the project's purpose was to provide all those involved in health care evaluation with a comprehensive and unique source of information on PRO and HRQOL measures available through the Internet. PROQOLID currently describes more than 470 PRO instruments in a structured format. It is available in two levels, non-subscribers and subscribers, at http://www.proqolid.org. The first level is free of charge and contains 14 categories of basic useful information on the instruments (e.g. author, objective, original language, list of existing translations, etc.. The second level provides significantly more information about the instruments. It includes review copies of over 350 original instruments, 120 user manuals and 350 translations. Most are available in PDF format. This level is only accessible to annual subscribers. PROQOLID is updated in close collaboration with the instruments' authors on a regular basis. Fifty or more new instruments are added to the database annually. Today, all of the major pharmaceutical companies, prestigious institutions (such as the FDA, the NIH's National Cancer Institute, the U.S. Veterans Administration, dozens of universities, public institutions and researchers subscribe to PROQOLID on a yearly basis. More than 800 users per day routinely visit the database.
Kampen, D.A. van
In this thesis we investigated the use of clinical information and Patient Reported Outcome Measures (PROMs) for patient evaluation in orthopaedic surgery and sports medicine. In the first part, we showed that the Dutch version of the Simple Shoulder Test (SST) is a valid and reliable
Kampen, D.A. van
In this thesis we investigated the use of clinical information and Patient Reported Outcome Measures (PROMs) for patient evaluation in orthopaedic surgery and sports medicine. In the first part, we showed that the Dutch version of the Simple Shoulder Test (SST) is a valid and reliable instrumen
Brouwers, P.J.; Werkhoven, E. van; Bartelink, H.; Fourquet, A.; Lemanski, C.; Loon, J. van; Maduro, J.H.; Russell, N.S.; Scheijmans, L.J.; Schinagl, D.A.X.; Westenberg, A.H.; Poortmans, P.; Boersma, L.J.
PURPOSE: To investigate which factors are related to patient reported cosmetic outcome (PRCO) after breast conserving therapy. METHODS: From 2004 to 2011, 2421 cT1-2N0-2a breast cancer patients were randomised in the Young Boost Trial between a 16 and a 26Gy boost to the tumour bed. Cosmesis was sco
Brouwers, Patricia J. A. M.; van Werkhoven, Erik; Bartelink, Harry; Fourquet, Alain; Lemanski, Claire; van Loon, Judith; Maduro, John H.; Russell, Nicola S.; Scheijmans, Luc J. E. E.; Schinagl, Dominic A. X.; Westenberg, Antonia H.; Poortmans, Philip; Boersma, Liesbeth J.
Purpose: To investigate which factors are related to patient reported cosmetic outcome (PRCO) after breast conserving therapy. Methods: From 2004 to 2011, 2421 cT1-2N0-2a breast cancer patients were randomised in the Young Boost Trial between a 16 and a 26 Gy boost to the tumour bed. Cosmesis was sc
Alonso, J.; Bartlett, S.J.; Rose, M.; Aaronson, N.K.; Chaplin, J.; Efficace, F.; Leplège, A.; Aiping, L.U.; Tulsky, D.S.; Raat, H.; Ravens-Sieberer, U.; Revicki, D.; Terwee, C.B.; Valderas, J.M.; Cella, D.; Forrest, C.B.
Patient-reported outcomes (PROs) play an increasingly important role in clinical practice and research. Modern psychometric methods such as item response theory (IRT) enable the creation of item banks that support fixed-length forms as well as computerized adaptive testing (CAT), often resulting in
J. Alonso (Jordi); S.J. Bartlett (Susan); M. Rose (Matthias); N.K. Aaronson (Neil); J.E. Chaplin (John); F. Efficace (Fabio); A. Leplège (Alain); A. LU (Aiping); D.S. Tulsky (David); H. Raat (Hein); U. Ravens-Sieberer (Ulrike); D. Revicki (Dennis); C.B. Terwee (Caroline); J.M. Valderas (Jose); D. Cella (David); C.B. Forrest (Christopher)
textabstractPatient-reported outcomes (PROs) play an increasingly important role in clinical practice and research. Modern psychometric methods such as item response theory (IRT) enable the creation of item banks that support fixed-length forms as well as computerized adaptive testing (CAT), often r
Brouwers, Patricia J. A. M.; van Werkhoven, Erik; Bartelink, Harry; Fourquet, Alain; Lemanski, Claire; van Loon, Judith; Maduro, John H.; Russell, Nicola S.; Scheijmans, Luc J. E. E.; Schinagl, Dominic A. X.; Westenberg, Antonia H.; Poortmans, Philip; Boersma, Liesbeth J.
Purpose: To investigate which factors are related to patient reported cosmetic outcome (PRCO) after breast conserving therapy. Methods: From 2004 to 2011, 2421 cT1-2N0-2a breast cancer patients were randomised in the Young Boost Trial between a 16 and a 26 Gy boost to the tumour bed. Cosmesis was sc
van Cranenburgh, O.D.
The overall aim of this thesis was to examine and integrate patient reported outcomes (PROs) in dermatological care. In part I, we specifically examined health-related quality of life (HRQoL), treatment satisfaction, and experiences with care in patients with chronic skin diseases. Our results
Full Text Available Abstract Background Patient-reported outcomes are increasingly seen as complementary to biomedical measures. However, their prognostic importance has yet to be established, particularly in female long-term myocardial infarction (MI survivors. We aimed to determine whether 10-year survival in older women after MI relates to patient-reported outcomes, and to compare their survival with that of the general female population. Methods We included all women aged 60-80 years suffering MI during 1992-1997, and treated at one university hospital in Norway. In 1998, 145 (60% of those alive completed a questionnaire package including socio-demographics, the Sense of Coherence Scale (SOC-29, the World Health Organization Quality of Life Instrument Abbreviated (WHOQOL-BREF and an item on positive effects of illness. Clinical information was based on self-reports and hospital medical records data. We obtained complete data on vital status. Results The all-cause mortality rate during the 1998-2008 follow-up of all patients was 41%. In adjusted analysis, the conventional predictors s-creatinine (HR 1.26 per 10% increase and left ventricular ejection fraction below 30% (HR 27.38, as well as patient-reported outcomes like living alone (HR 6.24, dissatisfaction with self-rated health (HR 6.26, impaired psychological quality of life (HR 0.60 per 10 points difference, and experience of positive effects of illness (HR 6.30, predicted all-cause death. Major adverse cardiac and cerebral events were also significantly associated with both conventional predictors and patient-reported outcomes. Sense of coherence did not predict adverse events. Finally, 10-year survival was not significantly different from that of the general female population. Conclusion Patient-reported outcomes have long-term prognostic importance, and should be taken into account when planning aftercare of low-risk older female MI patients.
Phillips, John S; Yung, Matthew W
The purpose of this review was to systematically appraise the world literature to identify existing patient-reported outcome measures (PROMs) for the assessment of outcomes in patients with chronic suppurative otitis media, to verify the diversity of the individual questionnaire items, to report the methods employed to evaluate the questionnaires, and to identify areas for development in the future. Embase (January 1980-November 2014), MEDLINE (January 1946-November 2014), Cumulative Index to Nursing and Allied Health Literature (January 1981-November 2014), and PsycINFO (January 1806-November 2014). A systematic literature search was independently undertaken by the two authors according to predefined inclusion and exclusion criteria. Nine original articles were identified, which overall outlined the evaluation of four different questionnaires. This systematic appraisal of the world literature has identified four PROM questionnaires for use in patients with chronic suppurative otitis media. All four questionnaires evaluate reliability and validity using different psychometric methods. The Chronic Ear Survey questionnaire has been most broadly evaluated and disseminated. All four questionnaires assess static health status. There are many advantages to developing a dynamic one-hit questionnaire to assess the health status of patients having undergone an intervention for chronic suppurative otitis media. NA Laryngoscope, 126:1458-1463, 2016. © 2015 The American Laryngological, Rhinological and Otological Society, Inc.
Granja, C; Amaro, A; Dias, C; Costa-Pereira, A
Problems survivors face after intensive care unit (ICU) discharge begin while they are still in the ward, where many of their specific problems may run unrecognized, but they assume a heavy weight when they arrive at their homes and face several kind of limitations, from being unable to climb stairs because of weight loss, asthenia, dyspnea or joint stiffness to anxiety, depression or post-traumatic stress disorder. Follow-up consultations have given us a better understanding of these specific problems, and the information gained has been used to improve intensive care itself and promote a quality service for patients and relatives. The aim of this article is to provide an overview on adult ICU outcome studies and discuss how they have influenced and improved the delivery of intensive care. We will explain how we went from real patients to outcome studies and what we have learned concerning the consequences of critical illness and critical care. Development of outcome studies, what we have learned through them and our own experience will be outlined focusing mainly in four topics: mortality, physical disability, neuropsychological disability and health-related quality of life. Interventions to improve outcome on these main topics will be presented, and we will explain how we went from outcome studies to clinical interventions, focusing on the most recent proposals of intervention to improve outcome. © 2012 The Authors. Acta Anaesthesiologica Scandinavica © 2012 The Acta Anaesthesiologica Scandinavica Foundation.
马明; 周卫; 张世民; 李星; 章永东; 黎作旭; 吴冠男; 张兆杰
目的:初步研制腰腿痛患者报告结局量表.方法:通过对前期量表修订、典型病例访谈、核心小组讨论、专家咨询、借鉴国内外相关量表等方法,形成初步量表.于2010年6月-2011年2月间,纳入住院患者100例:男性44例,女性56例;年龄22～65岁,平均51.8岁;腰椎间盘突出症66例,腰椎管狭窄症34例.应用该量表对患者进行测评,并对其进行认知性、信度和效度分析.结果:形成一个由3个域16个条目组成的腰腿痛患者报告结局量表.32例病情无变化患者两次评分比较差异无统计学意义(P＞0.05),且有显著相关性(r＞0.9);该量表总体克朗巴赫a系数为0.936;每个条目得分与其所在域得分的相关系数均大于0.5;该量表同Oswestry功能障碍指数问卷表对患者的评分结果具有显著相关性(r＞0.9).结论:该量表有较好的认知性、信度和效度,可尝试作为腰腿痛患者临床疗效的评价工具,但仍需进一步的评价和修订.%Objective:To develop a patient reported outcome instrument for low back and leg pain preliminarily. Methods: The primary instrument was formed by revising early scale, interviewing typical cases, focus group discussion, expert consulting, referring to related instrument,and etc. 100 inpatients(44 males and 56 females)were analyzed from June 2010 to February 2011. They aged from 22 to 65 years old(mean, 51. 8 years), including 66 lumbar disc herniation and 34 lumber spinal stenosis. They were tested by the instrument, and then analyzed the cognition, reliability and validity of the scale. Results: A 3-domain,16-item instrument was developed. For 32 patients with no changes in illness, there was no statistical significant difference between two scores(P>0. 05)and it showed significant correlation r>0. 9). The total Cronbach's Alpha of the instrument was 0. 936. The correlation coefficients of the score of each item and its corresponding domain were more than 0. 5. Measurement
Canestaro, W J; Edwards, T C; Patrick, D L
New therapeutics are moving into phase 3 clinical trials for the treatment of coeliac disease, a condition with no established therapies other than gluten-free diet. These trials will require a meaningful, validated and fit for purpose patient-reported outcome measure (PROM) to quantify the symptomatic improvement of patients. To evaluate existing PROMs for suitability in a Food and Drug Administration (FDA) approval trial for a coeliac disease therapeutic. We performed a systematic search in five online databases (MedLine, EmBase, Web of Science, CENTRAL, CINAHL) for studies that enrolled patients with coeliac disease and used PROMs. Studies included in this review had to measure some PROM concept, be patient administered and based upon a previously validated instrument with published measurement properties. Our literature search identified 2706 unique records of which 199 ultimately qualified for abstraction. The majority of PROMs used in studies of coeliac disease was generic and did not measure numerous symptoms or concerns of interest to patients. Four PROMs were found to contain appropriate content for use in an FDA trial: the coeliac disease-specific modification of the Gastrointestinal Symptoms Rating Scale (CeD-GSRS), Psychological General Well-Being Index (PGWB), the Celiac Disease Symptom Diary (CDSD) and the Celiac Disease Patient Reported Outcome (CeD-PRO). The GSRS and PGWB are most often used together and are two of the most extensively used measures in coeliac disease. The CDSD and CeD-PRO were developed exclusively for trials in coeliac disease but have much less published information on their measurement properties. While we did not find PROMs that currently meet the stated expectations of the FDA for regulatory purposes, four PROMs (CeD-GSRS, PGWB, CDSD and CeD-PRO) appear to contain appropriate content and with modest additional validation work could meet scientific standards for valid and sensitive measures of disease and treatment outcome
moderate disability, while pulmonary fibrosis and pulmonary arterial hypertension patients displayed severe disability. Patients with chronic obstructive pulmonary disease had the worst pain level, whereas patients with pulmonary fibrosis had the worst emotion and cognition levels. A random-effect model confirmed that development of broncholitis obliterans syndrome was the most important determinant of health status (P = 0.03 compared to other variables, such as cytomegalovirus infections and underlying diagnoses.Conclusion: Descriptions of patients’ HRQL among different diagnosis groups could be used by clinicians to assist individualized patient care.Keywords: patient-reported outcomes, health-related quality of life measures, underlying diagnoses in lung transplant recipients, health utilities index
Green, Andrew; Liles, Clive; Rushton, Alison; Kyte, Derek G
This systematic review investigated the measurement properties of disease-specific patient-reported outcome measures used in Patellofemoral Pain Syndrome. Two independent reviewers conducted a systematic search of key databases (MEDLINE, EMBASE, AMED, CINHAL+ and the Cochrane Library from inception to August 2013) to identify relevant studies. A third reviewer mediated in the event of disagreement. Methodological quality was evaluated using the validated COSMIN (Consensus-based Standards for the Selection of Health Measurement Instruments) tool. Data synthesis across studies determined the level of evidence for each patient-reported outcome measure. The search strategy returned 2177 citations. Following the eligibility review phase, seven studies, evaluating twelve different patient-reported outcome measures, met inclusion criteria. A 'moderate' level of evidence supported the structural validity of several measures: the Flandry Questionnaire, Anterior Knee Pain Scale, Functional Index Questionnaire, Eng and Pierrynowski Questionnaire and Visual Analogue Scales for 'usual' and 'worst' pain. In addition, there was a 'Limited' level of evidence supporting the test-retest reliability and validity (cross-cultural, hypothesis testing) of the Persian version of the Anterior Knee Pain Scale. Other measurement properties were evaluated with poor methodological quality, and many properties were not evaluated in any of the included papers. Current disease-specific outcome measures for Patellofemoral Pain Syndrome require further investigation. Future studies should evaluate all important measurement properties, utilising an appropriate framework such as COSMIN to guide study design, to facilitate optimal methodological quality.
Fuller, Garth; Bolus, Roger; Whitman, Cynthia; Talley, Jennifer; Erder, M Haim; Joseph, Alain; Silberg, Debra G; Spiegel, Brennan
Most patients with gastroesophageal reflux disease (GERD) experience relief following treatment with proton pump inhibitors (PPIs) (Vakil et al. in Am J Gastroenterol 101:1900-1920, 2006; Everhart and Ruhl in Gastroenterology 136:376-386, 2009). As many as 17-44% of patients, however, exhibit only partial response to therapy. Most extant GERD patient-reported outcome (PRO) instruments fail to meet development best practices as described by the FDA (Talley and Wiklund in Qual Life Res 14:21-33, 2005; Van Pinxteren et al. in Cochrane Database Syst Rev 18:CD002095, 2004; El-Serag et al. in Aliment Pharmacol Ther 32:720-737, 2010). To develop and validate a PRO instrument for clinical trials involving patients with GERD who are PPI partial responders. We prepared a systematic literature review, held patient focus groups, convened an expert panel, and conducted cognitive interviews to establish content validity. Eligible participants took PPI therapy for at least 8 weeks, had undergone an upper endoscopy, and scored at least 8 points on the GerdQ . Qualitative data guided development of 26 draft items. Items were reviewed by expert panels and debriefed with patients. The resulting 21-item instrument underwent psychometric evaluation during a Phase IIB trial. During the trial, confirmatory factor analysis (n = 220) resulted in a four-factor model displaying the highest goodness of fit. All domains had a high inter-item correlation (Cronbach's α > 0.8). Test-retest reliability and convergent validity were strong, with highly significant (p PRISM scores and severity anchors and significant (p PRISM. Developed in line with FDA guidance on PROs, PRISM represents an important new outcome measure for patients with GERD with a partial response to PPI therapy.
Park, Kt; Harris, Merissa; Khavari, Nasim; Khosla, Chaitan
Patients with celiac disease (CD) are increasingly interconnected through social media, exchanging patient experiences and health-tracking information between individuals through various web-based platforms. Social media represents potentially unique communication interface between gastroenterologists and active social media users - especially young adults and adolescents with celiac disease-regarding adherence to the strict gluten-free diet, gastrointestinal symptoms, and meaningful discussion about disease management. Yet, various social media platforms may be underutilized for research purposes to collect patient-reported outcomes data. In this commentary, we summarize the scientific rationale and potential for future growth of social media in patient-reported outcomes research, focusing on college freshmen with celiac disease as a case study and provide overview of the methodological approach. Finally, we discuss how social media may impact patient care in the future through increasing mobile technology use.
Baker, Joshua F; Conaghan, Philip G; Emery, Paul;
PURPOSE: We assessed whether MRI measures of synovitis, osteitis and bone erosion were associated with patient-reported outcomes (PROs) in a longitudinal clinical trial setting among patients with rheumatoid arthritis (RA). METHODS: This longitudinal cohort of 291 patients with RA was derived from...... across treatment groups. CONCLUSIONS: MRI measures of inflammation and structural damage correlate independently with physical function, pain and patient global assessments. These observations support the validity of MRI biomarkers. TRIAL REGISTRATION NUMBER: NCT00264537; Post-results....
Kyte, DG; Draper, H; Ives, J.; Liles, C; Gheorghe, A.; Calvert, M
BACKGROUND: Patient reported outcomes (PROs) are increasingly assessed in clinical trials, and guidelines are available to inform the design and reporting of such trials. However, researchers involved in PRO data collection report that specific guidance on 'in-trial' activity (recruitment, data collection and data inputting) and the management of 'concerning' PRO data (i.e., data which raises concern for the well-being of the trial participant) appears to be lacking. The purpose of this revie...
Full Text Available Abstract Background A critical component that influences the measurement properties of a patient-reported outcome (PRO instrument is the rating scale. Yet, there is a lack of general consensus regarding optimal rating scale format, including aspects of question structure, the number and the labels of response categories. This study aims to explore the characteristics of rating scales that function well and those that do not, and thereby develop guidelines for formulating rating scales. Methods Seventeen existing PROs designed to measure vision-related quality of life dimensions were mailed for self-administration, in sets of 10, to patients who were on a waiting list for cataract extraction. These PROs included questions with ratings of difficulty, frequency, severity, and global ratings. Using Rasch analysis, performance of rating scales were assessed by examining hierarchical ordering (indicating categories are distinct from each other and follow a logical transition from lower to higher value, evenness (indicating relative utilization of categories, and range (indicating coverage of the attribute by the rating scale. Results The rating scales with complicated question format, a large number of response categories, or unlabelled categories, tended to be dysfunctional. Rating scales with five or fewer response categories tended to be functional. Most of the rating scales measuring difficulty performed well. The rating scales measuring frequency and severity demonstrated hierarchical ordering but the categories lacked even utilization. Conclusion Developers of PRO instruments should use a simple question format, fewer (four to five and labelled response categories.
Schick-Makaroff, Kara; Molzahn, Anita
Mobile devices are increasingly being used for data collection in research. However, many researchers do not have experience in collecting data electronically. Hence, the purpose of this short report was to identify issues that emerged in a study that incorporated electronic capture of patient-reported outcomes in clinical settings, and strategies used to address the issues. The issues pertaining to electronic patient-reported outcome data collection were captured qualitatively during a study on use of electronic patient-reported outcomes in two home dialysis units. Fifty-six patients completed three surveys on tablet computers, including the Kidney Disease Quality of Life-36, the Edmonton Symptom Assessment Scale, and a satisfaction measure. Issues that arose throughout the research process were recorded during ethics reviews, implementation process, and data collection. Four core issues emerged including logistics of technology, security, institutional and financial support, and electronic design. Although use of mobile devices for data collection has many benefits, it also poses new challenges for researchers. Advance consideration of possible issues that emerge in the process, and strategies that can help address these issues, may prevent disruption and enhance validity of findings.
Schuster, Jessica; Saraiya, Siddharth; Tennyson, Nathan; Nedelka, Michele; Mukhopadhyay, Nitai; Weiss, Elisabeth
Palmar and plantar fibromatosis (PPF) is a progressive connective tissue disorder of the hand/foot that often leads to debilitating functional impairment. In Europe, orthovoltage radiation therapy (RT) has been demonstrated to prevent local disease progression for up to 80% of patients with early-stage PPF. There are limited data reporting outcomes for populations outside of Europe or using electron RT. Between 2008 and 2013, 44 early-stage PPF cases received RT. RT fields involved clinically defined targets encompassing involved areas (skin changes, cords, nodules) with at least 1.5-cm margins. En face electrons (6-12 MeV) and bolus (0.5-1 cm) were selected individually. Outcomes are reported for patients who participated in an institutional review board-approved standardized questionnaire and chart review. Thirty-three patients received 66 treatments (45 hands/15 feet and 6 reirradiations). Most frequent dose schemes were 21 Gy (3 Gy in 7 fractions) and 30 Gy (3 Gy in 10 fractions with 6- to 8-week breaks after 15 Gy). Median time to follow-up survey was 31 months. Disease progression at any location within or outside the RT treatment field occurred in 20 of 33 patients (61%). Fourteen of 60 sites (23%) developed in-field progression, but 4 sites were successfully reirradiated with final local control in 50 of 60 sites (83%). RT improved pretreatment symptoms of pain with strain at 30 of 37 sites (81%) and itch/burn sensations at 17 of 21 sites (81%). There were no reported grade ≥2 late toxicities even with reirradiation. Patient reported overall success with treatment was 31 of 33 patients (94%). PPF is a progressive disease. En face electron RT is an effective therapy that stabilizes or improves symptoms in the majority of patients. Reirradiation can be considered as a treatment option for in-field progression. Patients report minimal toxicity and a high rate of satisfaction with treatment. Copyright © 2015 American Society for Radiation Oncology. Published
MacDermid, Joy C
Patient-reported outcome measures (PRO) can provide reliable and valid estimates of patient status and response to interventions to complete the final step in an evidence-based patient interaction. A variety of PRO are relevant to upper extremity surgery and rehabilitation outcomes. PRO provide feasible tools for clinical research or practice, although use in clinical decision making lags behind research applications. Recent trends in clinical measurement include better integration of International Classification of Functioning, Disability and Health in content validation, more modern methods of evaluating scaling properties (Rasch analysis), consensus exercise on establishing core measures, electronic data collection, and computer-adaptive testing.
Straatman, Jennifer; van der Wielen, Nicole; Joosten, Pieter J; Terwee, Caroline B; Cuesta, Miguel A; Jansma, Elise P; van der Peet, Donald L
Gastric cancer is responsible for 10 % of all cancer-related deaths worldwide. With improved operative techniques and neo-adjuvant therapy, survival rates are increasing. Outcomes of interest are shifting to quality of life (QOL), with many different tools available. The aim of this study was to assess which patient-reported outcome measures (PROMs) are used to measure QOL after a gastrectomy for cancer. A comprehensive search was conducted for original articles investigating QOL after gastrectomy. Two authors independently selected relevant articles, conducted clinical appraisal and extracted data (P.J. and J.S.). Out of 3414 articles, 26 studies were included, including a total of 4690 patients. These studies included ten different PROMs, which could be divided into generic, symptom-specific and disease-specific questionnaires. The EORTC and the FACT questionnaires use an oncological overall QOL module and an organ-specific module. Only one validation study regarding the use of the EORTC after surgery for gastric cancer was available, demonstrating good psychometric properties and clinical validity. A great variety of PROMs are being used in the measurement of QOL after surgery for gastric cancer. A questionnaire with a general module along with a disease-specific module for the assessment of QOL seems most desirable, such as the EORTC and the FACT with their specific modules. Both are developed in different treatment modalities, such as in surgical patients. EORTC is the most widely used questionnaire and therefore allows for comparison of new studies to existing data. Future studies are needed to assess content validity in surgical gastric cancer patients.
Bekelis, Kimon; Calnan, Daniel; Simmons, Nathan; MacKenzie, Todd A; Kakoulides, George
To investigate the effect of exposure to a virtual reality (VR) environment preoperatively on patient-reported outcomes for surgical operations. There is a scarcity of well-developed quality improvement initiatives targeting patient satisfaction. We performed a randomized controlled trial of patients undergoing cranial and spinal operations in a tertiary referral center. Patients underwent a 1:1 randomization to an immersive preoperative VR experience or standard preoperative experience stratified on type of operation. The primary outcome measures were the Evaluation du Vecu de l'Anesthesie Generale (EVAN-G) score and the Amsterdam Preoperative Anxiety and Information (APAIS) score, as markers of the patient's experience during the surgical encounter. During the study period, a total of 127 patients (mean age 55.3 years, 41.9% females) underwent randomization. The average EVAN-G score was 84.3 (standard deviation, SD, 6.4) after VR, and 64.3 (SD, 11.7) after standard preoperative experience (difference, 20.0; 95% confidence interval, CI, 16.6-23.3). Exposure to an immersive VR experience also led to higher APAIS score (difference, 29.9; 95% CI, 24.5-35.2). In addition, VR led to lower preoperative VAS stress score (difference, -41.7; 95% CI, -33.1 to -50.2), and higher preoperative VAS preparedness (difference, 32.4; 95% CI, 24.9-39.8), and VAS satisfaction (difference, 33.2; 95% CI, 25.4-41.0) scores. No association was identified with VAS stress score (difference, -1.6; 95% CI, -13.4 to 10.2). In a randomized controlled trial, we demonstrated that patients exposed to preoperative VR had increased satisfaction during the surgical encounter. Harnessing the power of this technology, hospitals can create an immersive environment that minimizes stress, and enhances the perioperative experience.
Engler, Kim; Lessard, David; Lebouché, Bertrand
The use of patient-reported outcome (PRO) measures to provide added feedback to health providers is receiving interest as a means of improving clinical care and patient outcomes, and contributing to more patient-centered care. In human immunodeficiency virus (HIV), while PROs are used in research, their application in clinical practice has been limited despite their potential utility. PRO selection is an important consideration when contemplating their use. As past reviews of PROs in HIV have focused on particular areas (e.g. disability, satisfaction with care), a more comprehensive review could better inform on the available instruments and their scope. This article reviews HIV-specific PROs to produce an inventory and to identify the central concepts targeted over time. Seven databases were searched (HAPI, MEDLINE, PsychINFO, PubMed, EMBASE, CINAHL, Google Scholar), generating 14,794 records for evaluation. From these records, 117 HIV-specific PROs were identified and categorized based on a content analysis of their targeted concept: Health-Related Quality of Life (23; 20 %), ART and Adherence-Related Views and Experiences (19; 16 %), Healthcare-Related Views and Experiences (15; 13 %), Psychological Challenges (12; 10 %), Symptoms (12; 10 %), Psychological Resources (10; 9 %), HIV Self-Management and Self-Care (8; 7 %), HIV-Related Stigma (8; 7 %), Body and Facial Appearance (4; 3 %), Social Support (3; 3 %), Sexual and Reproductive Health (2; 2 %), and Disability (1; 1 %). This review highlights the variety and evolution of HIV-specific PROs, with the arrival of seven categories of PROs only after the advent of highly-active antiretroviral therapy. Our inventory also offers a useful resource. However, the interest of further HIV-specific PRO development should be explored in sexual health, which received little independent attention.
Hsu, Tina; Speers, Caroline H; Kennecke, Hagen F; Cheung, Winson Y
Patient-reported outcomes (PROs) are increasingly used in clinical settings. Prior research suggests that PROs collected at baseline may be associated with cancer survival, but most of those studies were conducted in patients with breast or lung cancer. The objective of this study was to determine the correlation between prospectively collected PROs and cancer-specific outcomes in patients with early stage colorectal cancer. Patients who had newly diagnosed stage II or III colorectal cancer from 2009 to 2010 and had a consultation at the British Columbia Cancer Agency completed the brief Psychosocial Screen for Cancer (PSSCAN) questionnaire, which collects data on patients' perceived social supports, quality of life (QOL), anxiety and depression, and general health. PROs from the PSSCAN were linked with the Gastrointestinal Cancers Outcomes Database, which contains information on patient and tumor characteristics, treatment details, and cancer outcomes. Cox regression models were constructed for overall survival (OS), and Fine and Gray regression models were developed for disease-specific survival (DSS). In total, 692 patients were included. The median patient age was 67 years (range, 26-95 years), and the majority had colon cancer (61%), were diagnosed with stage III disease (54%), and received chemotherapy (58%). In general, patients felt well supported and reported good overall health and QOL. On multivariate analysis, increased fatigue was associated with worse OS (hazard ratio [HR], 1.99; P = .00007) and DSS (HR, 1.63; P = .03), as was lack of emotional support (OS: HR, 4.36; P = .0003; DSS: HR, 1.92; P = .02). Although most patients described good overall health and QOL and indicated that they were generally well supported, patients who experienced more pronounced fatigue or lacked emotional support had a higher likelihood of worse OS and DSS. These findings suggest that abbreviated PROs can inform and assist clinicians to identify patients who have a worse
Background Hip arthroscopies are often used in the treatment of intra-articular hip injuries. Patient-reported outcomes (PRO) are an important parameter in evaluating treatment. It is unclear which PRO questionnaires are specifically available for hip arthroscopy patients. The aim of this systematic review was to investigate which PRO questionnaires are valid and reliable in the evaluation of patients undergoing hip arthroscopy. Methods A search was conducted in Pubmed, Medline, CINAHL, the Cochrane Library, Pedro, EMBASE and Web of Science from 1931 to October 2010. Studies assessing the quality of PRO questionnaires in the evaluation of patients undergoing hip arthroscopy were included. The quality of the questionnaires was evaluated by the psychometric properties of the outcome measures. The quality of the articles investigating the questionnaires was assessed by the COSMIN list. Results Five articles identified three questionnaires; the Modified Harris Hip Score (MHHS), the Nonarthritic Hip Score (NAHS) and the Hip Outcome Score (HOS). The NAHS scored best on the content validity, whereas the HOS scored best on agreement, internal consistency, reliability and responsiveness. The quality of the articles describing the HOS scored highest. The NAHS is the best quality questionnaire. The articles describing the HOS are the best quality articles. Conclusions This systematic review shows that there is no conclusive evidence for the use of a single patient-reported outcome questionnaire in the evaluation of patients undergoing hip arthroscopy. Based on available psychometric evidence we recommend using a combination of the NAHS and the HOS for patients undergoing hip arthroscopy. PMID:21619610
Kitchen, H; Cordingley, L; Young, H; Griffiths, C E M; Bundy, C
As a long-term condition, psoriasis demands significant personal and professional input for optimal self-management. Low levels of well-being and high levels of psychological distress in patients with psoriasis are associated with reduced resources for self-care. Patient-reported outcome (PRO) measures can be used to assess physical, social and psychological functioning in order to guide treatment. In this article, we systematically reviewed the development and validation of existing PRO measures. PubMed (Medline), PsycINFO and CINAHL were searched systematically using predefined search terms. The search was limited to articles in the English language relating to human subjects. Articles were selected for full review through explicit inclusion/exclusion criteria. PRO measures were critically reviewed in accordance with the published guidelines and theory on the development and validation of PROs. The search identified 967 abstracts; 71 of these articles met the criteria for full review. In these 71 articles, 45 PRO measures were found: 16 were specific to psoriasis, 21 assessed other dermatological conditions and eight were developed for generic nondermatological health conditions. The review revealed several limitations of the existing measures, including: (i) a composite structure assessing multiple, poorly-defined concepts; (ii) a lack of evidence for face and content validity; (iii) a failure to include both patient and clinician perspectives and requirements and (iv) a lack of evidence regarding the feasibility and acceptability for patients and physicians. No single PRO measure with adequate evidence of validity, reliability and sensitivity to change captures patient well-being in psoriasis. A valid, sensitive, specific and acceptable PRO that assesses the full impact of psoriasis on well-being is needed for the comprehensive clinical management of psoriasis.
Ali, Shehzad; Littlewood, Elizabeth; McMillan, Dean; Delgadillo, Jaime; Miranda, Alfonso; Croudace, Tim; Gilbody, Simon
Background Variability in patient-reported outcomes of psychological treatments has been partly attributed to therapists – a phenomenon commonly known as therapist effects. Meta-analytic reviews reveal wide variation in therapist-attributable variability in psychotherapy outcomes, with most studies reporting therapist effects in the region of 5% to 10% and some finding minimal to no therapist effects. However, all except one study to date have been conducted in high-intensity or mixed intervention groups; therefore, there is scarcity of evidence on therapist effects in brief low-intensity psychological interventions. Objective To examine therapist effects in low-intensity interventions for depression and anxiety in a naturalistic setting. Data and Analysis Session-by-session data on patient-reported outcome measures were available for a cohort of 1,376 primary care psychotherapy patients treated by 38 therapists. Outcome measures included PHQ-9 (sensitive to depression) and GAD-7 (sensitive to general anxiety disorder) measures. Three-level hierarchical linear modelling was employed to estimate therapist-attributable proportion of variance in clinical outcomes. Therapist effects were evaluated using the intra-cluster correlation coefficient (ICC) and Bayesian empirical predictions of therapist random effects. Three sensitivity analyses were conducted: 1) using both treatment completers and non-completers; 2) a sub-sample of cases with baseline scores above the conventional clinical thresholds for PHQ-9 and GAD-7; and 3) a two-level model (using patient-level pre- and post-treatment scores nested within therapists). Results The ICC estimates for all outcome measures were very small, ranging between 0% and 1.3%, although most were statistically significant. The Bayesian empirical predictions showed that therapist random effects were not statistically significantly different from each other. Between patient variability explained most of the variance in outcomes
Lyman, Stephen; Yin, Kaitlyn L
Total knee arthroplasty is a large contributor to Medicare costs. In an effort to lower costs and improve outcomes, the Centers for Medicaid and Medicare Services has implemented the Comprehensive Care for Joint Replacement model, which incentivizes surgeons to submit both general health and joint-specific patient-reported outcome measures (PROMs). However, in addition to using PROMs for reporting purposes, surgeons should also consider incorporating PROMs into clinical practice. Currently, PROMs are not widely implemented in the clinical setting despite their value in measuring factors such as patients' expectations and mental state, which impact outcomes. Furthermore, as technology improves, PROM collection will become faster and more efficient. The information collected by PROMs can inform treatment decisions and facilitate communication between the surgeon and the patient.
Kashikar-Zuck, Susmita; Carle, Adam; Barnett, Kimberly; Goldschneider, Kenneth R; Sherry, David D; Mara, Constance A; Cunningham, Natoshia; Farrell, Jennifer; Tress, Jenna; DeWitt, Esi Morgan
The Patient-Reported Outcomes Measurement Information System (PROMIS) initiative is a comprehensive strategy by the National Institutes of Health to support the development and validation of precise instruments to assess self-reported health domains across healthy and disease-specific populations. Much progress has been made in instrument development, but there remains a gap in the validation of PROMIS measures for pediatric chronic pain. The purpose of this study was to investigate the construct validity and responsiveness to change of 7 PROMIS domains for the assessment of children (ages: 8-18) with chronic pain--Pain Interference, Fatigue, Anxiety, Depression, Mobility, Upper Extremity Function, and Peer Relationships. The PROMIS measures were administered at the initial visit and 2 follow-up visits at an outpatient chronic pain clinic (CPC; N = 82) and at an intensive amplified musculoskeletal pain day-treatment program (N = 63). Aim 1 examined construct validity of PROMIS measures by comparing them with corresponding "legacy" measures administered as part of usual care in the CPC sample. Aim 2 examined sensitivity to change in both CPC and amplified musculoskeletal pain samples. Longitudinal growth models showed that PROMIS' Pain Interference, Anxiety, Depression, Mobility, Upper Extremity, and Peer Relationship measures and legacy instruments generally performed similarly with slightly steeper slopes of improvement in legacy measures. All 7 PROMIS domains showed responsiveness to change. Results offered initial support for the validity of PROMIS measures in pediatric chronic pain. Further validation with larger and more diverse pediatric pain samples and additional legacy measures would broaden the scope of use of PROMIS in clinical research.
Carroll, Thomas L; Lee, Stella E; Lindsay, Robin; Locandro, Drew; Randolph, Gregory W; Shin, Jennifer J
The assessment of patient-reported outcome measures (PROMs) in the outpatient setting is gaining momentum in clinical and research venues. Implementing this data capture into one's practice, however, is not a one-size-fits-all venture, and it is critical to determine when, how, and where to include these patient-centered assessments. This installment of the "Evidence-Based Medicine in Otolaryngology" series provides insight into the implementation process and experiences with successful incorporation of PROMs into clinical practice. Specifically, 4 differing clinical scenarios and collection techniques are described, including data acquisition protocols, formats for clinician data usage, and applications of PROM results in clinical and research scenarios.
Overgaard, Dorthe; Schrader, Anne-Marie; Lisby, Karen H
Objectives: Data on patient-reported outcomes (PROs) in patients with single-ventricle physiology (SVP) are scarce. We sought (1) to describe the perceived health status, quality of life, symptoms of anxiety and depression, and sense of coherence in adult survivors with SVP, (2) to compare PROs...... across functional classes, and (3) to compare PROs between patients and controls. Methods: A case-control study in two adult congenital heart programmes with 62 adult survivors with SVP were matched to 172 healthy controls. A wide range of PROs were measured using validated questionnaires. The treating...
Full Text Available Abstract Background A strong consensus exists for a systematic approach to linguistic validation of patient reported outcome measures (PROMs and discrete methods for assessing their psychometric properties. Despite the need for robust evidence of the appropriateness of measures, transition from linguistic to psychometric validation is poorly documented or evidenced. This paper demonstrates the importance of linking linguistic and psychometric testing through a purposeful stage which bridges the gap between translation and large-scale validation. Findings Evidence is drawn from a study to develop a Welsh language version of the Beck Depression Inventory-II (BDI-II and investigate its psychometric properties. The BDI-II was translated into Welsh then administered to Welsh-speaking university students (n = 115 and patients with depression (n = 37 concurrent with the English BDI-II, and alongside other established depression and quality of life measures. A Welsh version of the BDI-II was produced that, on administration, showed conceptual equivalence with the original measure; high internal consistency reliability (Cronbach’s alpha = 0.90; 0.96; item homogeneity; adequate correlation with the English BDI-II (r = 0.96; 0.94 and additional measures; and a two-factor structure with one overriding dimension. Nevertheless, in the student sample, the Welsh version showed a significantly lower overall mean than the English (p = 0.002; and significant differences in six mean item scores. This prompted a review and refinement of the translated measure. Conclusions Exploring potential sources of bias in translated measures represents a critical step in the translation-validation process, which until now has been largely underutilised. This paper offers important findings that inform advanced methods of cross-cultural validation of PROMs.
Frost, Marlene H; Reeve, Bryce B; Liepa, Astra M; Stauffer, Joseph W; Hays, Ron D
This article focuses on the necessary psychometric properties of a patient-reported outcomes (PROs) measure. Topics include the importance of reliability and validity, psychometric approaches used to provide reliability and validity estimates, the kinds of evidence needed to indicate that a PRO has a sufficient level of reliability and validity, contexts that may affect psychometric properties, methods available to evaluate PRO instruments when the context varies, and types of reliability and validity testing that are appropriate during different phases of clinical trials. Points discussed include the perspective that the psychometric properties of reliability and validity are on a continuum in which the more evidence one has, the greater confidence there is in the value of the PRO data. Construct validity is the type of validity most frequently used with PRO instruments as few "gold standards" exist to allow the use of criterion validity and content validity by itself only provides beginning evidence of validity. Several guidelines are recommended for establishing sufficient evidence of reliability and validity. For clinical trials, a minimum reliability threshold of 0.70 is recommended. Sample sizes for testing should include at least 200 cases and results should be replicated in at least one additional sample. At least one full report on the development of the instrument and one on the use of the instrument are deemed necessary to evaluate the PRO psychometric properties. Psychometric testing ideally occurs before the initiation of Phase III trials. When testing does not occur prior to a Phase III trial, considerable risk is posed in relation to the ability to substantiate the use of the PRO data. Various qualitative (e.g., focus groups, behavioral coding, cognitive interviews) and quantitative approaches (e.g., differential item functioning testing) are useful in evaluating the reliability and validity of PRO instruments.
Bouman, M.B.; Sluis, W.B. van der; Woudenberg Hamstra, L.E. van; Buncamper, M.E.; Kreukels, B.P.; Meijerink, W.J.H.J.; Mullender, M.G.
INTRODUCTION: Puberty-suppressing hormonal treatment may result in penoscrotal hypoplasia in transgender women, making standard penile inversion vaginoplasty not feasible. For these patients, intestinal vaginoplasty is a surgical alternative, but knowledge on patient-reported postoperative outcomes
Alonso, Jordi; Bartlett, Susan J; Rose, Matthias; Aaronson, Neil K; Chaplin, John E; Efficace, Fabio; Leplège, Alain; Lu, Aiping; Tulsky, David S; Raat, Hein; Ravens-Sieberer, Ulrike; Revicki, Dennis; Terwee, Caroline B; Valderas, Jose M; Cella, David; Forrest, Christopher B
Patient-reported outcomes (PROs) play an increasingly important role in clinical practice and research. Modern psychometric methods such as item response theory (IRT) enable the creation of item banks that support fixed-length forms as well as computerized adaptive testing (CAT), often resulting in improved measurement precision and responsiveness. Here we describe and discuss the case for developing an international core set of PROs building from the US PROMIS® network.PROMIS is a U.S.-based cooperative group of research sites and centers of excellence convened to develop and standardize PRO measures across studies and settings. If extended to a global collaboration, PROMIS has the potential to transform PRO measurement by creating a shared, unifying terminology and metric for reporting of common symptoms and functional life domains. Extending a common set of standardized PRO measures to the international community offers great potential for improving patient-centered research, clinical trials reporting, population monitoring, and health care worldwide. Benefits of such standardization include the possibility of: international syntheses (such as meta-analyses) of research findings; international population monitoring and policy development; health services administrators and planners access to relevant information on the populations they serve; better assessment and monitoring of patients by providers; and improved shared decision making.The goal of the current PROMIS International initiative is to ensure that item banks are translated and culturally adapted for use in adults and children in as many countries as possible. The process includes 3 key steps: translation/cultural adaptation, calibration, and validation. A universal translation, an approach focusing on commonalities, rather than differences across versions developed in regions or countries speaking the same language, is proposed to ensure conceptual equivalence for all items. International item
Kearns, Tara; Cornally, Nicola; Molloy, William
End-of-life (EoL) care(1) is increasingly used as a generic term in preference to palliative care or terminal care, particularly with reference to individuals with chronic disease, who are resident in community and long-term care (LTC) settings. This review evaluates studies based on patient reported outcome measures (PROMS) of quality of EoL care across all health-care settings. From 1041 citations, 12 studies were extracted by searches conducted in EBSCO, Scopus, Web of Science, PubMed, Cochrane, Open Grey and Google Scholar databases. At present, the evidence base for EoL care is founded on cancer care. This review highlights the paucity of studies that evaluate quality of EoL care for patients with chronic disease outside the established cancer-acute care paradigm, particularly in LTC. This review highlights the absence of any PROMs for the estimated 60% of patients in LTC with cognitive impairment. Patient-reported outcomes (PROs) are critical to understanding how EoL care services and practices affect patients' health and EoL experience. PROMs describe the quality of care from the patient's perspective and add balance to existing clinical or proxy-derived knowledge on the quality of care and services provided.
Ruszniewski, Philippe; Valle, Juan W; Lombard-Bohas, Catherine; Cuthbertson, Daniel J; Perros, Petros; Holubec, Luboš; Delle Fave, Gianfranco; Smith, Denis; Niccoli, Patricia; Maisonobe, Pascal; Atlan, Philippe; Caplin, Martyn E
Lanreotide Autogel/Depot effectively controls symptoms in patients with carcinoid syndrome associated with neuroendocrine tumours. Data on patient-reported outcomes are sparse. To evaluate the effect of lanreotide on patient-reported outcomes (PROs) with carcinoid syndrome. This was an international, open-label, observational study of adults with neuroendocrine tumours and history of diarrhoea, receiving lanreotide for >3 months for relief of carcinoid syndrome symptoms. The primary PRO measure was satisfaction with diarrhoea control. Secondary PRO measures included severity, change in symptoms and impact on daily life of diarrhoea; and patient satisfaction with flushing control. Of 273 patients enrolled, 76% were 'completely' or 'rather' satisfied with diarrhoea control; 79% reported improvement in diarrhoea with lanreotide. The proportion of patients with 'mild', 'minimal', or 'no diarrhoea' increased from 33% before treatment to 75% during treatment; 75% were unconcerned about the impact of diarrhoea on daily life. Satisfaction with flushing control amongst patients with significant flushing at treatment initiation was 73%. Lanreotide treatment was associated with improvements in symptoms as well as a range of PROs in patients with neuroendocrine tumours and carcinoid syndrome (ClinicalTrials.gov: NCT01234168). Copyright © 2016 The Authors. Published by Elsevier Ltd.. All rights reserved.
Background: Patient reported outcome-measures (PROs) are increasingly used in orthopedics. Information on number of patients needed in different settings is warranted. Aim: To assess the number of patients needed for different PROs to discriminate between subgroups of age, gender, and diagnosis...... with sample size calculations or by power calculations and simulated ANOVA F tests, depending on the number of groups. Results: To discriminate between gender, the least number needed to find a statistically significant difference in mean sum score in each group was 298 (OHS) while HOOS QoL required the most....... Methods: 5777 primary THA patients, operated 1‐2, 5‐6, and 10‐11 years ago. SF‐12 Health Survey (SF-12), EQ-5D, Oxford 12‐item Hip Score (OHS), and Hip dysfunction and Osteoarthritis Outcome Score (HOOS) were included. The different PRO subscales abilities to discriminate between groups were studied using...
Flosadottir, Vala; Roos, Ewa M; Ageberg, Eva
BACKGROUND/AIM: Consequences of an anterior cruciate ligament (ACL) injury include worse patient-reported outcomes (PROs) and a decrease in activity level. Muscle function can be improved by targeted exercise. Our aims were to investigate cross-sectional and longitudinal associations among lower...... extremity muscle function and PROs after ACL injury. METHODS: Fifty-four participants (15 women, mean 30 years) with ACL injury or reconstruction, from the Knee Anterior Cruciate Ligament, Nonsurgical versus Surgical Treatment (KANON) trial (ISRCTN84752559), were assessed with hop performance, muscle power...... and postural orientation 3 years (SD 0.85) after ACL injury. PROs at 3 and 5 years after injury included Knee Injury and Osteoarthritis Outcome Score (KOOS) subscales Function in sport and recreation (KOOS Sport/rec) and Knee-related Quality of life (KOOS QoL), KOOS item Q3 (KOOS Q3), Tegner Activity Scale...
Paulsen, Aksel; Pedersen, Alma B; Overgaard, Søren
Background and purpose Feasibility is an important parameter when choosing which patient-reported outcomes (PRO) to use in a study. We assessed the feasibility of PROs in a hip registry setting. Methods Primary total hip arthroplasty (THA) patients (n = 5,747) who had been operated on 1-2, 5......-6, or 10-11 years previously were randomly selected from the Danish Hip Arthroplasty Register and sent 2 PRO questionnaires: 1 generic (EuroQoL-5D or SF-12 health survey) and 1 disease-specific (hip dysfunction and osteoarthritis outcome score (HOOS) or Oxford 12-item hip score). We compared response rates......, floor and ceiling effects, missing items, and the need for manual validation of forms. Results 4,784 patients (mean age 71 years, 57% females) were included (83%). The response rates ranged from 82-84%. Statistically significantly different floor and ceiling effects ranged from 0% to 0.5% and from 6...
Full Text Available Lauren M Nelson,1 Valerie SL Williams,1 Sheri E Fehnel,1 Robyn T Carson,2 James MacDougall,3 Mollie J Baird,3 Stavros Tourkodimitris,2 Caroline B Kurtz,3 Jeffrey M Johnston31RTI Health Solutions, Durham, NC, USA; 2Forest Research Institute, Jersey City, NJ, USA; 3Ironwood Pharmaceuticals, Cambridge, MA, USABackground: Measures assessing treatment outcomes in previous CC clinical trials have not met the requirements described in the US Food and Drug Administration's guidance on patient-reported outcomes.Aim: Psychometric analyses using data from one Phase IIb study and two Phase III trials of linaclotide for the treatment of chronic constipation (CC were conducted to document the measurement properties of patient-reported CC Symptom Severity Measures.Study methods: Each study had a multicenter, randomized, double-blind, placebo-controlled, parallel-group design, comparing placebo to four doses of oral linaclotide taken once daily for 4 weeks in the Phase IIb dose-ranging study (n=307 and to two doses of linaclotide taken once daily for 12 weeks in the Phase III trials (n=1,272. The CC Symptom Severity Measures addressing bowel function (Bowel Movement Frequency, Stool Consistency, Straining and abdominal symptoms (Bloating, Abdominal Discomfort, Abdominal Pain were administered daily using interactive voice-response system technology. Intraclass correlations, Pearson correlations, factor analyses, F-tests, and effect sizes were computed.Results: The CC Symptom Severity Measures demonstrated satisfactory test–retest reliability and construct validity. Factor analyses indicated one factor for abdominal symptoms and another for bowel symptoms. Known-groups F-tests substantiated the discriminating ability of the CC Symptom Severity Measures. Responsiveness statistics were moderate to strong, indicating that these measures are capable of detecting change.Conclusion: In large studies of CC patients, linaclotide significantly improved abdominal and
L.A. 't Hoen (Lisette)
markdownabstractAbstract Pelvic floor disorders are characterized by four domains: urinary symptoms, anorectal symptoms, pelvic organ prolapse and sexual dysfunction. The symptoms of the different domains have a significant impact on patient’s quality of life. Traditional outcome measures, such a
Nüssler, Emil Karl; Greisen, Susanne; Kesmodel, Ulrik Schiøler
Abstract INTRODUCTION AND HYPOTHESIS: The aim of this study was to compare patient reported outcomes and complications after repair of recurrent anterior vaginal wall prolapse in routine health care settings using standard anterior colporrhaphy or non-absorbable mesh. METHODS: The study is based...... on prospective data from the Swedish National Register for Gynaecological Surgery. 286 women were operated on for recurrent anterior vaginal wall prolapse in 2008-2010; 157 women had an anterior colporrhaphy and 129 were operated on with a non-absorbable mesh. Pre-, and perioperative data were collected from...... were found more often in the mesh group. However, no differences in serious complications were found. Thus, an organ lesion was found in 2.3 % after mesh implant compared with 2.5 % after anterior colporrhaphy (p = 0.58). Two patients in the mesh group (1.2 %) were re-operated compared with 1 patient...
Moura, Lidia M V R; Schwamm, Eli; Moura Junior, Valdery; Seitz, Michael P; Hsu, John; Cole, Andrew J; Schwamm, Lee H
To determine whether patients could self-report physical and mental health assessments in the waiting room and whether these assessments would be associated with modified Rankin Scale (mRS) and Quality of Life in Epilepsy (QOLIE-10) scores. We offered iPad-based surveys to consecutive adult neurology patients at check-in to collect patient-reported outcome measures (PROMs). We collected demographic and clinical data on 6,075 patients through survey or administrative claims and PROMs from participating patients. We compared demographic characteristics of participants and nonparticipants and tested associations between physical and mental health scores and mRS and QOLIE-10. Of 6,075 patients seen by neurologists during the study period, 2,992 (49.3%) participated in the survey. Compared to nonparticipating patients, participating patients more often were privately insured (53.5% vs 42.7%, p neurology (nonsubspecialty) clinics (53.1% vs 46.6%, p Neurology.
Vissers, Pauline A J; Falzon, Louise; van de Poll-Franse, Lonneke V
PURPOSE: This systematic review aims to summarize the current literature regarding potential effects of having both cancer and diabetes on patient-reported outcomes (PROs) and to provide directions for future research. METHODS: MEDLINE, The Cochrane Library, CINAHL, and PsycINFO were searched from...... inception to January 2015. All English peer-reviewed studies that included patients with both cancer and diabetes and assessed PROs were included. All included studies were independently assessed on methodological quality by two investigators. RESULTS: Of the 3553 identified studies, 10 studies were...... such as depression, patient empowerment and self-management. IMPLICATIONS FOR CANCER SURVIVOR: Having both cancer and diabetes might result in worse PROs, however, more research is needed as current evidence is scarce....
Flosadottir, Vala; Roos, Ewa M; Ageberg, Eva
BACKGROUND/AIM: Consequences of an anterior cruciate ligament (ACL) injury include worse patient-reported outcomes (PROs) and a decrease in activity level. Muscle function can be improved by targeted exercise. Our aims were to investigate cross-sectional and longitudinal associations among lower...... extremity muscle function and PROs after ACL injury. METHODS: Fifty-four participants (15 women, mean 30 years) with ACL injury or reconstruction, from the Knee Anterior Cruciate Ligament, Nonsurgical versus Surgical Treatment (KANON) trial (ISRCTN84752559), were assessed with hop performance, muscle power...... and Activity Rating Scale (ARS). Partial Spearman's rank-order correlation was used to analyse correlations between muscle function and PROs, controlling for gender and treatment. RESULTS: Numerous cross-sectional correlations were observed between muscle function and PROs (rsp≈0.3-0.5, p≤0.045). Worse hop...
Mukesh, M B; Qian, W; Wah Hak, C C; Wilkinson, J S; Barnett, G C; Moody, A M; Wilson, C; Coles, C E
Breast radiotherapy-associated toxicity is often reported using clinical and photographic assessments. The addition of patient-reported outcome measures (PROMs) is becoming more common. This study investigated the concordance between clinician- and patient-reported outcomes. The Cambridge Breast Intensity-modulated Radiotherapy (IMRT) trial prospectively collected data on clinician assessment and PROMs at 2 and 5 years after breast radiotherapy. Clinician assessment included physical examination and photographic assessment. PROMs included European Organization for Research and Treatment of Cancer (EORTC) BR23 questionnaire and four breast radiotherapy-specific questions. The correlation between patient and clinician scores were analysed on an independent patient basis using percentage agreement, Cohen's kappa coefficient (k) and Bowker's test of symmetry. The analysis was repeated after stratifying patients based on age, baseline Hospital Anxiety and Depression Score (HADS) and baseline body image score. At 2 and 5 years, a weak level of concordance was seen between the clinician-based assessment and PROMS for all the five toxicity end points (k = 0.05-0.21), with individual patient-based agreement of 32.9-78.3% and a highly discordant Bowker's test of symmetry (P < 0.001). The most frequently reported moderate-severe toxicity by patients was change in breast appearance (14% at both 2 and 5 years), whereas it was breast induration (36% and 25% at 2 and 5 years, respectively) by the clinicians. The lack of concordance was not affected by patient's age, baseline HADS and baseline body image score. This study found that moderate-severe toxicity reported by patients is low and the overall concordance between clinicians and patients is low. This could be due to methodological limitations or alternatively reflects the subjective nature of PROMs. Incorporation of a patient's perception on treatment-related toxicity will have important implications for treatment
Buckley, Sarah A; Kirtane, Kedar; Walter, Roland B; Lee, Stephanie J; Lyman, Gary H
Outcomes for acute myeloid leukemia remain poor, and treatment decisions must consider not just quantity, but also quality of life (QOL). We conducted a systematic review of studies in patients with acute myeloid leukemia or high-risk myelodysplastic syndrome that incorporated patient-reported outcome (PRO) measures. PubMed and PsycINFO were searched for articles published from January 2000 through June 2016. Forty-one were relevant for our review with more published in recent years. There was considerable inter-study heterogeneity in which instruments were used, and many studies employed multiple (often overlapping) instruments. Longitudinal studies in particular suffered from both high attrition rates due to disease-related mortality as well as waning compliance with questionnaire completion. There remain significant challenges to incorporation of PROs into leukemia trials. Despite these limitations, however, well-implemented PROs can provide important information beyond objective response outcomes and highlight areas of focus for clinicians caring for patients and for future research endeavors. Copyright © 2017 Elsevier Ltd. All rights reserved.
Lyman, Stephen; Hidaka, Chisa
The Center for Medicaid and Medicare Services has recently announced the inclusion of several patient-reported outcome measures (PROMs), including the abbreviated Hip Disability and Osteoarthritis Outcome Score and Knee Injury and Osteoarthritis Outcome Score for joint replacement (HOOS, JR and KOOS, JR) for the purpose of quality assessment in total hip and total knee replacement (THR and TKR). Historically, Center for Medicaid and Medicare Services and other agencies have used measures of process (eg, % vaccinated) or adverse events (eg, infection rates, readmission rates) for quality assessment. However, the use of PROMs has become a priority based on stated goals by the National Quality Strategy and Institute of Medicine for a more patient-centered approach. Here, we review several general health and joint-specific PROMs, which have been extensively used in research to assess treatment efficacy and discuss their relevance to the new criteria for quality assessment, particularly for THR and TKR. Although we expect HOOS, JR and KOOS, JR to yield much useful information in the near term, these surveys are likely an interim solution. In the future, we anticipate that novel measurement platforms, such as wearable technologies or patient-specific surveys, may open new and exciting avenues of research to discover which types of data-perhaps not previously available-best represent patient quality of life and satisfaction after THR, TKR, or other orthopedic procedures. Copyright © 2016 Elsevier Inc. All rights reserved.
Abel, Gregory A; Albelda, Randy; Khera, Nandita; Hahn, Theresa; Salas Coronado, Diana Y; Odejide, Oreofe O; Bona, Kira; Tucker-Seeley, Reginald; Soiffer, Robert
Although hematopoietic cell transplantation (HCT) is the only curative therapy for many advanced hematologic cancers, little is known about the financial hardship experienced by HCT patients nor the association of hardship with patient-reported outcomes. We mailed a 43-item survey to adult patients approximately 180 days after their first autologous or allogeneic HCT at 3 high-volume centers. We assessed decreases in household income; difficulty with HCT-related costs, such as need to relocate or travel; and 2 types of hardship: hardship_1 (reporting 1 or 2 of the following: dissatisfaction with present finances, difficulty meeting monthly bill payments, or not having enough money at the end of the month) and "hardship_2" (reporting all 3). Patient-reported stress was measured with the Perceived Stress Scale-4, and 7-point scales were provided for perceptions of overall quality of life (QOL) and health. In total, 325 of 499 surveys (65.1%) were received. The median days since HCT was 173; 47% underwent an allogeneic HCT, 60% were male, 51% were > 60 years old, and 92% were white. Overall, 46% reported income decline after HCT, 56% reported hardship_1, and 15% reported hardship_2. In multivariable models controlling for income, those reporting difficulty paying for HCT-related costs were more likely to report financial hardship (odds ratio, 6.9; 95% confidence interval, 3.8 to 12.3). Hardship_1 was associated with QOL below the median (odds ratio, 2.9; 95% confidence interval, 1.7 to 4.9), health status below the median (odds ratio, 2.2; 95% confidence interval, 1.3 to 3.6), and stress above the median (odds ratio, 2.1; 95% confidence interval, 1.3 to 3.5). In this sizable cohort of HCT patients, financial hardship was prevalent and associated with worse QOL and higher levels of perceived stress. Interventions to address patient financial hardship-especially those that ameliorate HCT-specific costs-are likely to improve patient-reported outcomes.
Carle, Adam C; Cella, David; Cai, Li; Choi, Seung W; Crane, Paul K; Curtis, S McKay; Gruhl, Jonathan; Lai, Jin-Shei; Mukherjee, Shubhabrata; Reise, Steven P; Teresi, Jeanne A; Thissen, David; Wu, Eric J; Hays, Ron D
In 2002, the NIH launched the 'Roadmap for Medical Research'. The Patient-Reported Outcomes Measurement Information System (PROMIS(®)) is one of the Roadmap's key aspects. To create the next generation of patient-reported outcome measures, PROMIS utilizes item response theory (IRT) and computerized adaptive testing. In 2009, the NIH funded the second wave of PROMIS studies (PROMIS II). PROMIS II studies continue PROMIS's agenda, but also include new features, including longitudinal analyses and more sociodemographically diverse samples. PROMIS II also includes increased emphasis on pediatric populations and evaluation of PROMIS item banks for clinical research and population science. These aspects bring new psychometric challenges. To address this, investigators associated with PROMIS gathered at the Third Psychometric Summit in September 2010 to identify, describe and discuss pressing psychometric issues and new developments in the field, as well as make analytic recommendations for PROMIS. The summit addressed five general themes: linking, differential item functioning, dimensionality, IRT models for longitudinal applications and new IRT software. In this article, we review the discussions and presentations that occurred at the Third PROMIS Psychometric Summit.
Çelik, Derya; Çoban, Özge; Kılıçoğlu, Önder
Purpose: MCID scores for outcome measures are frequently used evidence-based guides to gage meaningful changes. To conduct a systematic review of the quality and content of the the minimal clinically important difference (MCID) relating to 16 patient-rated outcome measures (PROM) used in lower extremity. Methods: We conducted a systematic literature review on articles reporting MCID in lower extremity outcome measures and orthopedics from January 1, 1980, to May 10, 2016. We evaluated MCID of the 16 patient reported outcome measures (PROM) which were Harris Hip Score (HHS), Oxford Hip Score (OHS), Hip Outcome Score (HOS), Hip Disability and Osteoarthritis Outcome Score (HOOS), The International Knee Documentation Committee Subjective Knee Form (IKDC), The Lysholm Scale, The Western Ontario Meniscal Evaluation Tool (WOMET), The Anterior Cruciate Ligament Quality of Life Questionnaire (ACL-QOL), The Lower Extremity Functional Scale (LEFS), The Western Ontario and Mcmaster Universities Index (WOMAC), Knee İnjury And Osteoarthritis Outcome Score (KOOS), Oxford Knee Score (OKS), Kujala Anterior Knee Pain Scale, The Victorian Institute of Sports Assessment Patellar Tendinosis (Jumper’s Knee) (VİSA-P), Tegner Activity Rating Scale, Marx Activity Rating Scale, Foot And Ankle Outcome Score (FAOS), The Foot Function Index (FFI), Foot And Ankle Ability Measure (FAAM), The Foot And Ankle Disability Index Score and Sports Module, Achill Tendon Total Rupture Score(ATRS), The Victorian İnstitute Of Sports Assesment Achilles Questionnaire(VİSA-A), American Orthopaedic Foot and Ankle Society (AOFAS). A search of the PubMed/MEDLINE, PEDro and Cochrane Cen¬tral Register of Controlled Trials and Web of Science databases from the date of inception to May 1, 2016 was conducted. The terms “minimal clinically important difference,” “minimal clinically important change”, “minimal clinically important improvement” “were combined with one of the PROM as mentioned above
Jain, Amit; Kebaish, Khaled M; Sciubba, Daniel M; Hassanzadeh, Hamid; Scheer, Justin K; Neuman, Brian J; Lafage, Virginie; Bess, Shay; Protopsaltis, Themistocles S; Burton, Douglas C; Smith, Justin S; Shaffrey, Christopher I; Hostin, Richard A; Ames, Christopher P
For patients with adult spinal deformity (ASD), surgical treatment may improve their health-related quality of life. This study investigates when the greatest improvement in outcomes occurs and whether incremental improvements in patient-reported outcomes during the first postoperative year predict outcomes at 3 years. Using a multicenter registry, we identified 84 adults with ASD treated surgically from 2008 to 2012 with complete 3-year follow-up. Pairwise t tests and multivariate regression were used for analysis. Significance was set at P years (both P year, ODI and SRS-22r scores improved by 19 and 0.5 points, respectively (both P year predicted 3-year outcomes in ODI and SRS-22r scores (adjusted R(2) = 0.52 and 0.42, respectively). There were no significant differences in the measured or predicted 3-year ODI (P = 0.991) or SRS-22r scores (P = 0.986). In surgically treated patients with ASD, the greatest improvements in outcomes occurred between 6 weeks and 1 year postoperatively. A model with incremental improvements from baseline to 6 weeks and from 6 weeks to 1 year can be used to predict ODI and SRS-22r scores at 3 years. Copyright © 2017 Elsevier Inc. All rights reserved.
Pilkonis, Paul A.; Choi, Seung W.; Reise, Steven P.; Stover, Angela M.; Riley, William T.; Cella, David
The authors report on the development and calibration of item banks for depression, anxiety, and anger as part of the Patient-Reported Outcomes Measurement Information System (PROMIS[R]). Comprehensive literature searches yielded an initial bank of 1,404 items from 305 instruments. After qualitative item analysis (including focus groups and…
Klose, Marianne; Krag, Kirstine Stochholm; Janukonyté, Jurgita
OBJECTIVE: Posttraumatic pituitary hormone deficiency is often suggested. The impact of these predominantly mild and often irreproducible deficiencies on outcome is less clear. The aim of the present study was to describe patient reported outcome in a national a priori unselected cohort of patien...
Bhuskute, Aditi; Skirko, Jonathan R; Roth, Christina; Bayoumi, Ahmed; Durbin-Johnson, Blythe; Tollefson, Travis T
Patients with cleft palate and other causes of velopharyngeal insufficiency (VPI) suffer adverse effects on social interactions and communication. Measurement of these patient-reported outcomes is needed to help guide surgical and nonsurgical care. To further validate the VPI Effects on Life Outcomes (VELO) instrument, measure the change in quality of life (QOL) after speech surgery, and test the association of change in speech with change in QOL. Prospective descriptive cohort including children and young adults undergoing speech surgery for VPI in a tertiary academic center. Participants completed the validated VELO instrument before and after surgical treatment. The main outcome measures were preoperative and postoperative VELO scores and the perceptual speech assessment of speech intelligibility. The VELO scores are divided into subscale domains. Changes in VELO after surgery were analyzed using linear regression models. VELO scores were analyzed as a function of speech intelligibility adjusting for age and cleft type. The correlation between speech intelligibility rating and VELO scores was estimated using the polyserial correlation. Twenty-nine patients (13 males and 16 females) were included. Mean (SD) age was 7.9 (4.1) years (range, 4-20 years). Pharyngeal flap was used in 14 (48%) cases, Furlow palatoplasty in 12 (41%), and sphincter pharyngoplasty in 1 (3%). The mean (SD) preoperative speech intelligibility rating was 1.71 (1.08), which decreased postoperatively to 0.79 (0.93) in 24 patients who completed protocol (P after surgery (Pafter surgery (P = .36). Speech Intelligibility was correlated with preoperative and postoperative total VELO score (P after surgery was correlated with change in speech intelligibility. Speech surgery improves VPI-specific quality of life. We confirmed validation in a population of untreated patients with VPI and included pharyngeal flap surgery, which had not previously been included in validation studies. The VELO
Jennifer Ai-Lian Woo
Full Text Available Purpose: Stereotactic body radiation therapy (SBRT is increasingly utilized as primary treatment for clinically localized prostate cancer. Consensus regarding the appropriate patient-reported outcome (PRO endpoints for clinical trials for early stage prostate cancer RT is lacking. To aid in trial design, this study presents PROs over 36 months following SBRT for clinically localized prostate cancer. Methods: 174 hormone-naïve patients were treated with 35-36.25 Gy SBRT in 5 fractions. Patients completed the EPIC-26 questionnaire at baseline and all follow-ups; the proportion of patients developing a clinically significant decline in each EPIC domain was determined. The minimally important difference (MID was defined as a change of one-half SD from the baseline. Per RTOG 0938, we examined the percentage of patients who reported decline in EPIC urinary summary score of >2 points and EPIC bowel summary score of >5 points from baseline to one year. Results: 174 patients received SBRT with minimum follow-up of 36 months. The proportion of patients reporting a clinically significant decline in EPIC urinary/bowel scores was 34%/30%, 40%/32.2%, and 32.8%/21.5% at 6, 12, and 36 months. The percentage of patients reporting decline in the EPIC urinary summary score of >2 points was 43.2%, 51.6% and 41.8% at 6, 12, and 36 months. The percentage of patients reporting decline in EPIC bowel domain summary score of >5 points was 29.6% 29% and 22.4% at 6, 12, and 36 months. Conclusion: Our treatment protocol meets the RTOG 0938 criteria for advancing to a Phase III trial compared to conventionally fractionated RT. Between 12-36 months, the proportion of patients reporting decrease in both EPIC urinary and bowel scores declined, suggesting late improvement in these domains. Further investigation is needed to elucidate 1 which domains bear the greatest influence on post-treatment QOL, and 2 at what time point PRO endpoint(s should be assessed.
Jackson, Matthew J; N'Dow, James; Pickard, Rob
Patient-reported outcome measures (PROMs) are now recognised as the most appropriate instruments to assess the effectiveness of healthcare interventions from the patient's perspective. The purpose of this review was to identify recent publications describing the use of PROMs following reconstructive urological surgery. A wide systematic search identified only three original articles published in the last 2 years that prospectively assessed effectiveness using a patient-completed condition-specific or generic health-related quality of life (HRQoL) instrument. These publications illustrate the need to administer PROMs at a postoperative interval relevant to the anticipated recovery phase of individual procedures. They also highlight the difference in responsiveness of generic HRQoL instruments to symptomatic improvement between straightforward conditions such as pelviureteric junction obstruction and complex multidimensional conditions such as meningomyelocele. PROMs uptake and awareness is increasing in reconstructive urology but more work is required to demonstrate the effectiveness of surgical procedures for patients and healthcare funders alike. Healthcare policy-makers now rely on these measures to determine whether specific treatments are worth financing and to compare outcomes between institutions.
Snowden, Austyn; Telfer, Iain
Chaplains are employed by health organizations around the world to support patients in recognizing and addressing their spiritual needs. There is currently no generalizable measure of the impact of these interventions and so the clinical and strategic worth of chaplaincy is difficult to articulate. This article introduces the Scottish PROM, an original five-item patient reported outcome measure constructed specifically to address this gap. It describes the validation process from its conceptual grounding in the spiritual care literature through face and content validity cycles. It shows that the Scottish PROM is internally consistent and unidimensional. Responses to the Scottish PROM show strong convergent validity with responses to the Warwick and Edinburgh Mental Well-Being Scale, a generic well-being scale often used as a proxy for spiritual well-being. In summary, the Scottish PROM is fit for purpose. It measures the outcomes of spiritual care as delivered by chaplains in this study. This novel project introduces an essential and original breakthrough; the possibility of generalizable international chaplaincy research.
Khavanin, Nima; Clemens, Mark W; Pusic, Andrea L; Fine, Neil A; Hamill, Jennifer B; Kim, H Myra; Qi, Ji; Wilkins, Edwin G; Kim, John Y S
Since the 2012 approval of shaped implants, their use in breast reconstruction has increased in the United States. However, large-scale comparisons of complications and patient-reported outcomes are lacking. The authors endeavored to compare surgical and patient-reported outcomes across implant types. The Mastectomy Reconstruction Outcomes Consortium database was queried for expander/implant reconstructions with at least 1-year postexchange follow-up (mean, 18.5 months). Outcomes of interest included postoperative complications, 1-year revisions, and patient-reported outcomes. Bivariate and mixed-effects regression analyses evaluated the effect of implant type on patient outcomes. Overall, 822 patients (73.5 percent) received round and 297 patients (26.5 percent) received shaped implants. Patients undergoing unilateral reconstructions with round implants underwent more contralateral symmetry procedures, including augmentations (round, 18.7 percent; shaped, 6.8 percent; p = 0.003) and reductions (round, 32.2 percent; shaped, 20.5 percent; p = 0.019). Shaped implants were associated with higher rates of infection (shaped, 6.1 percent; round, 2.3 percent; p = 0.002), that remained significant after multivariable adjustment. Other complication rates did not differ significantly between cohorts. Round and shaped implants experienced similar 2-year patient-reported outcome scores. This prospective, multicenter study is the largest evaluating outcomes of shaped versus round implants in breast reconstruction. Although recipients of round implants demonstrated lower infection rates compared with shaped implants, these patients were more likely to undergo contralateral symmetry procedures. Both implant types yielded comparable patient-reported outcome scores. With appropriate patient selection, both shaped and round implants can provide acceptable outcomes in breast reconstruction. Therapeutic, III.
Fam, Maged D; Zeineddine, Hussein A; Nassir, Rafiq Muhammed; Bhatt, Pragnesh; Kamel, Mahmoud H
Background: Transverse (type II) odontoid process fracture is among the most commonly encountered cervical spine fractures. Nonsurgical management through external immobilization is occasionally preferred to surgical management but is criticized for its higher rates of failure and lower patient satisfaction. Our aim is to analyze patient-reported outcomes in patients who underwent nonsurgical treatment for type II odontoid fractures. Methods: We identified patients >18-year-old who underwent external immobilization as a treatment for isolated type II odontoid fracture between 2007 and 2012. We collected demographic parameters, clinical presentation, mode of injury, imaging studies and modality and duration of treatment (soft collar, halo-vest, or both). Patients were contacted by telephone to participate in a 15-min survey addressing their recovery including their subjective rate of return to preinjury level of functioning. Results: Fifteen patients met the inclusion/exclusion criteria and participated in our survey. Patients were followed up for an average of 19 months after injury. Overall mean age was 61 years. Injury followed a mechanical fall or a road traffic accident in 11 and 4 cases, respectively. External immobilization was achieved by halo vest only in nine patients, soft collar only in two patients (13%), and through a sequential combination in the remaining 4 (27%). This was deployed for a mean of 7.8 months. Radiological studies at the last follow-up showed bony healing (27%), fibrous nonunion (60%), and persistent instability (13%). Patients reported gradual recovery of function throughout the 1st year after injury with levels above 70% of preinjury functioning achieved by 13% of patients at 6 months, 33% at 9 months, and 47% at 12 months. Overall satisfaction with nonsurgical management was 68%. Conclusion: In selected patients with type II odontoid fractures, external immobilization represents a good option with acceptable course of recovery. PMID
Basch, Ethan; Deal, Allison M; Kris, Mark G; Scher, Howard I; Hudis, Clifford A; Sabbatini, Paul; Rogak, Lauren; Bennett, Antonia V; Dueck, Amylou C; Atkinson, Thomas M; Chou, Joanne F; Dulko, Dorothy; Sit, Laura; Barz, Allison; Novotny, Paul; Fruscione, Michael; Sloan, Jeff A; Schrag, Deborah
There is growing interest to enhance symptom monitoring during routine cancer care using patient-reported outcomes, but evidence of impact on clinical outcomes is limited. We randomly assigned patients receiving routine outpatient chemotherapy for advanced solid tumors at Memorial Sloan Kettering Cancer Center to report 12 common symptoms via tablet computers or to receive usual care consisting of symptom monitoring at the discretion of clinicians. Those with home computers received weekly e-mail prompts to report between visits. Treating physicians received symptom printouts at visits, and nurses received e-mail alerts when participants reported severe or worsening symptoms. The primary outcome was change in health-related quality of life (HRQL) at 6 months compared with baseline, measured by the EuroQol EQ-5D Index. Secondary endpoints included emergency room (ER) visits, hospitalizations, and survival. Among 766 patients allocated, HRQL improved among more participants in the intervention group than usual care (34% v 18%) and worsened among fewer (38% v 53%; P < .001). Overall, mean HRQL declined by less in the intervention group than usual care (1.4- v 7.1-point drop; P < .001). Patients receiving intervention were less frequently admitted to the ER (34% v 41%; P = .02) or hospitalized (45% v 49%; P = .08) and remained on chemotherapy longer (mean, 8.2 v 6.3 months; P = .002). Although 75% of the intervention group was alive at 1 year, 69% with usual care survived the year (P = .05), with differences also seen in quality-adjusted survival (mean of 8.7 v. 8.0 months; P = .004). Benefits were greater for participants lacking prior computer experience. Most patients receiving intervention (63%) reported severe symptoms during the study. Nurses frequently initiated clinical actions in response to e-mail alerts. Clinical benefits were associated with symptom self-reporting during cancer care. © 2015 by American Society of Clinical Oncology.
Kadakia, Kunal C.; Snyder, Claire F.; Kidwell, Kelley M.; Seewald, Nicholas J.; Flockhart, David A.; Skaar, Todd C.; Desta, Zereunesay; Rae, James M.; Otte, Julie L.; Carpenter, Janet S.; Storniolo, Anna M.; Hayes, Daniel F.; Stearns, Vered
Background. Early discontinuation of aromatase inhibitors (AIs) is common and leads to poor outcomes but is challenging to predict. In the Exemestane and Letrozole Pharmacogenetics trial, a high rate of early discontinuation due to intolerance was observed. We hypothesized that early changes in patient-reported outcomes (PROs) predict AI discontinuation and that biochemical factors are associated with changes in PROs. Patients and Methods. Postmenopausal women with early-stage breast cancer enrolled in a prospective randomized trial of exemestane versus letrozole completed questionnaires at baseline and serially over 24 months to assess overall quality of life (EuroQOL Visual Analog Scale [VAS]); mood; and multiple symptoms, including a musculoskeletal symptom cluster. A joint mixed-effects/survival model was used to estimate the effect of the change in PROs on AI discontinuation. Associations between biochemical factors and change in PROs were examined. Results. A total of 490 patients were analyzed. Worsening of EuroQOL VAS and the musculoskeletal cluster were associated with the highest risk for early discontinuation (hazard ratio [HR], 2.77 [95% confidence interval (CI), 2.72–2.81; p = .015]; HR, 4.39 [95% CI, 2.40–8.02; p < .0001], respectively). Pharmacokinetics and estrogen metabolism were not consistently associated with change in PRO measures. No clinically significant differences in any PRO between AIs were observed. Conclusion. Changes in PROs early during AI therapy were associated with treatment discontinuation. Identification of these changes could be used to target interventions in patients at high risk for early discontinuation. Implications for Practice: Early changes in patient-reported outcomes (PROs) can predict nonpersistence to aromatase inhibitor therapy. If used in clinical practice, PROs might identify women at highest risk for early discontinuation and allow for interventions to improve tolerance before significant toxicities develop
Brettschneider, Christian; Lühmann, Dagmar; Raspe, Heiner
"Patient-Reported Outcome" (PRO) is used as an umbrella term for different concepts for measuring subjectively perceived health status e. g. as treatment effects. Their common characteristic is, that the appraisal of the health status is reported by the patient himself. In order to describe the informative value of PRO in Health Technology Assessment (HTA) first an overview of concepts, classifications and methods of measurement is given. The overview is complemented by an empirical analysis of clinical trials and HTA-reports on rheumatoid arthritis and breast cancer in order to report on type, frequency and consequences of PRO used in these documents. For both issues systematic reviews of the literature have been performed. The search for methodological literature covers the publication period from 1990 to 2009, the search for clinical trials of rheumatoid arthritis and breast cancer covers the period 2005 to 2009. Both searches were performed in the medical databases of the German Institute of Medical Documentation and Information (DIMDI). The search for HTA-reports and methodological papers of HTA-agencies was performed in the CRD-Databases (CRD = Centre for Reviews and Dissemination) and by handsearching the websites of INAHTA member agencies (INAHTA = International Network of Agencies for Health Technology Assessment). For all issues specific inclusion and exclusion criteria were defined. The methodological quality of randomized controlled trials (RCT) was assessed by a modified version of the Cochrane Risk of Bias Tool. For the methodological part information extraction from the literature is structured by the report's chapters, for the empirical part data extraction sheets were constructed. All information is summarized in a qualitative manner. Concerning the methodological issues the literature search retrieved 158 documents (87 documents related to definition or classification, 125 documents related to operationalisation of PRO). For the empirical analyses
Carlson, Eve B.; Field, Nigel P.; Ruzek, Josef I.; Bryant, Richard A.; Dalenberg, Constance J.; Keane, Terence M.; Spain, David A.
Objectives Ambulatory assessment data collection methods are increasingly used to study behavior, experiences, and patient reported outcomes (PROs) such as emotions, cognitions, and symptoms in clinical samples. Data collected close in time at frequent and fixed intervals can assess PROs that are discrete or changing rapidly and provide information about temporal dynamics or mechanisms of change in clinical samples and individuals, but clinical researchers have not yet routinely and systematically investigated the reliability and validity of such measures or their potential added value over conventional measures. The present study provides a comprehensive, systematic evaluation of the psychometrics of several Proximal Intensive Assessment (PIA) measures in a clinical sample and investigates whether PIA appears to assess meaningful differences in phenomena over time. Methods Data was collected on a variety of psychopathology constructs on handheld devices every 4 hours for 7 days from 62 adults recently exposed to traumatic injury of themselves or a family member. Data was also collected on standard self-report measures of the same constructs at the time of enrollment, one week after enrollment, and two months after injury. Results For all measure scores, results showed good internal consistency across items and within persons over time, provided evidence of convergent, divergent, and construct validity, and showed significant between and within-subject variability. Conclusions Results indicate that PIA measures can provide valid measurement of psychopathology in a clinical sample. PIA may be useful to study mechanisms of change in clinical contexts, identify targets for change, and gauge treatment progress. PMID:26567018
Behrend, Henrik; Zdravkovic, Vilijam; Giesinger, Johannes M; Giesinger, Karlmeinrad
To measure joint awareness in patients who have undergone anterior cruciate ligament (ACL) reconstruction and to investigate medium- and long-term results of the procedure. All patients who had undergone ACL reconstruction with the same arthroscopic surgical technique at our institution between 2011 and 2014 (medium-term follow-up group (Group I)) or between 2000 and 2005 (long-term follow-up group (Group II)) were considered for inclusion in the study. A group of healthy controls were recruited to obtain reference values for the FJS-12 (Forgotten Joint Score-12). Propensity score matching was applied to improve comparability of patients and healthy controls in terms of sex and age. Fifty-eight patients of the Group I (mean follow-up 31.5 (SD13.4) months, range 12-54), 57 patients of the Group II (mean follow-up 139 (SD15.2) months, range 120-179), and the healthy control samples (100 individuals) were analysed. Significantly lower FJS-12 was found in both groups (Group I: 71.6 and Group II: 70.1), compared to the two matched control groups (88.1 and 90.0). The concept of joint awareness was successfully applied to evaluate medium- and long-term results of ACL reconstruction. The clinical relevance of this study is that it extends the construct of joint awareness as a patient-reported outcome parameter to ACL reconstruction surgery. Level III.
Full Text Available Objective. Patient-reported outcomes (PROs have become an essential part of the assessment of patients with rheumatoid arthritis (RA. We aimed to evaluate the agreement and correlation between PROs and the physician's measurements. Methods. This was a cross-sectional analytical study in which 135 patients with RA were clinically evaluated during two different sessions of focus group interviews. Rheumatologist recorded 28 swollen (SJCs and tender joint counts (TJCs. The patients filled out the PROs instruments (MDHAQ, RADAI, RAPID3, 4, and 5 and self-report articular index (SAI diagram for pain and joint swelling. DAS28 was calculated (C-reactive protein. An adjusted multiple lineal regression model was done (DAS28 as dependent variable. Results. Highly significant agreements were found between SJC and TJC registered by the physician and patient. There was moderate correlation between DAS28 with patient SJC (r=0.52, patient TJC (r=0.55, RADAI (r=0.56, RAPID3 (r=0.52, RAPID4 (r=0.56, RAPID5 (r=0.66, and VAS-Global (r=0.51. Likewise, we found moderate to high correlations between CDAI and SDAI with all variable measurements done by the patients. The resulting predictive equation was DAS28(CRP=2.02+0.037×RAPID4+0.042× patient SJC. Conclusion. PROs applied in focus groups interview are a useful tool for managing patients with RA regardless of gender, educational level, and duration of disease.
Caetano, Thais Angelina; Ribeiro, Adriana Barbosa; Della Vecchia, Maria Paula; Cunha, Tatiana Ramirez; Chaves, Carolina de Andrade Lima
PURPOSE The aim of this study was to determine whether two methods of documentation, print and electronic forms, for the assessment of patient-reported outcomes (PRO) in complete denture wearers provide comparable results. The study also quantified the time needed for filling the forms by each method. MATERIALS AND METHODS Thirty participants enrolled in a university clinic answered two forms (a questionnaire for denture satisfaction and OHIP-EDENT). They provided answers with two application methods in a random order, with a one-month interval between them: (1) electronic forms on a tablet computer; and (2) print forms. The methods were compared in terms of mean results, correlation/agreement, internal consistency, and spent time. RESULTS Mean results for both methods were similar for each denture satisfaction item (100-mm VAS) and OHIP-EDENT summary score. Both questionnaires presented good internal consistency regardless of the application method (Cronbach's α=0.86 or higher). Correlation and agreement between the methods regarding specific items was at least moderate for the majority of cases. Mean time for the electronic and print forms were 9.2 and 8.5 minutes, respectively (paired t test, P=.06, non-significant). CONCLUSION The electronic method is comparable to print forms for the assessment of important PRO of prosthetic treatment for edentulism, considering the results and time needed. Findings suggest the viability of replacing print forms with a tablet for applying the tested inventories in clinical trials. PMID:28018563
Amaya-Amaya, Jenny; Botello-Corzo, Diana; Calixto, Omar-Javier; Calderón-Rojas, Rolando; Domínguez, Aura-Maria; Cruz-Tapias, Paola; Montoya-Ortiz, Gladis; Mantilla, Ruben-Dario; Anaya, Juan-Manuel; Rojas-Villarraga, Adriana
Objective. Patient-reported outcomes (PROs) have become an essential part of the assessment of patients with rheumatoid arthritis (RA). We aimed to evaluate the agreement and correlation between PROs and the physician's measurements. Methods. This was a cross-sectional analytical study in which 135 patients with RA were clinically evaluated during two different sessions of focus group interviews. Rheumatologist recorded 28 swollen (SJCs) and tender joint counts (TJCs). The patients filled out the PROs instruments (MDHAQ, RADAI, RAPID3, 4, and 5 and self-report articular index (SAI) diagram for pain and joint swelling). DAS28 was calculated (C-reactive protein). An adjusted multiple lineal regression model was done (DAS28 as dependent variable). Results. Highly significant agreements were found between SJC and TJC registered by the physician and patient. There was moderate correlation between DAS28 with patient SJC (r = 0.52), patient TJC (r = 0.55), RADAI (r = 0.56), RAPID3 (r = 0.52), RAPID4 (r = 0.56), RAPID5 (r = 0.66), and VAS-Global (r = 0.51). Likewise, we found moderate to high correlations between CDAI and SDAI with all variable measurements done by the patients. The resulting predictive equation was DAS28(CRP) = 2.02 + 0.037 × RAPID4 + 0.042× patient SJC. Conclusion. PROs applied in focus groups interview are a useful tool for managing patients with RA regardless of gender, educational level, and duration of disease. PMID:23097701
Friedlander, M; Mercieca-Bebber, R L; King, M T
Despite increased recognition of the value of including patient-reported outcomes (PROs) as important end points in phase III clinical trials, there has been a lack of pre-specified PRO hypotheses and shortcomings with the analyses and interpretation of PROs in many ovarian cancer trials. This paper discusses and provides examples of the so-called lost opportunities in ovarian cancer trials. These include: (i) no clear pre-specified PRO hypotheses; (ii) PRO end points not included; (iii) insensitive PRO end point selection; (iv) collection of poor-quality PRO data not suitable for analysis; (v) differences in PROs between treatment arms ignored; and (vi) poor reporting quality. We can learn from the past and with relatively little additional effort, improve the collection and interpretation of PRO data in future ovarian cancer trials. The importance of doing so is underpinned by recent initiatives to improve the standard and usefulness of PRO data in clinical trials. These include the Food and Drug Administration (FDA) Guidance for PROs to support labelling claims, the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO MCBS), the International Society for Quality-of-Life Research PRO reporting guidance and the Consolidated Standards of Reporting Clinical Trials (CONSORT)-PRO-extension statement which includes a checklist of recommended items to include in PRO sections of trial protocols. Promoting the importance of hypothesis-driven PROs in ovarian cancer clinical trials will lead to improvements in the design of these trials and the interpretation of their results.
Derek G Kyte
Full Text Available BACKGROUND: Patient reported outcomes (PROs are increasingly assessed in clinical trials, and guidelines are available to inform the design and reporting of such trials. However, researchers involved in PRO data collection report that specific guidance on 'in-trial' activity (recruitment, data collection and data inputting and the management of 'concerning' PRO data (i.e., data which raises concern for the well-being of the trial participant appears to be lacking. The purpose of this review was to determine the extent and nature of published guidelines addressing these areas. METHODS AND FINDINGS: Systematic review of 1,362 articles identified 18 eligible papers containing 'in-trial' guidelines. Two independent authors undertook a qualitative content analysis of the selected papers. Guidelines presented in each of the articles were coded according to an a priori defined coding frame, which demonstrated reliability (pooled Kappa 0.86-0.97, and validity (<2% residual category coding. The majority of guidelines present were concerned with 'pre-trial' activities (72%, for example, outcome measure selection and study design issues, or 'post-trial' activities (16% such as data analysis, reporting and interpretation. 'In-trial' guidelines represented 9.2% of all guidance across the papers reviewed, with content primarily focused on compliance, quality control, proxy assessment and reporting of data collection. There were no guidelines surrounding the management of concerning PRO data. CONCLUSIONS: The findings highlight there are minimal in-trial guidelines in publication regarding PRO data collection and management in clinical trials. No guidance appears to exist for researchers involved with the handling of concerning PRO data. Guidelines are needed, which support researchers to manage all PRO data appropriately and which facilitate unbiased data collection.
Flosadottir, Vala; Roos, Ewa M; Ageberg, Eva
Background/aim Consequences of an anterior cruciate ligament (ACL) injury include worse patient-reported outcomes (PROs) and a decrease in activity level. Muscle function can be improved by targeted exercise. Our aims were to investigate cross-sectional and longitudinal associations among lower extremity muscle function and PROs after ACL injury. Methods Fifty-four participants (15 women, mean 30 years) with ACL injury or reconstruction, from the Knee Anterior Cruciate Ligament, Nonsurgical versus Surgical Treatment (KANON) trial (ISRCTN84752559), were assessed with hop performance, muscle power and postural orientation 3 years (SD 0.85) after ACL injury. PROs at 3 and 5 years after injury included Knee Injury and Osteoarthritis Outcome Score (KOOS) subscales Function in sport and recreation (KOOS Sport/rec) and Knee-related Quality of life (KOOS QoL), KOOS item Q3 (KOOS Q3), Tegner Activity Scale and Activity Rating Scale (ARS). Partial Spearman's rank-order correlation was used to analyse correlations between muscle function and PROs, controlling for gender and treatment. Results Numerous cross-sectional correlations were observed between muscle function and PROs (rsp≈0.3–0.5, p≤0.045). Worse hop performance and worse postural orientation were associated with worse KOOS scores 2 years later (rsp≥0.280, p≤0.045). Worse muscle power was associated with lower future ARS scores (rsp=0.281, p=0.044). Conclusions The moderate associations suggest that improving muscle function during rehabilitation could improve present and future PROs. PMID:27900196
Sadeghi, Salva; Fayed, Nora; Ronen, Gabriel M
Patient-reported outcome (PRO) measures that assess the effect of epilepsy on children's lives include the concepts of health, health-related quality of life (HRQOL), and quality of life (QOL). They also contain varied health and health-related content. Our objectives were to identify what generic and epilepsy-specific PRO instruments are used in childhood epilepsy research and to make explicit their conceptual approach and biopsychosocial content. MEDLINE, EMBASE, and PsycINFO were searched from 2001 to 2011 for PRO measures used in pediatric epilepsy. Measures were analyzed on an item-by-item basis according to World Health Organization (WHO) definitions of QOL and the International Classification of Functioning, Disability and Health for Children and Youth (ICF-CY) biopsychosocial health framework to distinguish the conceptual approach within each measure. The health content analysis coded each item according to specific ICF-CY components of body function, activity and participation, environment, or personal factors to determine the health content for each measure. Three generic and 13 epilepsy-specific PRO measures were identified; 10 of 16 measures utilized a biopsychosocial health approach rather than an HRQOL or QOL approach. Content analysis showed that in 11 of 16 measures, >25% of the items represented participation and activity components of the ICF-CY, whereas a high proportion of environment items were found in only one epilepsy-specific measure. This comprehensive review provides information aiding clinicians and researchers in the selection of the appropriate PRO instruments for children with epilepsy on the basis of content. Most epilepsy-specific and generic PROs use a biopsychosocial health approach as opposed to a subjective HRQOL/QOL approach to measurement. Clinicians and researchers must be aware of these concepts and content when intending to measure outcomes validly. Wiley Periodicals, Inc. © 2014 International League Against Epilepsy.
Shortell, Stephen M; Poon, Bing Ying; Ramsay, Patricia P; Rodriguez, Hector P; Ivey, Susan L; Huber, Thomas; Rich, Jeremy; Summerfelt, Tom
depression symptoms (OR = 2.26; CI 1.79, 2.86), better physical health (OR = 2.56; CI 2.00, 3.27), and better social health functioning (OR = 4.12; CI 3.21, 5.29). Patient activation (PAM-13) mediated the positive association between patients' experience of chronic illness care and each of the three patient-reported outcome measures-fewer depression symptoms, better physical health, and better social health. Relational coordination and shared decision-making activities reported by practices were not significantly associated with higher patient-reported outcome scores. Diabetic and CVD patients who received care from ACO-affiliated practices with more developed patient-centered cultures reported lower PHQ-4 depression symptom scores and better physical functioning. Diabetic and CVD patients who were more highly activated to participate in their care reported lower PHQ-4 scores and better physical and social outcomes of care.
Full Text Available Blayne Welk,1 Sarah A Morrow,2 Wendy Madarasz,3 Patrick Potter,4 Keith Sequeira41Department of Surgery, Division of Urology, 2Department of Clinical Neurosciences, Western University, London, ON, Canada; 3St Joseph's Health Care, London Ontario, Canada; 4Department of Physical Medicine and Rehabilitation, Western University, London, ON, CanadaBackground: There is no single patient-reported instrument that was developed specifically to assess symptoms and bladder-related consequences for neurogenic bladder dysfunction. The purpose of this study was to identify and consolidate items for a novel measurement tool for this population.Methods: Item generation was based on a literature review of existing instruments, open-ended semistructured interviews with patients, and expert opinion. Judgment-based item reduction was performed by a multidisciplinary expert group. The proposed questionnaire was sent to external experts for review.Results: Eight neurogenic quality of life measures and 29 urinary symptom-specific instruments were identified. From these, 266 relevant items were extracted and used in the creation of the new neurogenic symptom score. Qualitative interviews with 16 adult patients with neurogenic bladder dysfunction as a result of spinal cord injury, multiple sclerosis, or spina bifida were completed. Dominant themes included urinary incontinence, urinary tract infections, urgency, and bladder spasms. Using the literature review and interview data, 25 proposed items were reviewed by 12 external experts, and the questions evaluated based on importance on a scale of 1 (not important to 5 (very important. Retained question domains had high mean importance ratings of 3.1 to 4.3 and good agreement with answer hierarchy.Conclusion: The proposed neurogenic bladder symptom score is a novel patient-reported outcome measure. Further work is underway to perform a data-based item reduction and to assess the validity and reliability of this instrument
Feasibility test of a UK-scalable electronic system for regular collection of patient-reported outcome measures and linkage with clinical cancer registry data: The electronic Patient-reported Outcomes from Cancer Survivors (ePOCS system
Full Text Available Abstract Background Cancer survivors can face significant physical and psychosocial challenges; there is a need to identify and predict which survivors experience what sorts of difficulties. As highlighted in the UK National Cancer Survivorship Initiative, routine post-diagnostic collection of patient reported outcome measures (PROMs is required; to be most informative, PROMs must be linked and analysed with patients' diagnostic and treatment information. We have designed and built a potentially cost-efficient UK-scalable electronic system for collecting PROMs via the internet, at regular post-diagnostic time-points, for linking these data with patients' clinical data in cancer registries, and for electronically managing the associated patient monitoring and communications; the electronic Patient-reported Outcomes from Cancer Survivors (ePOCS system. This study aims to test the feasibility of the ePOCS system, by running it for 2 years in two Yorkshire NHS Trusts, and using the Northern and Yorkshire Cancer Registry and Information Service. Methods/Design Non-metastatic breast, colorectal and prostate cancer patients (largest survivor groups, within 6 months post-diagnosis, will be recruited from hospitals in the Yorkshire Cancer Network. Participants will be asked to complete PROMS, assessing a range of health-related quality-of-life outcomes, at three time-points up to 15 months post-diagnosis, and subsequently to provide opinion on the ePOCS system via a feedback questionnaire. Feasibility will be examined primarily in terms of patient recruitment and retention rates, the representativeness of participating patients, the quantity and quality of collected PROMs data, patients' feedback, the success and reliability of the underpinning informatics, and the system running costs. If sufficient data are generated during system testing, these will be analysed to assess the health-related quality-of-life outcomes reported by patients, and to explore
Abma, I.L.; Wees, P.J. van der; Veer, V.; Westert, G.P.; Rovers, M.M.
This systematic review summarizes the evidence regarding the quality of patient-reported outcome measures (PROMs) validated in patients with obstructive sleep apnea (OSA). We performed a systematic literature search of all PROMs validated in patients with OSA, and found 22 measures meeting our
Hendrikx, J.; Fransen, J.; Riel, P.L.C.M. van
OBJECTIVES: The objective of this proof of concept study was to evaluate alerts generated by a patient-reported outcome measure (PROM)-based algorithm for monitoring patients with rheumatoid arthritis (RA). METHODS: The algorithm was constructed using an example PROM score of an equally weighted mea
Pedersen, Susanne S; Versteeg, Henneke; Nielsen, Jens Cosedis;
Few studies have investigated the association between implantable cardioverter defibrillators (ICDs) and lead advisory notifications and patient-reported outcomes (PROs). We examined (i) whether the mode used to inform patients about a device advisory is associated with PROs, and (ii) whether pat...
Roder, Esther; Berger, Marjolein Y.; Hop, Wim C. J.; de Groot, Hans; van Wijk, Roy Gerth
Background: Patient-reported outcomes (PROs) are the only instruments available to assess the efficacy of an intervention in patients with allergic rhinoconjunctivitis. As allergic rhinoconjunctivitis is a systemic disease, it is now recommended to use not only PROs focusing at classical symptoms, b
Draak, T.H.; Gorson, K.C.; Vanhoutte, E.K.; Nes, S.I. van; Doorn, P.A. van; Cornblath, D.R.; Berg, L.H. van den; Faber, C.G.; Merkies, I.S.; Raaphorst, J.
BACKGROUND AND PURPOSE: There is increasing interest in using patient-reported outcome measures (PROMs) in clinical studies to capture individual changes over time. However, PROMs have also been criticized because they are entirely subjective. Our objective was to examine the relationship between a
Hewlett, Sarah E.; Nicklin, Joanna; Bode, Christina; Carmona, Loretto; Dures, Emma; Engelbrecht, Matthias; Hagel, Sofia; Kirwan, John R.; Molto, Anna; Redondo, Marta; Gossec, Laure
Objective. Cross-cultural translation of patient-reported outcome measures (PROMs) is a lengthy process, often performed professionally. Cognitive interviewing assesses patient comprehension of PROMs. The objective was to evaluate the usefulness of cognitive interviewing to assess translations and c
Mahieu, M A; Ahn, G E; Chmiel, J S; Dunlop, D D; Helenowski, I B; Semanik, P; Song, J; Yount, S; Chang, R W; Ramsey-Goldman, R
Fatigue is a common symptom in systemic lupus erythematosus (SLE), and engaging in physical activity may reduce fatigue. We aimed to characterize relationships between fatigue, other health status measures assessed with the Patient Reported Outcomes Measurement Information System (PROMIS) instruments, and accelerometer-based physical activity measurements in patients with SLE. The internal consistency of each PROMIS measure in our SLE sample was also evaluated. This cross-sectional study analyzed 123 adults with SLE. The primary fatigue outcome was Fatigue Severity Scale score. Secondary outcomes were PROMIS standardized T-scores in seven health status domains. Accelerometers were worn for seven days, and mean daily minutes of light, moderate/vigorous, and bouted (10 minutes) moderate/vigorous physical activity were estimated. Cronbach's alpha was determined for each PROMIS measure to assess internal consistency. Relationships between Fatigue Severity Scale, PROMIS, and physical activity were summarized with Spearman partial correlation coefficients (r), adjusted for average daily accelerometer wear time. Mean Fatigue Severity Scale score (4.3, SD 1.6) was consistent with clinically relevant levels of fatigue. Greater daily and bouted moderate/vigorous physical activity minutes correlated with lower Mean Fatigue Severity Scale score (r = -0.20, p = 0.03 and r = -0.30, p = 0.0007, respectively). For PROMIS, bouted moderate/vigorous physical activity minutes correlated with less fatigue (r = -0.20, p = 0.03). PROMIS internal consistency was excellent, with Cronbach's alpha > 0.90 for each domain. Mean PROMIS T-scores for fatigue, pain interference, anxiety, sleep disturbance, sleep-related impairment, and physical function were worse than reported for the general US population. More moderate/vigorous physical activity minutes were associated with less pain interference (r = -0.22, p = 0.01). Both light physical activity and
Houston, Megan N; Hoch, Johanna M; Hoch, Matthew C
A comprehensive systematic literature review of the health-related quality-of-life (HRQOL) differences among individuals with chronic ankle instability (CAI), ankle-sprain copers, and healthy control participants has not been conducted. It could provide a better indication of the self-reported deficits that may be present in individuals with CAI. To systematically summarize the extent to which HRQOL deficits are present in individuals with CAI. We searched for articles in the electronic databases of EBSCO Host and PubMed Central using key words chronic, functional, mechanical, coper, instability, sprains, and patient-assessed. We also performed a hand search of reference lists, authors, and patient-reported outcomes (PROs) of the articles screened for inclusion. Studies were included if they (1) incorporated a PRO as a participant descriptor or as a study outcome to compare adults with CAI to ankle-sprain copers or healthy controls, (2) were written in English, and (3) were published in peer-reviewed journals. Two authors independently assessed methodologic quality using the modified Downs and Black Index. Articles were filtered into 3 categories based on between-groups comparisons: CAI and copers, CAI and healthy control participants, copers and healthy participants. We calculated Hedges g effect sizes and 95% confidence intervals to examine PRO group differences. Of the 124 studies assessed for eligibility, 27 were included. A total of 24 articles compared PROs in individuals with CAI and healthy controls, 7 compared individuals with CAI and copers, and 4 compared copers and healthy controls. Quality scores on the modified Downs and Black Index ranged from 52.9% to 88.2%, with 8 high-, 16 moderate-, and 3 low-quality studies. Overall, we observed moderate to strong evidence that individuals with CAI displayed deficits on generic and region-specific PROs compared with copers and healthy controls. However, evidence that differences exist between copers and healthy
Full Text Available To describe patient preferences in selecting specific biologics and compare clinical response using patient reported outcomes (PROs among patients with rheumatoid arthritis (RA started on different anti-tumor necrosis factor (TNF therapies.Participants were enrollees in Kaiser Permanente Northern California. Patients with RA who had at least two provider visits and started a new anti-TNF therapy from 10/2010-8/2011, were eligible for participation in this longitudinal study. Using a telephone survey, patient preferences in biologic selection and RAPID3, MDHAQ, and SF-12 scores were collected at baseline and at 6 months. Patient scores rating injection/infusion-site burning and stinging (ISBS were collected at 6 months.In all, 267 patients with RA responded to the baseline survey, of whom 57% preferred an injectable biologic, 22% preferred an infused biologic, and 21% had no preference. Motivation for injectable biologics was convenience (92% and for infusion therapy was dislike or lack of self-efficacy for self-injection (16%. After 6 months of treatment with anti-TNF, 70% of the 177 patients who answered the ISBS question reported ISBS with the last dose; on a scale of 1 (none to 10 (worst, 41% of these reported a score of 2-5; and 29% reported a score of 6-10. Adalimumab users experienced 3.2 times (95% confidence interval 1.2-8.6 the level of ISBS that etanercept users experienced. There were no significant differences in RAPID3, MDHAQ, or SF-12 scores between etanercept or adalimumab initiators.Convenience and fear of self-injection were important considerations to patients selecting a biologic drug. Although more convenient, adalimumab associated with more ISBS than did etanercept, and this rate was higher than reported in clinical trials. At 6 months, PROs did not differ between etanercept and adalimumab users.
Matsumoto, Mikio; Baba, Tomonori; Homma, Yasuhiro; Kobayashi, Hideo; Ochi, Hironori; Yuasa, Takahito; Behrend, Henrik; Kaneko, Kazuo
The Forgotten Joint Score-12 (FJS-12) is for patients to forget their artificial joint and is reportedly a useful patient-reported outcome tool for artificial joints. The purpose of this study was to determine whether the FJS-12 is as useful as the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) or the Japanese Orthopaedic Association Hip Disease Evaluation Questionnaire (JHEQ) in Japan. All patients who visited our hospital's hip joint specialists following unilateral THA from August 2013 to July 2014 were evaluated. Medical staff members other than physicians administered three questionnaires. Items evaluated were (1) the reliability of the FJS-12 and (2) correlations between the FJS-12 and the total and subscale scores of the WOMAC or JHEQ. Of 130 patients, 22 were excluded. Cronbach's α coefficient was 0.97 for the FJS-12. The FJS-12 showed a significantly lower score than the WOMAC or JHEQ (p < 0.01). The FJS-12 was moderately correlated with the total WOMAC score (r = 0.522) and its subscale scores for "stiffness" (r = 0.401) and "function" (r = 0.539) and was weakly correlated with the score for "pain" (r = 0.289). The FJS-12 was favorably correlated with the total JHEQ score (r = 0.686) and its subscale scores (r = 0.530-0.643). The FJS-12 was correlated with and showed reliability similar to that of the JHEQ and WOMAC. The FJS-12, which is not affected by culture or lifestyle, may be useful in Japan.
Tan, Wah Ching; Krishnaswamy, Gita; Ong, Marianne M A; Lang, Niklaus P
To compare patient-reported outcome measures (PROMs) after different dental surgical procedures over a 1-week post-surgical period and in relation to duration of the surgery, and periosteal releasing incisions. To evaluate the prevalence of post-surgical complications. Four hundred and sixty-eight healthy dental patients requiring surgeries, such as crown lengthening (CL), open flap debridement (OFD) and implant installation (IMP) in the National Dental Centre, Singapore (2009-2011), were consecutively recruited. PROMs on bleeding, swelling, pain and bruising were obtained using Visual Analogue Scales (VAS) on days 0, 3, 5 and 7 post-operatively. On the day of surgery, the IMP procedure gave the lowest median VAS for all four PROM parameters. After a week, OFD still had a significantly higher VAS for swelling, pain and bruising. Patients who underwent procedures lasting more than 60 min. had higher VAS for all parameters except bleeding. After considering other important confounders, type of surgery procedure was no longer associated with the VAS score for any of the parameters. Time after surgery, male gender and shorter surgery duration reduced post-operative VAS for one or more of the parameters. Longer surgeon experience helps reduce VAS scores only for bleeding. Prevalence for tenderness to palpation was 11.6%, 8.9% and 12.2% for IMP, CL and OFD, respectively, 1-week post-operatively. Swelling and suppuration occurred rarely. The median VAS scores for all PROM parameters were generally low and reduced to near zero over a week following all three surgical procedures tested. Time after surgery and shorter surgery duration were associated with lower VAS scores in all the PROM parameters in this cohort of patients. Surgery type was not associated significantly with VAS after adjustment with other important confounders. Low prevalences of post-surgical complications were reported. © 2014 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.
Full Text Available Siva Narayanan,1 Victoria Guyatt,2 Alessandra Franceschetti,3 Emily L Hautamaki1 1Ipsos Healthcare, Columbia, MD, USA; 2Ipsos Ethnography Centre of Excellence (ECE, London, UK; 3Ipsos Healthcare, London, UK Objectives: To assess the impact of psoriasis on health-related quality of life (HRQoL.Methods: An ethnographic study of patients with moderate to severe psoriasis was conducted in the US, France, Germany, Italy, Spain, UK, Brazil, and Canada to explore patients' views on treatment and the impact of psoriasis on HRQoL. Anthropologists and ethnographers spent a minimum of 5.5 hours with each consented patient and filmed their behaviors in everyday situations. Visual data and notes were analyzed to identify HRQoL-related themes.Results: The study included 50 adult patients. Patients described their appearance with disgust and self-loathing. Frustration was expressed due to a perceived lack of control of their lives. Prior to initiation of biologic treatment, daily rituals absorbed a good part of their day, including applying creams, checking one's appearance, and covering the body. Due to a lack of cultural discourse and patient's difficulty in articulating the impact of psoriasis, partners and family did not know how to react nor did they realize the full extent of the problem, and many patients experienced perceived social discrimination due to psoriasis, leaving them with feelings of isolation. Patients established on biologic treatment noticed a significant physical improvement and regained confidence, but psychosocial impacts, including social isolation, remained.Conclusion: This ethnographic study vividly depicted the unarticulated and emotional impact of psoriasis on the everyday lives of patients and presents an effective method of assessing HRQoL in chronic diseases.Keywords: psoriasis, health-related quality of life, ethnography, patient reported outcomes, conceptual model
Angulo, J C; Brenes, F J; Lizarraga, I; Rejas, J; Trillo, S; Ochayta, D; Arumi, D
To explore the impact of urgency urinary incontinence (UUI) on well-being in non-institutionalized patients with overactive bladder (OAB) in a community sample. A cross-sectional web-based study was conducted in the general population, including males and females, >18 years of age. Patients with probable OAB were identified using a validated algorithm together with a score ≥8 on the OAB-V8 scale. Presence of coping behavior was considered determinant for the clinical diagnosis of OAB. Individual well-being was determined through a battery of patient-reported outcomes (PRO) measurements including assessment of health-related quality of life (EQ-5D), sleep disturbances (MOS Sleep), and life satisfaction (LISAT-8). Patients were grouped according to the number of daily UUI episodes (UUI severity): 0 (dry OAB),1, 2-3, or ≥4. Multivariate analysis to evaluate factors independently affecting quality of life was undertaken. A total of 396 patients (52.5% women, mean age: 55.3 [11.1] years, OAB-V8 mean score: 14.5 [7.9]) out of 2035 subjects participating from the general population met the criteria for OAB: 203 (51.3%) with 0episodes, 119 (30.1%) with 1, 52 (13.1%) with 2 or 3, and 22 (5.6%) with ≥4 episodes. A statistically significant linear adjusted association was found between number of UUI episodes and PRO scores. Participants with more episodes had poorer health profiles and self-evaluated quality of life, worse life satisfaction, and more sleep disturbances and fewer hours of sleep per night. Number of incontinence episodes was independent factor to affect quality of life using both LISAT-8 and MOS questionnaires. Severity of UUI was significantly associated with poorer individual well-being in subjects with OAB in a community sample in Spain. Copyright © 2015 AEU. Publicado por Elsevier España, S.L.U. All rights reserved.
Kelsall, David C; Arnold, Renée J G; Lionnet, Leonard
To assess patient-reported outcomes (PROs) in individuals with significant residual low-frequency hearing and severe-to-profound high-frequency sensorineural hearing loss (SNHL) who received the hybrid cochlear implant (CI). Prospective, multicenter, nonrandomized, single-arm repeated measures, single-subject design. Tertiary centers, ambulatory care. Fifty adults with severe-to-profound high-frequency SNHL and residual low-frequency hearing with aided word recognition scores between 10 and 60% in the ear to be implanted, and in the contralateral ear greater than or equal to implant ear less than or equal to 80%. Therapeutic; hybrid CI. Speech, spatial and qualities of hearing scale (SSQ), device use questionnaire (DUQ), University of Washington Clinical Assessment of Music Perception (UW-CAMP) assessed preoperatively and after 6 and 12 (SSQ and DUQ only) months of hybrid CI use. Significant improvements in mean SSQ ratings were demonstrated at 6 and 12 months postactivation overall and for domains related to speech hearing, spatial hearing, and sound quality. Significant improvement was also found for overall satisfaction on the DUQ and across a number of specific listening situations in addition to aspects related to social engagement. UW-CAMP pitch discrimination and melody and timbre recognition abilities were not compromised postoperatively, allowing hybrid subjects to maintain superior music perception abilities than typically observed with standard CIs. Patients who received the hybrid CI demonstrated significant PRO benefits on the SSQ and the DUQ after 6 and 12 months of CI use. In addition, given the opportunity to maintain useful low-frequency acoustic hearing, patients retained music listening abilities, as assessed by the UW-CAMP.
Boye Kristina S
Full Text Available Abstract Background Prior to using a generic patient-reported outcome measure (PRO, the measure should be validated within the target population. The purpose of the current study was to validate two generic measures in patients with type 2 diabetes. Methods Patients with type 2 diabetes in Scotland and England completed two generic measures: EQ-5D and Psychological General Well-Being Index (PGWB. Two diabetes-specific measures were administered: ADS and DSC-R. Analyses assessed reliability and validity. Results There were 130 participants (53 Scotland; 77 England; 64% male; mean age = 55.7 years. Responses on the EQ-5D and PGWB reflected moderate impairment consistent with previous diabetes samples: mean EQ-5D Index score, 0.75; EQ-5D VAS, 68.8; PGWB global score, 67.9. All scales of the PGWB demonstrated good internal consistency reliability (Cronbach's alpha = 0.77 to 0.97. The EQ-5D and PGWB demonstrated convergent validity through significant correlations with the ADS (r = 0.48 to 0.61, DSC-R scales (r = 0.33 to 0.81 except ophthalmology subscale, and Body Mass Index (r = 0.15 to 0.38. The EQ-5D and PGWB discriminated between groups of patients known to differ in diabetes-related characteristics (e.g., history of hypoglycemia. Conclusion Results support the use of the EQ-5D and PGWB among patients with type 2 diabetes, possibly in combination with condition-specific measures.
Hagell, Peter; Reimer, Jan; Nyberg, Per
Patient-reported health status questionnaires intend to assess illness and therapy from the patients' perspective. To provide fair and valid assessments, they should be equally relevant to major subsets of respondents. Furthermore, disease-specific measures are assumed to be perceived as more relevant than generic ones. This study assessed these assumptions among people with Parkinson's disease. Cross-sectional data from 202 people with Parkinson's disease (54% men; mean age, 70) were analyzed regarding patient-rated relevance and predictors of patient-rated poor relevance of two generic [the 36-item Short Form Health Survey (SF-36) and Nottingham Health Profile (NHP)] and one disease-specific [Parkinson's Disease Questionnaire (PDQ-39)] health status questionnaire. There were no differences in relevance ratings across the questionnaires. Poorer overall quality of life [odds ratio (OR), 2.459] and mental health (OR, 1.023) were associated with poorer patient-rated relevance of the SF-36, and higher age was associated with poorer patient-rated relevance of the PDQ-39 (OR, 1.040). No significant predictors were found for the NHP. The PDQ-39 failed to meet the assumption that disease-specific scales are more relevant than generic ones. Nevertheless, the most important implication of this study is an ethical one. Because the relevance of the SF-36 and PDQ-39 is perceived as poorer by those who fare least well and by older people, these scales may not reflect the perspectives of these groups. This challenges bioethical principles and threatens scientific validity. Perceived relevance of patient-centered outcomes needs to be considered, or the voice of vulnerable groups may be silenced, fair inferences prohibited, and opportunities for improved care lost.
Raffaele Pezzilli; Davide Campana; Antonio M Morselli-Labate; Maria C Fabbri; Emilio Brocchi; Paola Tomassetti
AIM: To assess the patient-reported outcomes (PROs) of pancreatic neuroendocrine tumor (PNET) patients. METHODS: Fifty-one consecutive patients (21 male,30 female, 61.0 ± 10.3 years) with proven PNETs were studied. An SF-12 questionnaire capable of exploring the physical (PCS) and mental (MCS) aspects of daily life was used. Four questionnaires were also used [12 items General Health Questionnaire (GHQ-12) for nonpsychotic psychiatric disorders, State Trait Anxiety Inventory (STAI) Y-1 and Y-2 for anxiety and BDI-Ⅱ for depressive symptoms] to explore the psychological aspects of the disease. Forty-four sex- and age-matched Italian normative subjects were included and evaluated using the SF-12, STAI Y-1 and Y-2 questionnaires.RESULTS: Seven patients refused to participate to the study; they were clinically similar to the 44 participants who agreed to complete the questionnaires. PNET patients had a PCS score (44.7 ± 11.0) were not significantly different from the norms (46.1 ± 9.9, P = 0.610), whereas the MCS score was significantly lower in patients (42.4 ± 13.0) as compared to the norms (48.2 ± 9.8, P = 0.036). GHQ-12 identified 11 patients (25.0%) as having non-psychotic psychiatric disorders. The STAI scores were similar in the patients and in the normative population. Finally, BDI-Ⅱ identified eight patients (18.2%) with moderate depression and 9 (20.5%) with mild depression whereas 27 patients (61.4%) had no depression. CONCLUSION: The PNET patients had a good physical but an impaired mental component of their quality of life; in addition, mild or moderate depressive symptoms are present in about 40% of PNET patients.
Full Text Available Abstract Background Several small, uncontrolled studies have found improvements in self-care behaviors and reductions in clinical risk in persons with type 2 diabetes who received care from licensed naturopathic physicians. To extend these findings and determine the feasibility and promise of a randomized clinical trial, we conducted a prospective study to measure the effects of adjunctive naturopathic care (ANC in primary care patients with inadequately controlled type 2 diabetes. Methods Forty patients with type 2 diabetes were invited from a large integrated health care system to receive up to eight ANC visits for up to one year. Participants were required to have hemoglobin A1c (HbA1c values between 7.5-9.5 % and at least one additional cardiovascular risk factor (i.e., hypertension, hyperlipidemia or overweight. Standardized instruments were administered by telephone to collect outcome data on self-care, self-efficacy, diabetes problem areas, perceived stress, motivation, and mood. Changes from baseline scores were calculated at 6- and 12-months after entry into the study. Six and 12-month changes in clinical risk factors (i.e., HbA1c, lipid and blood pressure were calculated for the ANC cohort, and compared to changes in a cohort of 329 eligible, non-participating patients constructed using electronic medical records data. Between-cohort comparisons were adjusted for age, gender, baseline HbA1c, and diabetes medications. Six months was pre-specified as the primary endpoint for outcome assessment. Results Participants made 3.9 ANC visits on average during the year, 78 % of which occurred within six months of entry into the study. At 6-months, significant improvements were found in most patient-reported measures, including glucose testing (P = 0.001, diet (P = 0.001, physical activity (P = 0.02, mood (P = 0.001, self-efficacy (P = 0.0001 and motivation to change lifestyle (P = 0.003. Improvements in glucose testing, mood, self-efficacy and
Full Text Available David Price,1,2 Anders Østrem,3 Mike Thomas,4 Tobias Welte5 1Department of Primary Care Respiratory Medicine, Division of Applied Health Sciences, University of Aberdeen, Aberdeen, UK; 2Observational and Pragmatic Research Institute, Singapore; 3Gransdalen Legesenter, Oslo, Norway; 4Department of Primary Care Research, University of Southampton, Southampton, UK; 5Department of Pulmonary Medicine, Hannover Medical School, Hannover, Germany Abstract: Several fixed-dose combinations (FDCs of long-acting bronchodilators (a long-acting muscarinic antagonist [LAMA] plus a long-acting β2-agonist [LABA] are available for the treatment of COPD. Studies of these FDCs have demonstrated substantial improvements in lung function (forced expiratory volume in 1 second in comparison with their respective constituent monocomponents. Improvements in patient-reported outcomes (PROs, such as symptoms and health status, as well as exacerbation rates, have been reported compared with a LABA or LAMA alone, but results are less consistent. The inconsistencies may in part be owing to differences in study design, methods used to assess study end points, and patient populations. Nevertheless, these observations tend to support an association between improvements in forced expiratory volume in 1 second and improvements in symptom-based outcomes. In order to assess the effects of FDCs on PROs and evaluate relationships between PROs and changes in lung function, we performed a systematic literature search of publications reporting randomized controlled trials of FDCs. Results of this literature search were independently assessed by two reviewers, with a third reviewer resolving any conflicting results. In total, 22 Phase III randomized controlled trials of FDC bronchodilators in COPD were identified, with an additional study including a post-literature search (ten for indacaterol–glycopyrronium once daily, eight for umeclidinium–vilanterol once daily, three for
Full Text Available Paul W Jones,1,2 Stephen Rennard,3,4 Maggie Tabberer,5 John H Riley,2 Mitra Vahdati-Bolouri,2 Neil C Barnes2,6 1Institute for Infection and Immunity, University of London, London, 2Global Respiratory Franchise, GlaxoSmithKline, Uxbridge, UK; 3Division of Pulmonary, Critical Care, Sleep and Allergy, Nebraska Medical Center, Omaha, NE, USA; 4Clinical Discovery Unit, AstraZeneca, Cambridge, 5Global R&D, GlaxoSmithKline, Uxbridge, 6William Harvey Institute, Bart’s and the London School of Medicine and Dentistry, London, UK Abstract: One of the challenges faced by the practising physician is the interpretation of patient-reported outcomes (PROs in clinical trials and the relevance of such data to their patients. This is especially true when caring for patients with progressive diseases such as COPD. In an attempt to incorporate the patient perspective, many clinical trials now include assessments of PROs. These are formalized methods of capturing patient-centered information. Given the importance of PROs in evaluating the potential utility of an intervention for a patient with COPD, it is important that physicians are able to critically interpret (and critique the results derived from them. Therefore, in this paper, a series of questions is posed for the practising physician to consider when reviewing the treatment effectiveness as assessed by PROs. The focus is on the St George’s Respiratory Questionnaire for worked examples, but the principles apply equally to other symptom-based questionnaires. A number of different ways of presenting PRO data are discussed, including the concept of the minimum clinically important difference, whether there is a ceiling effect to PRO results, and the strengths and weaknesses of responder analyses. Using a worked example, the value of including a placebo arm in a study is illustrated, and the influence of the study on PRO results is considered, in terms of the design, patient withdrawal, and the selection of
Holloway, Laura; Humphrey, Louise; Heron, Louise; Pilling, Claire; Kitchen, Helen; Højbjerre, Lise; Strandberg-Larsen, Martin; Hansen, Brian Bekker
Background Despite overall progress in treatment of autoimmune diseases, patients with systemic lupus erythematosus (SLE) experience many inflammatory symptoms representing an unmet medical need. This study aimed to create a conceptual model of the humanistic and economic burden of SLE, and review the patient-reported outcomes (PROs) used to measure such concepts in SLE clinical trials. Methods A conceptual model for SLE was developed from structured review of published articles from 2007 to ...
Full Text Available Assessment of patient-reported outcomes (PROs provides valuable information to inform patient-centered care, but may also reveal 'PRO alerts': psychological distress or physical symptoms that may require an immediate response. Ad-hoc management of PRO alerts in clinical trials may result in suboptimal patient care or potentially bias trial results. To gain greater understanding of current practice in PRO alert management we conducted a national survey of personnel involved in clinical trials with a PRO endpoint.We conducted a national cross-sectional survey of 767 UK-based research nurses, data managers/coordinators, trial managers and chief/principal investigators involved in clinical trials using PROs. Respondents were self-selected volunteers from a non-randomised sample of eligible individuals recruited via 55 UK Clinical Research Collaboration Registered Clinical Trials Units and 19 Comprehensive Local Research Networks. Questions centred on the proportion of trial personnel encountering alerts, how staff responded to PRO alerts and whether current guidance was deemed sufficient to support research personnel. We undertook descriptive analyses of the quantitative data and directed thematic analysis of free-text comments. 20% of research nurses did not view completed PRO questionnaires and were not in a position to discover alerts, 39-50% of the remaining respondent group participants reported encountering PRO alerts. Of these, 83% of research nurses and 54% of data managers/trial coordinators reported taking action to assist the trial participant, but less than half were able to record the intervention in the trial documentation. Research personnel reported current PRO alert guidance/training was insufficient.Research personnel are intermittently exposed to PRO alerts. Some intervene to help trial participants, but are not able to record this intervention in the trial documentation, risking co-intervention bias. Other staff do not check PRO
Koneru, Harsha; Cyr, Robyn; Feng, Li Rebekah; Bae, Edward; Danner, Malika T; Ayoob, Marilyn; Yung, Thomas M; Lei, Siyuan; Collins, Brian T; Saligan, Leorey; Simeng, Suy; Kumar, Deepak; Collins, Sean P
The relationship between obesity (Body Mass Index ->30 kg/m(2)) and quality of life (QoL) following prostate cancer (PCa) radiation therapy (RT) is unknown. Excess abdominal fat may compromise the precise delivery of radiation, putting surrounding organs at risk for greater radiation exposure. Stereotactic body radiation therapy (SBRT) utilizes a real-time tracking system that provides updated prostate position information and allows for correction of the therapeutic beam during treatment with high accuracy. In this study, we evaluate the impact of obesity on patient reported outcomes following SBRT for prostate cancer. Between February 2008 and April 2012, 88 obese and 178 non-obese patients with PCa were treated with SBRT at Georgetown University Hospital, Washington, DC. Health-related quality of life (HRQol) was assessed via the expanded prostate cancer index composite (EPIC)-26 at baseline, 6, 12, 18, and 24 months after 5-fraction delivery of 35-36.25 Gy with the CyberKnife. Patients who received androgen deprivation therapy (ADT) were excluded from this analysis due to its known negative impact on HRQoL. Pretreatment characteristics of obese and non-obese patient groups were similar except that obese patients had lower total testosterone levels. Urinary and bowel function and bother scores between the two patient cohorts were comparable at baseline and subsequent follow-ups. Sexual function and bother were also similar at baseline between both groups. Bother was defined by displeasure patients may experience from functional decline. At 24 months post-SBRT, obese men experienced borderline clinically significant decrease in sexual function and greater sexual bother compared to non-obese patients. Fatigue was significantly higher in obese patients compared to non-obese patients at 18 months post-SBRT. Prostate SBRT affects obese and non-obese patients similarly in total HRQoL scores and majority of its domains. Obesity has been associated with cancer
Luna, I E; Kehlet, H; Peterson, Barry
). Secondary measures were correlations to objectively assessed change in physical performance (paced-walk, chair-stand, stair-climb tests) at day 14 (THA) or 21 (TKA) and actual physical activity (actigraphy) measured at day 12 and 13 (THA) or 19 and 20 (TKA). RESULTS: Patients reported improved physical...
Patient-reported outcome measures in arthroplasty registries Report of the Patient-Reported Outcome Measures Working Group of the International Society of Arthroplasty Registries Part II. Recommendations for selection, administration, and analysis
Rolfson, Ola; Bohm, Eric; Franklin, Patricia;
- The International Society of Arthroplasty Registries (ISAR) Patient-Reported Outcome Measures (PROMs) Working Group have evaluated and recommended best practices in the selection, administration, and interpretation of PROMs for hip and knee arthroplasty registries. The 2 generic PROMs in common...... of a 1-item pain question ("During the past 4 weeks, how would you describe the pain you usually have in your [right/left] [hip/knee]?"; response: none, very mild, mild, moderate, or severe) and a single-item satisfaction outcome ("How satisfied are you with your [right/left] [hip/knee] replacement...
Kianifard Farid; Raut Monika; Mathias Susan D; Potter Lori P; Tavakkol Amir
Abstract Background This research was conducted to confirm the validity and reliability and to assess the responsiveness and clinical meaningfulness of the OnyCOE-t™, a questionnaire specifically designed to measure patient-reported outcomes (PRO) associated with toenail onychomycosis. Methods 504 patients with toenail onychomycosis randomized to receive 12 weeks of terbinafine 250 mg/day with or without target toenail debridement in the IRON-CLAD® trial completed the OnyCOE-t™ at baseline, w...
Wæhrens, E E; Amris, K; Bartels, E M
OBJECTIVES: To compare data based on computerized and paper versions of health status questionnaires (HSQs) for sampling patient-reported outcomes (PROs) in patients with fibromyalgia (FM). In addition, to examine associations between patient characteristics (age, education, computer experience......) and differences between versions. Finally, to evaluate the acceptability of computer-based questionnaires among patients with FM. METHOD: The study population comprised female patients diagnosed with FM. All patients completed six HSQs: the Fibromyalgia Impact Questionnaire (FIQ), the Major Depression Inventory...
Pedersen, Susanne Møller; Versteeg, Henneke; Nielsen, Jens C.
The Medtronic Sprint Fidelis Lead Advisory Notification has no adverse impact on patient reported outcomes in Danish implantable cardioverter defibrillator patients.......The Medtronic Sprint Fidelis Lead Advisory Notification has no adverse impact on patient reported outcomes in Danish implantable cardioverter defibrillator patients....
Pedersen, Susanne Møller; Versteeg, Henneke; Nielsen, Jens C.
The Medtronic Sprint Fidelis Lead Advisory Notification Has No Adverse Impact on Patient Reported Outcomes in Danish Implantable Cardioverter Defibrillator Patients.......The Medtronic Sprint Fidelis Lead Advisory Notification Has No Adverse Impact on Patient Reported Outcomes in Danish Implantable Cardioverter Defibrillator Patients....
Revicki, Dennis A; Gnanasakthy, Ari; Weinfurt, Kevin
The Food and Drug Administration (FDA) and European Medicines Agency (EMEA) are willing to consider including information on patient reported outcomes (PROs) in product labeling and advertising. Pharmaceutical industry researchers must provide sufficient evidence supporting PRO benefit before an approval may be granted. This report describes the purpose and content of a PRO Evidence Dossier, which consists of important information supporting PRO claims. The dossier should be completed by pharmaceutical industry or other researchers to document the planning of the PRO assessment strategy, psychometric evidence, desired target labeling statements, and the clinical trial evidence of PRO benefits. The systematic reporting and documentation of information on the rationale for including PROs, rationale for the selection of specific PRO instruments, evidence on the psychometric qualities of the PRO measures, and guidelines for interpreting PRO findings will facilitate achieving a PRO labeling or promotional claim. Combining all the relevant information into a single document will facilitate the review and evaluation process for clinical and regulatory reviewers. The PRO Evidence Dossier may also be helpful to industry and academic researchers in identifying further information that will need to be developed to support the clinical development program and the PRO endpoints.
Ingegnoli, Francesca; Carmona, Loreto; Castrejon, Isabel
The EULAR Outcome Measures Library (OML) is a freely available database of validated patient-reported outcomes (PROs). The aim of this study was to provide a comprehensive review of validated PROs specifically developed for systemic sclerosis (SSc) to feed the EULAR OML. A sensitive search was developed in Medline and Embase to identify all validation studies, cohort studies, reviews, or meta-analyses in which the objective were the development or validation of specific PROs evaluating organ involvement, disease activity or damage in SSc. A reviewer screened title and abstracts, selected the studies, and collected data concerning validation using ad hoc forms based on the COSMIN checklist. From 13,140 articles captured, 74 met the predefined criteria. After excluding two instruments as they were unavailable in English the selected 23 studies provided information on seven SSc-specific PROs on different SSc domains: burden of illness (symptom burden index), functional status (Scleroderma Assessment Questionnaire), functional ability (scleroderma Functional Score), Raynaud's phenomenon (Raynaud's condition score), mouth involvement (Mouth Handicap in SSc), gastro-intestinal involvement (University of California Los Angeles-Scleroderma Clinical Trial Consortium Gastro-Intestinal tract 2.0), and skin involvement (skin self-assessment). Each of them is partially validated and has different psychometric requirements. Seven SSc-specific PROs have a minimum validation and were included in the EULAR OML. Further development in the area of disease-specific PROs in SSc is warranted. Copyright © 2017 Elsevier Inc. All rights reserved.
Nikolova, Silviya; Harrison, Mark; Sutton, Matt
Reducing waiting times has been a major focus of the English National Health Service for many years, but little is known about the impact on health outcomes. The collection of data on patient-reported outcome measures for all patients undergoing four large-volume procedures facilitates analysis of the impact of waiting times on patient outcomes. The availability of patient-reported outcome measures before and after surgery allows us to estimate the impact of waiting times on the effectiveness of treatment, controlling for pre-surgery health and the endogeneity of waiting times caused by prioritisation with respect to pre-intervention health. We find that waiting time has a negative and statistically significant impact on the health gain from hip and knee replacement surgery and no impact on the effectiveness of varicose vein and hernia surgery. The magnitude of this effect at patient level is small, 0.1% of the outcome measure range for each additional week of waiting. However, the value of this effect is substantially larger than existing estimates of the disutility experienced during the waiting period. The health losses associated with an additional week of waiting for annual populations of hip and knee replacement patients are worth £11.1m and £11.5m, respectively. Copyright © 2015 John Wiley & Sons, Ltd.
Straatman, J; Joosten, P J M; Terwee, C B; Cuesta, M A; Jansma, E P; van der Peet, D L
Esophageal cancer is currently the eighth most common cancer worldwide. Improvements in operative techniques and neoadjuvant therapies have led to improved outcomes. Resection of the esophagus carries a high risk of severe complications and has a negative impact on health-related quality of life (QOL). The aim of this study was to assess which patient-reported outcome measures (PROMs) are used to measure QOL after esophagectomy for cancer. A comprehensive search of original articles was conducted investigating QOL after surgery for esophageal carcinoma. Two authors independently selected relevant articles, conducted clinical appraisal, and extracted data (PJ and JS). Out of 5893 articles, 58 studies were included, consisting of 41 prospective and 17 retrospective cohort studies, including a total of 6964 patients. These studies included 11 different PROMs. The existing PROMs could be divided into generic, symptom-specific, and disease-specific questionnaires. The European Organisation for Research and Treatment of Cancer (EORTC) QOL Questionnaire Core 30 (QLQ C-30) along with the EORTC QLQ-OESophagus module OES18 was the most widely used; in 42 and 32 studies, respectively. The EORTC and the Functional Assessment of Cancer Therapy (FACT) questionnaires use an oncological module and an organ-specific module. One validation study was available, which compared the FACT and EORTC, showing moderate to poor correlation between the questionnaires. A great variety of PROMs are being used in the measurement of QOL after surgery for esophageal cancer. A questionnaire with a general module along with a disease-specific module for assessment of QOL of different treatment modalities seem to be the most desirable, such as the EORTC and the FACT with their specific modules (EORTC QLQ-OES18 and FACT-E). Both are developed in different treatment modalities, such as in surgical patients. With regard to reproducibility of current results, the EORTC is recommended. © 2015
Full Text Available Leticia Delgado-Herrera,1 Kathryn Lasch,2 Ana Popielnicki,3 Akito Nishida,4 Rob Arbuckle,5 Benjamin Banderas,6 Susan Zentner,1 Ingrid Gagainis,1 Bernhardt Zeiher1 1Astellas Pharma Global Development, Northbrook, IL, 2Pharmerit International, Newton, MA, USA; 3TransPerfect, Linguistic Validation Group, Boston, MA, USA; 4Development Project Management, Astellas Pharma Inc, Tokyo, Japan; 5Patient-Centered Outcomes Adelphi Values, Bollington, UK; 6Patient-Centered Outcomes Adelphi Values, Boston, MA, USA Background and objective: Following a 2009 US Food and Drug Administration guidance, a new patient-reported outcome (PRO instrument was developed to support end points in multinational clinical trials assessing irritable bowel syndrome with diarrhea (IBS-D symptom severity. Our objective was to assess the translatability of the IBS-D PRO instrument into ten languages, and subsequently perform a cultural adaptation/linguistic validation of the questionnaire into Japanese and US Spanish. Materials and methods: Translatability assessments of the US English version of the IBS-D PRO were performed by experienced PRO translators who were native speakers of each target language and currently residing in target-language countries. Languages were Chinese (People’s Republic of China, Dutch (the Netherlands, French (Belgium, German (Germany, Japanese (Japan, Polish (Poland, Portuguese (Brazil, Russian (Russia, Spanish (Mexico, and Spanish (US. The project team assessed the instrument to identify potential linguistic and/or cultural adaptation issues. After the issues identified were resolved, the instrument was translated into Spanish (US and Japanese through a process of two forward translations, one reconciled translation, and one backward translation. The project team reviewed the translated versions before the instruments were evaluated by cognitive debriefing interviews with samples of five Spanish (US and five Japanese IBS-D patients. Results
Rodríguez-Rivera, Diana V; Rodríguez-Navedo, Yerania; Nieves-Plaza, Mariely; Vilá, Luis M
Objective: To determine patient-reported outcomes measures in indigent patients with systemic lupus erythematosus receiving their healthcare through the Puerto Rico government managed care system and compare these measures with non-indigent patients treated in a private fee-for-service setting. Methods: A cross-sectional study was conducted in a cohort of 98 Puerto Ricans with systemic lupus erythematosus. Patients from the public group (n = 40) were treated in a university-based specialized systemic lupus erythematosus clinic and the private group (n = 58) in a community-based rheumatology practice. Demographic and clinical features and patient-reported outcomes measures per LupusPRO instrument were determined. LupusPRO captures quality-of-life measures in 12 domains. Differences among study groups were examined using chi-square, Fisher’s exact, t-tests, and the Wilcoxon signed-rank test. Results: The mean (standard deviation) age of the study population was 44.9 (12.0) years; 94 (95.9%) were women. Patients in the public setting were younger and were more likely to have renal disease and elevated anti-double-stranded DNA antibodies, and being treated with azathioprine and cyclophosphamide. Patients from the public sector were more likely to have better quality-of-life measures in the LupusPRO domains of pain/vitality and coping. No significant differences were observed for the domains of lupus symptoms, physical health, emotional health, body image, cognition, procreation, lupus medications, desires/goals, social support, and satisfaction with medical care. Conclusion: Despite having a lower socioeconomic status and worse clinical status, systemic lupus erythematosus patients from the public sector had equal or better patient-reported outcomes measures than those treated in the private setting. This favorable outcome may be associated with the comprehensive healthcare received by these patients in a specialized lupus clinic.
Full Text Available Background: “Patient-Reported Outcome” (PRO is used as an umbrella term for different concepts for measuring subjectively perceived health status e. g. as treatment effects. Their common characteristic is, that the appraisal of the health status is reported by the patient himself. In order to describe the informative value of PRO in Health Technology Assessment (HTA first an overview of concepts, classifications and methods of measurement is given. The overview is complemented by an empirical analysis of clinical trials and HTA-reports on rheumatoid arthritis and breast cancer in order to report on type, frequency and consequences of PRO used in these documents. Methods: For both issues systematic reviews of the literature have been performed. The search for methodological literature covers the publication period from 1990 to 2009, the search for clinical trials of rheumatoid arthritis and breast cancer covers the period 2005 to 2009. Both searches were performed in the medical databases of the German Institute of Medical Documentation and Information (DIMDI. The search for HTA-reports and methodological papers of HTA-agencies was performed in the CRD-Databases (CRD = Centre for Reviews and Dissemination and by handsearching the websites of INAHTA member agencies (INAHTA = International Network of Agencies for Health Technology Assessment. For all issues specific inclusion and exclusion criteria were defined. The methodological quality of randomized controlled trials (RCT was assessed by a modified version of the Cochrane Risk of Bias Tool. For the methodological part information extraction from the literature is structured by the report’s chapters, for the empirical part data extraction sheets were constructed. All information is summarized in a qualitative manner. Results: Concerning the methodological issues the literature search retrieved 158 documents (87 documents related to definition or classification, 125 documents related to
Anthony, Chris A; Glass, Natalie; Hancock, Kyle; Bollier, Matt; Hettrich, Carolyn M; Wolf, Brian R
To evaluate the Patient-Reported Outcomes Measurement Information System upper extremity item bank (PROMIS UE) and physical function computerized adaptive test (PROMIS PF CAT) in patients with rotator cuff (RC) pathology at their preoperative clinic visit. Patient data were collected from January 2015 to September 2015. Patients with a preoperative diagnosis of RC pathology were prospectively enrolled at the time of their surgical indication for RC repair. Each patient was asked to fill out the Western Ontario Rotator Cuff Index (WORC), American Shoulder and Elbow Surgeons Shoulder Assessment Form, Marx Shoulder Activity Scale, Short Form 36 Health Survey Physical Function and General Health (SF-36 PF and GH), EuroQol-5 Dimension (EQ-5D), PROMIS PF CAT, and PROMIS UE. Correlation was defined as excellent (>0.7), excellent-good (0.61-0.7), good (0.4-0.6), and poor (0.2-0.3). Patient data were collected from January 2015 to September 2015. No patients were excluded from participation in the study. In 82 patients with preoperative RC pathology, the PROMIS UE showed excellent correlation with American Shoulder and Elbow Surgeons Shoulder Assessment Form (r = 0.77, P ceiling effects using the PROMIS UE item bank or PROMIS PF CAT. We report that in a patient population with preoperative RC pathology, the PROMIS UE and PROMIS CAT are valid patient-reported outcome alternatives that have high correlation with traditional shoulder and upper extremity patient-reported outcomes. We find a decreased question burden using the PROMIS PF CAT. We find no significant floor or ceiling effects present in the PROMIS UE or PROMIS PF CAT. Level II, prospective diagnostic study. Copyright © 2017 Arthroscopy Association of North America. Published by Elsevier Inc. All rights reserved.
Højmark, Karen; Støttrup, C; Carreon, L
. A structured phone interview was performed on these 57 non-responders. Non-responders were in general, a decade younger than responders, a greater proportion were males and smokers. Apart from EQ-5D, there was no difference in patient satisfaction, improvement in back pain or leg pain between the responders...... and non-responders. CONCLUSIONS: Missing data from 12 % of patients do not seem to bias conclusions that can be drawn from the DaneSpine database at the Center for Spine Surgery and Research at Lillebaelt Hospital.......PURPOSE: The purpose of this study is to determine if there are any demographic and reporting differences between patients who respond and those who refuse to respond to postal questionnaires from the Danish national spine database, DaneSpine. METHODS: DaneSpine collects patient-reported data...
Ageberg, Eva; Forssblad, Magnus; Herbertsson, Pär
BACKGROUND: Female gender is a risk factor for sustaining anterior cruciate ligament (ACL) injury. However, little is known about possible sex differences in patients with ACL injury/reconstruction. PURPOSE: To study sex differences in patient-reported outcomes before and at 1 and 2 years after ACL...... reconstruction and to present reference values. STUDY DESIGN: Cohort study; Level of evidence, 2. METHODS: Between 2005 and 2008, 10 164 patients (mean age, 27 years; SD, 9.8; 42% females) with primary ACL reconstruction were registered in the Swedish national knee ligament register. There were 4438 (44...
Full Text Available Abstract Background Patient-reported Outcomes (PROs capturing e.g., quality of life, fatigue, depression, medication side-effects or disease symptoms, have become important outcome parameters in medical research and daily clinical practice. Electronic PRO data capture (ePRO with software packages to administer questionnaires, storing data, and presenting results has facilitated PRO assessment in hospital settings. Compared to conventional paper-pencil versions of PRO instruments, ePRO is more economical with regard to staff resources and time, and allows immediate presentation of results to the medical staff. The objective of our project was to develop software (CHES – Computer-based Health Evaluation System for ePRO in hospital settings and at home with a special focus on the presentation of individual patient’s results. Methods Following the Extreme Programming development approach architecture was not fixed up-front, but was done in close, continuous collaboration with software end users (medical staff, researchers and patients to meet their specific demands. Developed features include sophisticated, longitudinal charts linking patients’ PRO data to clinical characteristics and to PRO scores from reference populations, a web-interface for questionnaire administration, and a tool for convenient creating and editing of questionnaires. Results By 2012 CHES has been implemented at various institutions in Austria, Germany, Switzerland, and the UK and about 5000 patients participated in ePRO (with around 15000 assessments in total. Data entry is done by the patients themselves via tablet PCs with a study nurse or an intern approaching patients and supervising questionnaire completion. Discussion During the last decade several software packages for ePRO have emerged for different purposes. Whereas commercial products are available primarily for ePRO in clinical trials, academic projects have focused on data collection and presentation in daily
L. Haverman (Lotte); M.A. Grootenhuis (Martha); H. Raat (Hein); M.A.J. van Rossum (Marion); E. van Dulmen-den Broeder (E.); K. Hoppenbrouwers (Karel); H. Correia (Helena); M. Cella (Massimo); L.D. Roorda (Lieuwe); C.B. Terwee (Caroline)
textabstractPurpose: The Patient-Reported Outcomes Measurement Information System (PROMIS®) is a new, state-of-the-art assessment system for measuring patient-reported health and well-being of adults and children. It has the potential to be more valid, reliable, and responsive than existing PROMs. T
Schwartz, Carolyn E; Patrick, Donald L
When planning a comparative effectiveness study comparing disease-modifying treatments, competing demands influence choice of outcomes. Current practice emphasizes parsimony, although understanding multidimensional treatment impact can help to personalize medical decision-making. We discuss both sides of this 'tug of war'. We discuss the assumptions, advantages and drawbacks of composite scores and multidimensional outcomes. We describe possible solutions to the multiple comparison problem, including conceptual hierarchy distinctions, statistical approaches, 'real-world' benchmarks of effectiveness and subgroup analysis. We conclude that comparative effectiveness research should consider multiple outcome dimensions and compare different approaches that fit the individual context of study objectives.
Strand, Vibeke; Reaney, Matthew; Chen, Chieh-I; Proudfoot, Clare W J; Guillonneau, Sophie; Bauer, Deborah; Mangan, Erin; Graham, Neil M H; van Hoogstraten, Hubert; Lin, Yong; Pacheco-Tena, César; Fleischmann, Roy
Objective To evaluate effects of the anti-interleukin-6 receptor monoclonal antibody sarilumab administered with conventional synthetic disease-modifying antirheumatic drugs (csDMARDs) on patient-reported outcomes (PROs) in the TARGET trial in patients with rheumatoid arthritis (RA) with inadequate response or intolerance to tumour necrosis factor inhibitors (TNF-IR). Methods 546 patients (81.9% female, mean age 52.9 years) were randomised to placebo, sarilumab 150 or 200 mg subcutaneously every 2 weeks + csDMARDs. PROs included patient global assessment (PtGA); pain and morning stiffness visual analogue scales; Health Assessment Questionnaire Disability Index (HAQ-DI); Short Form-36 Health Survey (SF-36); FACIT-Fatigue (FACIT-F); Work Productivity Survey-Rheumatoid Arthritis (WPS-RA) and Rheumatoid Arthritis Impact of Disease (RAID). Changes from baseline at weeks 12 and 24 were analysed using a mixed model for repeated measures; post hoc analyses included percentages of patients reporting improvements ≥ minimum clinically important differences (MCID) and scores ≥ normative values. Results Sarilumab + csDMARDs doses resulted in improvements from baseline at week 12 vs placebo + csDMARDs in PtGA, pain, HAQ-DI, SF-36 and FACIT-F that were maintained at week 24. Sarilumab improved morning stiffness and reduced the impact of RA on work, family, social/leisure activities participation (WPS-RA) and on patients' lives (RAID). Percentages of patients reporting improvements ≥MCID and ≥ normative scores were greater with sarilumab than placebo. Conclusions In patients with TNF-IR RA, 150 and 200 mg sarilumab + csDMARDs resulted in clinically meaningful patient-reported benefits on pain, fatigue, function, participation and health status at 12 and 24 weeks that exceeded placebo + csDMARDs, and were consistent with the clinical profile previously reported. Trial registration number NCT01709578; Results. PMID:28326189
Horn, Susan D; Gassaway, Julie
Comparative effectiveness research analyzes groups of patients and looks for associations between medical treatments and patient outcomes. To make meaningful comparisons of medical interventions, one must consider clinical heterogeneity of patient populations, intervention combinations, and outcomes. To explain how practice-based evidence (PBE) study methodology measures and controls for heterogeneity of patients, treatments, and outcomes seen in real-world clinical settings. Overview of PBE methodology. PBE study designs address comparative effectiveness by creating a comprehensive set of patient, treatment, and outcome variables, and analyzing them to identify treatments associated with better outcomes for specific types of patients. PBE studies are an alternative to randomized controlled trials, well suited to determine what works best for specific patient types, and provide clinicians with a rational basis for treatment recommendations for individual patients. They provide a holistic picture of patients, treatments, and outcomes, with no preset limits to the number of variables that can be included. Such an approach is needed for high quality comparative effectiveness research.
Brochmann, Nana; Zwisler, Ann-Dorthe; Kjerholt, Mette;
PURPOSE: An Internet-based tool for reporting and analysing patient-reported outcomes (PROs) has been developed. The tool enables merging PROs with blood test results and allows for computation of treatment responses. Data may be visualized by graphical analysis and may be exported for downstream...... statistical processing. The aim of this study was to investigate, whether patients with myeloproliferative neoplasms (MPNs) were willing and able to use the tool and fill out questionnaires regularly. METHODS: Participants were recruited from the outpatient clinic at the Department of Haematology, Roskilde...
Gundersen, Guri H; Norekvål, Tone M; Graven, Torbjørn; Haug, Hilde H; Skjetne, Kyrre; Kleinau, Jens O; Gustad, Lise T; Dalen, Håvard
We aimed to study whether patient-reported outcomes, measured by quality of life (QoL) and functional class, are sensitive to pleural effusion (PLE) in patients with heart failure (HF), and to study changes in QoL and functional class during follow-up of PLE. A cohort of 62 patients from an outpatient HF clinic was included. The amount of PLE was quantified using a pocket-sized ultrasound imaging device. Self-reports of QoL and functional class were collected using the Minnesota Living with Heart Failure Questionnaire (MLHFQ) and the New York Heart Association (NYHA) functional classification. At baseline, 26 (42%) patients had PLE of which 19 (31%) patients had moderate to severe amounts of PLE. Patients with no to mild PLE had a lower MLHFQ score (mean 42, SD 21) compared with patients with a moderate to severe amount of PLE (mean 55, SD 24), p=0.03. For 28 patients (45%) with follow-up data, we observed a linear improvement of the MLHFQ-score (3.2, 95% CI 1.2 to 5.1) with each centimetre reduction of PLE. Correspondingly, patient-reported NYHA-class followed the same pattern as the MLHFQ-score. Our study indicates that patient-reported outcome measures as MLHFQ may be sensitive tools to identify patients with HF at highest risk of symptomatic PLE and that treatment targeting reduction of PLE during follow-up is essential to improvement of QoL and functional capacity of outpatients with HF. NCT01794715; Results. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/.
Full Text Available Background: Health-related quality of life (HRQoL is a rapidly growing area of expertise and the most commonly used patient-reported outcome (PRO. The impact of CF on HRQoL is liable to be great, making CF patients ideal candidates for the application of HRQoL instruments. The aims of this study were to assess the affect of CF on HRQoL, to ascertain the reliability and validity of the UKSIP and the CFQoL in the adult CF population, and to examine their role in the management of patients. Methods: Seventy participants were recruited from the All Wales Adult Cystic Fibrosis Centre at Llandough Hospital, UK. There were two stages to the study; self-report of the UKSIP and CFQoL in the first stage, and completion of the same two questionnaires seven to ten days later for the second stage. Results: The areas of HRQoL most impaired by CF were employment and concerns regarding the future. The UKSIP and CFQoL showed high internal consistency (rα=0.89-0.93 and test-retest reliability (rs=0.57-0.94, p<0.005 in the CF population. Validity was variable; with the UKSIP showing discrimination across socio-demographic factors, whilst the CFQoL showed increased sensitivity to clinical variables. Many parameters influenced patient-reported HRQoL, with the greatest correlations seen with the Borg score (p<0.005. The use of a HRQoL instrument in CF annual reviews is recommended to provide holistic patient care. The results of this study underpin the value of HRQoL as a patient-reported outcome measure in the management of adult CF.
Hasserius, Johan; Hedbys, Josefine
Purpose. Congenital heart disease (CHD) is reported to be associated with Hirschsprung disease (HD). The aim was to evaluate any differences between children with HD with and without CHD, respectively, with regard to patient characteristics, medical care, and patient reported bowel function. Method. This is a retrospective chart study and a cross-sectional long-term follow-up of patients older than 4 years old, including all children with HD operated on with transanal endorectal pull-through (TERPT) at a tertiary center of pediatric surgery. Information about patient characteristics, diagnostics, surgery, and medical care was compiled. At long-term follow-up, bowel function was assessed by Bowel Function Score. Results. Included were 53 HD-patients, 13 with CHD and 40 without CHD. Children with CHD more commonly presented with failure to thrive; 4 (23%) compared to those without CHD (0%) (p < 0.01). In the long-term follow-up, including 32 patients (6 with CHD), constipation was more commonly reported by children with CHD 5 (83%) than by children without CHD 4 (27%) (p = 0.01). No differences were shown in the other parameters such as fecal control and incontinence. Conclusion. HD-patients with CHD more commonly presented with failure to thrive and more frequently reported constipation than HD-patients without CHD. The findings indicate that HD-patients with CHD might need special consideration in their initial care and long-term follow-up.
Symon, Andrew; Downe, Soo; Finlayson, Kenneth William; Knapp, Rebecca; Diggle, Peter
Using patient-reported outcome measures (PROMs) to assess Quality of Life (QoL) is well established, but commonly-used PROM item-sets do not necessarily capture what all respondents consider important...
Hedegaard, Ulla; Hallas, Jesper; Ravn-Nielsen, Lene Vestergaard
potential adherence problems. OBJECTIVES: To evaluate process outcomes and patient- and pharmacist-reported outcomes of a pharmacist adherence intervention for hypertensive patients treated in hospital outpatient clinics. Secondly, to determine the agreement between two different adherence metrics...... calls. Two tools were used to identify adherence problems: The Drug Adherence Work-up (DRAW) tool and an adherence questionnaire. Process data included drug-related problems (DRPs) with recommendations to the physicians, medication- and lifestyle problems identified at the patient interview, actions......-related and 40% lifestyle-related. In connection with the interview, 528 actions were taken within 8 different categories. MI was a central technique applicable for most problems and was employed in nearly all patients (94%). About half of the patients reported increased focus on lifestyle change, and 21...
Hedegaard, Ulla; Hallas, Jesper; Ravn-Nielsen, Lene Vestergaard; Kjeldsen, Lene Juel
Adherence to antihypertensive medications is suboptimal. Hospital pharmacist interventions including motivational interviewing (MI) might assist in improving adherence in patients with hypertension. For an intervention to be useful, it is important to have tools that can easily identify potential adherence problems. To evaluate process outcomes and patient- and pharmacist-reported outcomes of a pharmacist adherence intervention for hypertensive patients treated in hospital outpatient clinics. Secondly, to determine the agreement between two different adherence metrics: an adherence questionnaire used in the intervention and a prescription-based measure. The development of the intervention was based on adherence and behavioral theories and evidence of effective interventions. This included a focused medication review, a patient interview, and follow-up telephone calls. Two tools were used to identify adherence problems: The Drug Adherence Work-up (DRAW) tool and an adherence questionnaire. Process data included drug-related problems (DRPs) with recommendations to the physicians, medication- and lifestyle problems identified at the patient interview, actions taken and time spent on the intervention. In total, 91 DRPs in 8 categories generated recommendations to the physicians; 56 recommendations were generated at the medication review and 35 at the patient interview. At the interview, 421 problems were identified, of which 60% were medication-related and 40% lifestyle-related. In connection with the interview, 528 actions were taken within 8 different categories. MI was a central technique applicable for most problems and was employed in nearly all patients (94%). About half of the patients reported increased focus on lifestyle change, and 21-39% reported increased knowledge, confidence and skills in relation to their medication as well as better quality of life. The pharmacists found that the intervention elements were meaningful pharmacist tasks, and that the DRAW
Bæksted, Christina; Nissen, Aase; Pappot, Helle
CONTEXT: The Common Terminology Criteria for Adverse Events (CTCAE) is the basis for standardized clinician-based grading and reporting of adverse events in cancer clinical trials. The U.S. National Cancer Institute has developed the Patient-Reported Outcomes version of the CTCAE (PRO-CTCAE) to i......CONTEXT: The Common Terminology Criteria for Adverse Events (CTCAE) is the basis for standardized clinician-based grading and reporting of adverse events in cancer clinical trials. The U.S. National Cancer Institute has developed the Patient-Reported Outcomes version of the CTCAE (PRO...
Conclusions: This is the first study to validate the PGI-I as a PRO measure to surgery for BOO. This suggests a potential for the PGI-I to be used to assess surgical therapies for BPH and may be a valuable addition for measuring outcomes in clinical trials evaluating surgical interventions for BPH.
Huang, Hsiaomin; Grant, John A; Miller, Bruce S; Mirza, Faisal M; Gagnier, Joel J
Many patient-reported outcome instruments (or questionnaires) have been developed for use in patients with rotator cuff disease. Before an instrument is implemented, its psychometric properties should be carefully assessed, and the methodological quality of papers that investigate a psychometric component of an instrument must be carefully evaluated. Together, the psychometric evidence and the methodological quality can then be used to arrive at an estimate of an instrument's quality. To identify patient-reported outcome instruments used in patients with rotator cuff disease and to critically appraise and summarize their psychometric properties to guide researchers and clinicians in using high-quality patient-reported outcome instruments in this population. Systematic review. Systematic literature searches were performed to find English-language articles concerning the development or evaluation of a psychometric property of a patient-reported outcome instrument for use in patients with rotator cuff disease. Methodological quality and psychometric evidence were critically appraised and summarized through 2 standardized sets of criteria. A total of 1881 articles evaluating 39 instruments were found per the search strategy, of which 73 articles evaluating 16 instruments were included in this study. The Constant-Murley score, the DASH (Disability of the Arm, Shoulder, and Hand), and the Shoulder Pain and Disability Index were the 3 most frequently evaluated instruments. In contrast, the psychometric properties of the Korean Shoulder Scoring System, Shoulder Activity Level, Subjective Shoulder Value, and Western Ontario Osteoarthritis Shoulder index were evaluated by only 1 study each. The Western Ontario Rotator Cuff Index was found to have the best overall quality of psychometric properties per the established criteria, with positive evidence found in internal consistency, reliability, content validity, hypothesis testing, and responsiveness. The DASH, Shoulder Pain
Mills, Kathryn A G; Naylor, Justine M; Eyles, Jillian P
OBJECTIVE: To examine the influence of different analytical methods, baseline covariates, followup periods, and anchor questions when establishing a minimal important difference (MID) for individuals with knee osteoarthritis (OA). Second, to propose MID for improving and worsening on the Knee...... injury and Osteoarthritis Outcome Score (KOOS). METHODS: Retrospective analysis of prospectively collected data from 272 patients with knee OA undergoing a multidisciplinary nonsurgical management strategy. The magnitude and rate of change as well as the influence of baseline covariates were examined...
Schwitzer, Jonathan A; Albino, Frank P; Mathis, Ryan K; Scott, Amie M; Gamble, Laurie; Baker, Stephen B
Primary outcomes for orthognathic surgery and genioplasty patients include satisfaction with appearance, improved motor function, and enhanced quality of life. The goal of this study was to assess outcomes among patients undergoing these procedures, and to highlight the potential use of FACE-Q instrument for use in patients with dentofacial deformities. A total of 56 patients presenting for orthognathic surgery and/or osseous genioplasty completed the FACE-Q during preoperative and/or at postoperative visits. FACE-Q scores increased following surgery in satisfaction with facial appearance overall (+24.5, P orthognathic surgery either alone or in combination with genioplasty demonstrated statistically significant improvements in satisfaction with facial appearance overall (P orthognathic surgery combined with genioplasty demonstrated greater improvement in satisfaction with chin than patients who underwent genioplasty alone. In conclusion, patients who underwent orthognathic surgery and/or genioplasty demonstrated improvement in appearance and social confidence. The use of this model supports the successful outcomes possible for patients undergoing these procedures.
von Mackensen, S; Campos, I G; Acquadro, C
, important outcomes in clinical trials and clinical practice. As individuals' perception of their well-being often differs from that of their physician, it is recommended that self-report instruments are used to assess patient-reported outcomes (PROs). The way that the impact of haemophilia is perceived...... by the patient and their family can be different, so it is important to assess how parents perceive the impact on their children. A series of PRO instruments have been developed, adapted to different age groups and parents of patients with haemophilia. To allow the instruments to be used internationally......, culturally adapted and linguistically validated translations have been developed; some instruments have been translated into 61 languages. Here, we report the process used for cultural adaptation of the Haemo-QoL, Haem-A-QoL and Hemo-Sat into 28 languages. Equivalent concepts for 22 items that were difficult...
Kasturi, Shanthini; Szymonifka, Jackie; Burket, Jayme C; Berman, Jessica R; Kirou, Kyriakos A; Levine, Alana B; Sammaritano, Lisa R; Mandl, Lisa A
The aims of this study were to assess the construct validity and the test-retest reliability of Patient Reported Outcomes Measurement Information System (PROMIS) computerized adaptive tests (CAT) in patients with systemic lupus erythematosus (SLE). Adults with SLE completed the Medical Outcomes Study Short Form-36, LupusQoL-US version ("legacy instruments"), and 14 selected PROMIS CAT. Using Spearman correlations, PROMIS CAT were compared with similar domains measured with legacy instruments. CAT were also correlated with the Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus Disease Activity Index (SELENA-SLEDAI) disease activity and the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index (SDI) scores. Test-retest reliability was evaluated using ICC. There were 204 outpatients with SLE enrolled in the study and 162 completed a retest. PROMIS CAT showed good performance characteristics and moderate to strong correlations with similar domains in the 2 legacy instruments (r = -0.49 to 0.86, p < 0.001). However, correlations between PROMIS CAT and the SELENA-SLEDAI disease activity and SDI were generally weak and statistically insignificant. PROMIS CAT test-retest ICC were good to excellent, ranging from 0.72 to 0.88. To our knowledge, these data are the first to show that PROMIS CAT are valid and reliable for many SLE-relevant domains. Importantly, PROMIS scores did not correlate well with physician-derived measures. This disconnect between objective signs and symptoms and the subjective patient disease experience underscores the crucial need to integrate patient-reported outcomes into clinical care to ensure optimal disease management.
Rasulnia, Mazi; Burton, Billy Stephen; Ginter, Robert P; Wang, Tracy Y; Pleasants, Roy Alton; Green, Cynthia L; Lugogo, Njira
Low adherence and poor outcomes provide opportunity for digital coaching to engage patients with uncontrolled asthma in their care to improve outcomes. To examine the impact of a remote digital coaching program on asthma control and patient experience. We recruited 51 adults with uncontrolled asthma, denoted by albuterol use of > 2 times per week and/or exacerbations requiring corticosteroids, and applied a 12-week patient-centered remote digital coaching program using a combination of educational pamphlets, symptom trackers, best peak flow establishment, physical activity, and dietary counseling, as well as coaches who implemented emotional enforcement to motivate disease self-management through telephone, text, and email. Baseline and post-intervention measures were quality of life (QOL), spirometry, Asthma Control Test (ACT), Asthma Symptom Utility Index (ASUI), rescue albuterol use, and exacerbation history. Among 51 patients recruited, 40 completed the study. Eight subjects required assistance reading medical materials. Significant improvements from baseline were observed for PROMIS mental status (p = 0.010), body weight and outpatient exacerbation frequency (p = 0.028). The changes from baseline in ACT (p = 0.005) was statistically significant but did not achieve the pre-specified minimum clinically important difference (MCID). Whereas for ASUI, the MCID and statistical significance were achieved. Spirometry and rescue albuterol use were no different. A patient-oriented, remote digital coaching program that utilized trained health coaches and digital materials led to statistically significant improvement in mental status, outpatient exacerbations, body weight, and ASUI. Digital coaching programs may improve some outcomes in adults with uncontrolled asthma.
Hagelstein, V; Ortland, I; Wilmer, A; Mitchell, S A; Jaehde, U
Integrating the patient's perspective has become an increasingly important component of adverse event reporting. The National Cancer Institute has developed a Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE™). This instrument has been translated into German and linguistically validated; however, its quantitative measurement properties have not been evaluated. A German language survey that included 31 PRO-CTCAE items, as well as the EORTC QLQ-C30 and the Oral Mucositis Daily Questionnaire (OMDQ), was distributed at 10 cancer treatment settings in Germany and Austria. Item quality was assessed by analysis of acceptability and comprehensibility. Reliability was evaluated by using Cronbach's' alpha and validity by principal components analysis (PCA), multitrait-multimethod matrix (MTMM) and known groups validity techniques. Of 660 surveys distributed to the study centres, 271 were returned (return rate 41%), and data from 262 were available for analysis. Participants' median age was 59.7 years, and 69.5% of the patients were female. Analysis of item quality supported the comprehensibility of the 31 PRO-CTCAE items. Reliability was very good; Cronbach's' alpha correlation coefficients were >0.9 for almost all item clusters. Construct validity of the PRO-CTCAE core item set was shown by identifying 10 conceptually meaningful item clusters via PCA. Moreover, construct validity was confirmed by the MTMM: monotrait-heteromethod comparison showed 100% high correlation, whereas heterotrait-monomethod comparison indicated 0% high correlation. Known groups validity was supported; PRO-CTCAE scores were significantly lower for those with impaired versus preserved health-related quality of life. A set of 31 items drawn from the German PRO-CTCAE item library demonstrated favourable measurement properties. These findings add to the body of evidence that PRO-CTCAE provides a rigorous method to capture patient self-reports of
Delgado-Herrera, Leticia; Lasch, Kathryn; Popielnicki, Ana; Nishida, Akito; Arbuckle, Rob; Banderas, Benjamin; Zentner, Susan; Gagainis, Ingrid; Zeiher, Bernhardt
Background and objective Following a 2009 US Food and Drug Administration guidance, a new patient-reported outcome (PRO) instrument was developed to support end points in multinational clinical trials assessing irritable bowel syndrome with diarrhea (IBS-D) symptom severity. Our objective was to assess the translatability of the IBS-D PRO instrument into ten languages, and subsequently perform a cultural adaptation/linguistic validation of the questionnaire into Japanese and US Spanish. Materials and methods Translatability assessments of the US English version of the IBS-D PRO were performed by experienced PRO translators who were native speakers of each target language and currently residing in target-language countries. Languages were Chinese (People’s Republic of China), Dutch (the Netherlands), French (Belgium), German (Germany), Japanese (Japan), Polish (Poland), Portuguese (Brazil), Russian (Russia), Spanish (Mexico), and Spanish (US). The project team assessed the instrument to identify potential linguistic and/or cultural adaptation issues. After the issues identified were resolved, the instrument was translated into Spanish (US) and Japanese through a process of two forward translations, one reconciled translation, and one backward translation. The project team reviewed the translated versions before the instruments were evaluated by cognitive debriefing interviews with samples of five Spanish (US) and five Japanese IBS-D patients. Results Linguistic and cultural adaptation concerns identified during the translatability assessment required minor revisions, mainly the presentation of dates/times and word structure. During the cognitive debriefing interviews, two of five Spanish respondents misunderstood the term “bowel movement” to mean only diarrhea in the Spanish version. Consequently, the term was changed from “movimiento intestinal” to “evacuaciones”. None of the Japanese respondents identified issues with the Japanese version. Conclusion
Thorborg, K; Tijssen, M; Habets, B;
ratings of poor study methodology (23% and 31%, respectively), whereas IHOT-33 and HOS had a somewhat larger distribution (46%). These PROs all contain adequate measurement qualities for content validity (except HOS), test-retest reliability, construct validity, responsiveness and interpretability....... No information or poor quality rating on methodological aspects made it impossible to fully evaluate the remaining PROs at present. CONCLUSIONS: HAGOS, HOS, IHOT-12 and IHOT-33 can be recommended for assessment of young-aged to middle-aged adults with pain related to the hip joint, undergoing non......BACKGROUND/AIM: To recommend Patient-Reported Outcome (PRO) questionnaires to measure hip and groin disability in young-aged to middle-aged adults. METHODS: A systematic review was performed in June 2014. The methodological quality of the studies included was determined using the COnsensus...
Reaney, Matthew; Mathieu, Chantal; Ostenson, Claes-Göran
characteristics; therefore, no statistical comparisons of endpoints between main cohorts were conducted. RESULTS: There were 2388 patients eligible for analysis (exenatide BID cohort, n = 1114; insulin cohort, n = 1274). Mean positive changes in Impact of Weight on Quality of Life-Lite (IWQOL-Lite) total score......BACKGROUND: Improvements in the clinical condition of patients with type 2 diabetes are often accompanied by improvements in health-related quality of life and other patient-reported outcomes (PROs), but data assessing injectable treatment initiation from the patient's perspective in routine...... countries. Patients initiated exenatide twice daily (BID) or insulin based on a physician's clinical judgement. Clinical and PRO data were collected at baseline (injectable therapy initiation) and after approximately 3, 6, 12, 18 and 24 months. The two treatment cohorts had different baseline...
Gomes, Manuel; Gutacker, Nils; Bojke, Chris; Street, Andrew
Patient-reported outcome measures (PROMs) are now routinely collected in the English National Health Service and used to compare and reward hospital performance within a high-powered pay-for-performance scheme. However, PROMs are prone to missing data. For example, hospitals often fail to administer the pre-operative questionnaire at hospital admission, or patients may refuse to participate or fail to return their post-operative questionnaire. A key concern with missing PROMs is that the individuals with complete information tend to be an unrepresentative sample of patients within each provider and inferences based on the complete cases will be misleading. This study proposes a strategy for addressing missing data in the English PROM survey using multiple imputation techniques and investigates its impact on assessing provider performance. We find that inferences about relative provider performance are sensitive to the assumptions made about the reasons for the missing data.
Papachristos, Alexander; Edwards, Elton; Dowrick, Adam; Gosling, Cameron
Despite a number of injury prevention campaigns and interventions, horse riding continues to be a dangerous activity, resulting in more accidents per hour than motorcycling, skiing and football. Injuries are often serious, with one in four patients requiring admission to hospital. This study aims to describe the severity of equestrian-related injuries (ERIs) using both clinical parameters and patient-reported outcomes. A retrospective study of all patients aged ≥18 years admitted to The Alfred Hospital between January 2003 and January 2008 with an ERI was performed. Specific clinical data were extracted from the medical record. In addition, a questionnaire was conducted identifying the details of the accident, the required recovery time and levels of ongoing pain and physical disability. During the study period 172 patients met the inclusion criteria. There were three deaths (2%). Eighty-two patients (48%) suffered head injuries. Forty-one patients (24%) were admitted to the ICU and 31 patients (18%) required mechanical ventilation. On discharge, 41 patients (24%) required transfer to a sub-acute rehabilitation facility. One-hundred-and-twenty-four patients (72%) completed the questionnaire. Thirty-nine respondents (31%) were not wearing a helmet. Among patients injured for more than 6 months, 38 (35%) still experienced moderate or severe pain or disability. Ninety-five patients had returned to work at the time of review, among which 47(50%) required longer than 6 months to recover, and 40 (42%) returned at a reduced capacity. The clinical and patient-reported outcomes of ERIs requiring hospital admission are poor. Persistent pain and disability are common, even up to 5 years post-injury. A large proportion of patients required longer than 6 months to return to work and many return at a reduced capacity. Copyright © 2014 Elsevier Ltd. All rights reserved.
Razdan, Shantanu N; Patel, Vishal; Jewell, Sarah; McCarthy, Colleen M
Bilateral prophylactic mastectomy (BPM) is effective in reducing the risk of breast cancer in women with a well-defined family history of breast cancer or in women with BRCA 1 or 2 mutations. Evaluating patient-reported outcomes following BPM are thus essential for evaluating success of BPM from patient's perspective. Our systematic review aimed to: (1) identify studies describing health-related quality of life (HRQOL) in patients following BPM with or without reconstruction; (2) assess the effect of BPM with or without reconstruction on HRQOL; and (3) identify predictors of HRQOL post-BPM. We performed a systematic review of literature using the PRISMA guidelines. PubMed, Embase, PsycINFO, Web of Science, Scopus and Cochrane databases were searched. The initial search resulted in 1082 studies; 22 of these studies fulfilled our inclusion criteria. Post-BPM, patients are satisfied with the outcomes and report high psychosocial well-being and positive body image. Sexual well-being and somatosensory function are most negatively affected. Vulnerability, psychological distress and preoperative cancer distress are significant negative predictors of quality of life and body image post-BPM. There is a paucity of high-quality data on outcomes of different HRQOL domains post-BPM. Future studies should strive to use validated and breast-specific PRO instruments for measuring HRQOL. This will facilitate shared decision-making by enabling surgeons to provide evidence-based answers to women contemplating BPM.
Boezem, P.B. van den; Velthuis, S.; Lourens, H.J.; Samlal, R.A.; Cuesta, M.A.; Sietses, C.
OBJECTIVE: The aim of this study was to report the clinical and cosmetic results of transvaginal hybrid cholecystectomy (TVC). BACKGROUND: Natural orifice transluminal endoscopic surgery (NOTES) has been developed as a minimal invasive alternative for conventional laparoscopic cholecystectomy. Altho
Orri, Massimiliano; Sibeoni, Jordan; Labey, Mathilde; Bousquet, Guilhem; Verneuil, Laurence; Revah-Levy, Anne
The past decade has been characterised by movement from a doctor-centred to a patient-centred approach to treatment outcomes, in which doctors try to see the illness through their patients' eyes. Patients, family members and doctors are the three participants in cancer care, but their perspectives about what have been helpful during cancer treatment have never simultaneously and explicitly compared in the same qualitative study. The aim of this study project is to explore patients' perspectives about the care they receive, as well as families' and doctors' perspectives about what have been helpful for the patient. These three points of view will be compared and contrasted in order to analyse the convergences and divergences in these perspectives. This is a national multicentre qualitative study. Participants will be constituted by three different subsamples: (1) patients with cancer (skin, breast, urological and lung cancers), (2) their relatives, and (3) their referring physicians. Recruitment will follow the purposive sample technique, and the final sample size will be determined by data saturation. Data will be collected through open-ended semistructured interviews and independently analysed with NVivo V.10 software by three researchers according to the principles of Interpretative Phenomenological Analysis. The research protocol received approval from the University Paris Descartes review board (IRB number: 20140600001072), and participants will provide written consent. To the best of our knowledge, this is the first study to focus on the simultaneous exploration of the separate points of view of patients, families and doctors about the care received during the cancer care journey. We expect that our findings will help to improve communication and relationships between doctors, patients and families. Comparison of these three points of view will provide information about the convergences and divergences of these perspectives and how to address the needs of all
Guyatt Gordon H
Full Text Available Abstract Background Combining outcomes and the use of standardized effect measures such as effect size and standardized response mean across instruments allows more comprehensive meta-analyses and should avoid selection bias. However, such analysis ideally requires that the instruments correlate strongly and that the underlying assumption of similar responsiveness is fulfilled. The aim of the study was to assess the correlation between two widely used health-related quality of life instruments for patients with chronic obstructive pulmonary disease and to compare the instruments' responsiveness on a study level. Methods We systematically identified all longitudinal studies that used both the Chronic Respiratory Questionnaire (CRQ and the St. George's Respiratory Questionnaire (SGRQ through electronic searches of MEDLINE, EMBASE, CENTRAL and PubMed. We assessed the correlation between CRQ (scale 1 – 7 and SGRQ (scale 1 – 100 change scores and compared responsiveness of the two instruments by comparing standardized response means (change scores divided by their standard deviation. Results We identified 15 studies with 23 patient groups. CRQ change scores ranged from -0.19 to 1.87 (median 0.35, IQR 0.14–0.68 and from -16.00 to 3.00 (median -3.00, IQR -4.73–0.25 for SGRQ change scores. The correlation between CRQ and SGRQ change scores was 0.88. Standardized response means of the CRQ (median 0.51, IQR 0.19–0.98 were significantly higher (p Conclusion Investigators should be cautious about pooling the results from different instruments in meta-analysis even if they appear to measure similar constructs. Despite high correlation in changes scores, responsiveness of instruments may differ substantially and could lead to important between-study heterogeneity and biased meta-analyses.
Full Text Available Objective: To investigate patients’ reported outcome following medication with two brands of 400 mg ibuprofen used to alleviate musculoskeletal pains.Methods: Adult peasant manual laborers (85 who met criteria were randomly assigned to receive either of the brands (A or B. Data on pain alleviation were gathered using the Short-Form McGill Pain Questionnaire (SF-MPQ, Visual Analogue Scale (VAS, Present Pain Intensity (PPI, and Clinical Global Impression of Improvement (CGII scales. Interval data obtained from the two brands were compared using the Students’ t-test at 95% confidence interval.Results: There were 42 participants, mean age=29.2 (SD=1.37 assigned to brand A and 43 (mean age=28.8 SD=1.14 in brand B of ibuprofen 400 mg. Brand B was consistently rated higher than brand A. Scores for medication efficacy were 10.4 (SD=1.65 (brand A and 11.4 (SD=1.68 (brand B; t=2.768, P=0.007. Alleviation of pain symptoms: 10.8 (SD=1.64 and 11.6 (SD=1.72; t = 2.194, P=0.031. Similarly, rated scores on the impact of pain on quality of life were 10.5 (SD=2.00 and 12.1 (SD=1.85; t=3.830, P<0.001. There was a reduction in Present Pain Intensity scores by 32.7% and 34.3% for Brand A and brand B participants respectively. The decrease in Visual Analog pain scale score was 35.9% and 37.3% for brand A and brand B participants respectively. The decrease in SF-MPQ was by 85.1% and 69.9% for the brand A and brand B groups respectively. The clinical global impression of improvement for both groups of patients indicated an improvement rate of 71.4% and 61.9% for brand A and 81.4% and 74.4% for brand B participants. Conclusion: This clinical study infers that though the two brands of ibuprofen 400 mg are legally pharmaceutical equivalent, they are not clinically equivalent. In most of the parameters evaluated, brand B was rated more efficacious than brand A. This explains the patients’ preferences and demand for this brand of ibuprofen in the Nigerian community.
Yiu Yan Leung
Full Text Available OBJECTIVES: To investigate the effect of persistent neurosensory disturbance of the lingual nerve (LN or inferior alveolar nerve (IAN on general health and oral health- related quality of life (QoL. METHODS: The study design was a case-control study. Patients with persistent neurosensory deficit of LN or IAN after lower third molar surgery (for 12 months or more were the study group. The control group was an age and gender matched sample of patients who had dental extractions or lower third molar surgeries without trigeminal neurosensory deficit. The outcome variables were the general health and oral health-related QoL. General health-related QoL was assessed using the 36-item Short Form Health Survey (SF-36 and oral health-related QoL using the 14-item Short Form Oral Health Impact Profile (OHIP-14. Differences in SF-36 scores and OHIP-14 scores between the groups were compared. RESULTS: Forty-eight subjects (24 cases and 24 controls were recruited. When compared to the control group, patients with neurosensory deficits had poorer Mental-Health Component Scores (MCS (p = 0.005, General Health (p = 0.023, Vitality (p = 0.048, Social Functioning (p = 0.003, Role-emotion (p = 0.008 and Mental Health (p = 0.022. The OHIP-14 scores were also significantly worse in this patients with neurosensory deficits compared with the control group (p = 0.002. When compared within the study group, older patient with neurosensory deficit was found to correlate with worse Physical Health Component Scores (PCS (p = 0.02 and OHIP-14 scores (p = 0.02, while more severe visualized analog scaling rating of numbness was correlated with a worse PCS (p = 0.034. CONCLUSIONS: Patients with persistent LN or IAN deficit after lower third molar surgery have poorer health-related QoL and poorer oral health-related QoL than those without such deficits.
Polk, Anne; Rasmussen, Jeppe V; Brorson, Stig;
Patient-reported outcome measures (PROMs) are used by some arthroplasty registries to evaluate results after surgery, but non-response may bias the results. The aim was to identify a potential bias in the outcome scores of subgroups in a cohort of patients from the Danish Shoulder Arthroplasty...... Registry (DSR) and to characterize non-responders....
Hinman, Rana S; Dobson, Fiona; Takla, Amir; O'Donnell, John; Bennell, Kim L
The most reliable patient-reported outcomes (PROs) for people with femoroacetabular impingement (FAI) is unknown because there have been no direct comparisons of questionnaires. Thus, the aim was to evaluate the test-retest reliability of six existing PROs in a single cohort of young active people with hip/groin pain consistent with a clinical diagnosis of FAI. Young adults with clinical FAI completed six PRO questionnaires on two occasions, 1-2 weeks apart. The PROs were modified Harris Hip Score, Hip dysfunction and Osteoarthritis Score, Hip Outcome Score, Non-Arthritic Hip Score, International Hip Outcome Tool, Copenhagen Hip and Groin Outcome Score. 30 young adults (mean age 24 years, SD 4 years, range 18-30 years; 15 men) with stable symptoms participated. Intraclass correlation coefficient(3,1) values ranged from 0.73 to 0.93 (95% CI 0.38 to 0.98) indicating that most questionnaires reached minimal reliability benchmarks. Measurement error at the individual level was quite large for most questionnaires (minimal detectable change (MDC95) 12.4-35.6, 95% CI 8.7 to 54.0). In contrast, measurement error at the group level was quite small for most questionnaires (MDC95 2.2-7.3, 95% CI 1.6 to 11). The majority of the questionnaires were reliable and precise enough for use at the group level. Samples of only 23-30 individuals were required to achieve acceptable measurement variation at the group level. Further direct comparisons of these questionnaires are required to assess other measurement properties such as validity, responsiveness and meaningful change in young people with FAI.
Weinstock-Zlotnick, Gwen; Page, Carol; Ghomrawi, Hassan M K; Wolff, Aviva L
Clinical measurement. Few studies describe the responsiveness of functional outcomes measures in patients sustaining hand fractures. 1--To explore the responsiveness of three function-oriented Patient Report Outcome (PRO) measures with a cohort of hand fracture patients. 2--To examine patients' PRO preference. 60 participants with 74 hand fractures at an outpatient hospital-based hand therapy clinic consented to participate in this study. They completed the Disabilities of the Arm, Shoulder, and Hand Questionnaire (DASH), Michigan Hand Outcomes Questionnaire (MHQ), and Patient-Rated Wrist/Hand Evaluation (PRWHE) at three trials: T1 (evaluation), T2 (one month later), and T3 (two months later). Participants also identified which PRO they felt best reflected their hand use and which was easiest to complete. Descriptive statistics, analyses of variance (ANOVA), effect size, and standardized response mean (SRM) were employed to describe participants, determine functional change between trials, and examine and compare PRO responsiveness. Questionnaire preference at T1 was reported. Participants demonstrated functional improvement, as measured by the DASH, PRWHE, and MHQ. T1 scores: DASH = 41.85 (SD ± 22.78), MHQ = 50.13 (SD ± 18.36), and PRWHE = 48.18 (SD ± 22.07). T2 scores: DASH = 22.11 (SD ± 18.18), MHQ = 69.89 (SD ± 15.93), and PRWHE = 22.62 (SD ± 18.15). T3 scores: DASH = 17.56 (SD ± 18.01), MHQ = 75.37 (SD ± 19.19), and PRWHE = 22.40 (SD ± 19.04). Each PRO demonstrated significant test score differences between trials (p responsiveness (≥.80) was noted between T1 and T2: (effect size: .98-1.23; SRM: 1.31-1.49) and T1 and T3 (effect size: 1.21-1.54; SRM 1.49-1.84). Smaller responsiveness effects were noted between T2 and T3 (effect size: .35-.64, SRM: .38-.81). No significant differences between questionnaire responsiveness were found. Patients reported PRWHE easiest to complete and MHQ best reflecting their hand use. DASH, MHQ, and PRWHE were each able
Stephanie J. Sohl
Full Text Available Lifestyle change is recommended as treatment for adults at risk for metabolic syndrome (MetS, although adoption of new behavioral patterns is limited. In addition, most existing lifestyle interventions do not address psychological stress or quality of life, both of which impact the burden of MetS. Yoga, a form of physical activity that incorporates psychological components (e.g., maintaining attention, relaxation, is a promising intervention for improving the burden of MetS. This randomized controlled trial assessed the feasibility and preliminary efficacy of a 12-week yoga program coupled with an evidence-based health education program (HED compared to HED alone. A secondary, exploratory aim examined perceived stress, quality of life, and related psychological outcomes (mindfulness, perceived health competence, and mood. Sixty-seven adults at risk for MetS enrolled (mean age [SD]: 58  years; 50% male; 79% non-Hispanic White. Preliminary results revealed significantly larger improvements in two quality of life domains (role-physical and general health perceptions in the HED plus yoga group versus HED alone (ps<0.05. This is the first study that implemented lifestyle education along with yoga to evaluate the potential unique effects of yoga on participants at risk for MetS. A larger clinical trial is warranted to further investigate these promising patient-reported outcomes.
Howard, Michael A; Sisco, Mark; Yao, Katharine; Winchester, David J; Barrera, Ermilo; Warner, Jeremy; Jaffe, Jennifer; Hulick, Peter; Kuchta, Kristine; Pusic, Andrea L; Sener, Stephen F
The authors sought to study patient-reported outcomes following nipple-sparing mastectomy (NSM). From 2008 to 2011, the BREAST-Q was administered to women undergoing NSM surgery for cancer treatment or risk-reduction prior to surgery and at 2 years after completion of reconstruction. The change in score over time and the impact of surgical indication, complication occurrence, and laterality on scores were analyzed. The BREAST-Q was prospectively administered to 39 women undergoing NSM for cancer treatment (n = 17) or risk-reduction (RR) (n = 22). At 2 years after operation, median overall satisfaction with breasts was 75 (IQR = 67,100). There were significant postoperative increases in scores for overall satisfaction with breasts (+8, P = 0.021) and psychosocial well-being (+14, P = 0.003). Postoperatively, RR patients had significantly higher scores for psychosocial wellness, physical impact (chest), and overall satisfaction with outcome compared to cancer treatment patients (P quality of life as measured by BREAST-Q. Satisfaction level increased 2 years following operation. J. Surg. Oncol. 2016;114:416-422. © 2016 Wiley Periodicals, Inc. © 2016 Wiley Periodicals, Inc.
Khan, Nasim Ahmed; Spencer, Horace Jack; Nikiphorou, Elena; Naranjo, Antonio; Alten, Rieki; Chirieac, Rodica M; Drosos, Alexandros A; Géher, Pál; Inanc, Nevsun; Kerzberg, Eduardo; Ancuta, Codrina Mihaela; Müller, Rüediger; Ørnbjerg, Lykke; Sokka, Tuulliki
To assess intercentre variability in the ACR core set measures, DAS28 based on three variables (DAS28v3) and Routine Assessment of Patient Index Data 3 in a multinational study. Seven thousand and twenty-three patients were recruited (84 centres; 30 countries) using a standard protocol in the Quantitative Standard Monitoring of Patients with RA study. Analysis of variance (ANOVA) and mixed-effect analysis of covariance models were used to model the relationship between study centre and different patient-reported and physician-reported RA activity measures. These models were built to adjust for the remaining ACR core set measure (for each ACR core set measure or each composite index), socio-demographics and medical characteristics. ANOVA and analysis of covariance models yielded similar results, and ANOVA tables were used to present variance attributable to recruiting centre. The proportion of variances attributable to recruiting centre was lower for patient reported outcomes (PROs: pain, HAQ, patient global) compared with objective measures (joint counts, ESR, physician global) in all models. In the full model, variance in PROs attributable to recruiting centre ranged from 1.53% for patient global to 3.71% for HAQ compared with objective measures that ranged from 5.92% for physician global to 9.25% for ESR; and was lower for Routine Assessment of Patient Index Data 3 (2.6%) compared with DAS28v3 (11.75%). Intercentre variability in PROs is lower than objective measures of RA activity demonstrating that PROs may be more comparable across centres, and the need for standardization of objective measures.
Diener, Lawrence W.; Nahm, Meredith; Weinfurt, Kevin P.
New technologies may be required to integrate the National Institutes of Health’s Patient Reported Outcome Management Information System (PROMIS) into multi-center clinical trials. To better understand this need, we identified likely PROMIS reporting formats, developed a multi-center clinical trial process model, and identified gaps between current capabilities and those necessary for PROMIS. These results were evaluated by key trial constituencies. Issues reported by principal investigators fell into two categories: acceptance by key regulators and the scientific community, and usability for researchers and clinicians. Issues reported by the coordinating center, participating sites, and study subjects were those faced when integrating new technologies into existing clinical trial systems. We then defined elements of a PROMIS Tool Kit required for integrating PROMIS into a multi-center clinical trial environment. The requirements identified in this study serve as a framework for future investigators in the design, development, implementation, and operation of PROMIS Tool Kit technologies. PMID:20703765
刘力; 常玉双; 沈舒文; 宇文亚; 惠建萍; 黄毓娟
目的 编制慢性萎缩性胃炎癌前病变(PLGC)患者报告的临床结局(PRO)评价量表(PLGC-PRO量表),并对其信度及效度进行评价.方法 通过文献查询、Delphi法、预调查等方法构建条目池,筛选优化条目,形成初步量表,在陕西地区进行量表测试,并对204份有效问卷进行信度、效度分析.信度分析主要采用测量内在一致信度的克朗巴赫α系数法、折半系数法;效度分析主要采用t检验考察其区分效度,采用探索性因子分析和证实性因子分析评价其结构效度.结果 初步形成具有3个领域、30个条目的 PLGC-PRO量表.该量表总克朗巴赫α系数为0.892,分半信度系数为0.715.量表各条目都达到了t检验法的保留标准(P＜0.05);量表中30个条目经因子分析选出5个因子,累计方差贡献率为52.372%;量表的拟合优度指数(GFI)为0.954,生理领域、心理领域、社会环境领域的GFI分别为0.962、0.905和0.903.结论 初步表明PLGC-PRO量表具有较好的信度、效度,可尝试用于PLGC患者的临床疗效评价.%Objective To develop a precancerous lesion of atrophic gastritis patient reported outcome (PRO) scale (PLAGPRO) and analyze its reliability and validity. Methods The scale was developed by establishing item pool through literature retrieval, Delphi method and pre-investigation, and selecting and optimizing items. Preliminary investigation was conducted in Shaanxi province, and the data of 204 valid respondents was used to assess the reliability and validity. The reliability was measured by internal consistency Cronbach's α coefficient and split-half coefficient. The discriminate validity and constructive validity were assessed by T test and exploratory factor analysis and confirmatory factor analysis respectively. Results The PLAG-PRO scale with 3 field and 30-item in it was developed. Its Cronbach's α coefficient was 0. 892, split-half coefficient 0. 715, and all items reached the reservation
Methodological issues in examining measurement equivalence in patient reported outcomes measures: Methods overview to the two-part series, “Measurement equivalence of the Patient Reported Outcomes Measurement Information System® (PROMIS® short forms”
Jeanne A. Teresi
Full Text Available The purpose of this article is to introduce the methods used and challenges confronted by the authors of this two-part series of articles describing the results of analyses of measurement equivalence of the short form scales from the Patient Reported Outcomes Measurement Information System® (PROMIS®. Qualitative and quantitative approaches used to examine differential item functioning (DIF are reviewed briefly. Qualitative methods focused on generation of DIF hypotheses. The basic quantitative approaches used all rely on a latent variable model, and examine parameters either derived directly from item response theory (IRT or from structural equation models (SEM. A key methods focus of these articles is to describe state-of-the art approaches to examination of measurement equivalence in eight domains: physical health, pain, fatigue, sleep, depression, anxiety, cognition, and social function. These articles represent the first time that DIF has been examined systematically in the PROMIS short form measures, particularly among ethnically diverse groups. This is also the first set of analyses to examine the performance of PROMIS short forms in patients with cancer. Latent variable model state-of-the-art methods for examining measurement equivalence are introduced briefly in this paper to orient readers to the approaches adopted in this set of papers. Several methodological challenges underlying (DIF-free anchor item selection and model assumption violations are presented as a backdrop for the articles in this two-part series on measurement equivalence of PROMIS measures.
Full Text Available Dagmar Amtmann,1 Jiseon Kim,1 Hyewon Chung,2 Robert L Askew,3 Ryoungsun Park,4 Karon F Cook5 1Department of Rehabilitation Medicine, University of Washington, Seattle, WA, USA; 2Department of Education, Chungnam National University, Daejeon, Republic of Korea; 3Department of Psychology, Stetson University, Deland, FL, USA; 4Theoretical and Behavioral Foundations Division, Wayne State University, Detroit, MI, USA; 5Department of Medical Social Science, Northwestern University, Chicago, IL, USA Background: The minimally important difference (MID refers to the smallest change that is sufficiently meaningful to carry implications for patients' care. MIDs are necessary to guide the interpretation of scores. This study estimated MID for the Patient Reported Outcomes Measurement Information System (PROMIS pain interference (PI. Methods: Study instruments were administered to 414 people who participated in two studies that included treatment with low back pain (LBP; n=218 or depression (n=196. Participants with LBP received epidural steroid injections and participants with depression received antidepressants, psychotherapy, or both. MIDs were estimated for the changes in LBP. MIDs were included only if a priori criteria were met (ie, sample size ≥10, Spearman correlation ≥0.3 between anchor measures and PROMIS-PI scores, and effect size range =0.2–0.8. The interquartile range (IQR of MID estimates was calculated. Results: The IQR ranged from 3.5 to 5.5 points. The lower bound estimate of the IQR (3.5 was greater than mean of standard error of measurement (SEM both at time 1 (SEM =2.3 and at time 2 (SEM =2.5, indicating that the estimate of MID exceeded measurement error. Conclusion: Based on our results, researchers and clinicians using PROMIS-PI can assume that change of 3.5 to 5.5 points in comparisons of mean PROMIS-PI scores of people with LBP can be considered meaningful. Keywords: minimally important differences, pain interference, back pain
Strand, Vibeke; Lee, Eun Bong; Fleischmann, Roy; Koncz, Tamas; Zwillich, Samuel H; Gruben, David; Wilkinson, Bethanie; Krishnaswami, Sriram; Wallenstein, Gene
Objectives To compare patient-reported outcomes (PROs) in methotrexate (MTX)-naive patients (defined as no prior treatment or ≤3 doses) receiving tofacitinib versus MTX. Methods In the 24-month, phase III, randomised, controlled, ORAL Start trial (NCT01039688), patients were randomised 2:2:1 to receive tofacitinib 5 mg two times per day (n=373), tofacitinib 10 mg two times per day (n=397) or MTX (n=186). PROs assessed included Patient Global Assessment of disease (PtGA), pain, Health Assessment Questionnaire-Disability Index (HAQ-DI), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and health-related quality of life (Short Form-36 [SF-36]). Results PROs improved following tofacitinib and MTX treatment: benefits were sustained over 24 months. Patients receiving tofacitinib reported earlier responses which were significantly different between each tofacitinib dose and MTX at month 3 through month 24. At month 6 (primary end point), significant improvements versus MTX were observed in PtGA, pain, HAQ-DI, SF-36 Physical Component Summary (PCS), 5/8 domain scores and FACIT-F with tofacitinib 5 mg two times per day; all PROs, except SF-36 Mental Component Summary Score and Medical Outcomes Survey-Sleep, with tofacitinib 10 mg two times per day. At month 6, the proportion of patients reporting improvements ≥minimum clinically important difference were significant versus MTX with tofacitinib 5 mg two times per day in PtGA and 3/8 SF-36 domains; and with tofacitinib 10 mg two times per day in PtGA, pain, HAQ-DI, SF-36 PCS, 4/8 domains and FACIT-F. Conclusions Patients with rheumatoid arthritis receiving tofacitinib 5 and 10 mg two times per day monotherapy versus MTX reported statistically significant and clinically meaningful improvements in multiple PROs over 24 months; onset of benefit with tofacitinib treatment occurred earlier. Trial registration number NCT01039688.
Watt, Torquil; Barbesino, Giuseppe; Bjørner, Jakob
BACKGROUND AND PURPOSE: Thyroid diseases are common and often affect quality of life (QoL). No cross-culturally validated patient-reported outcome measuring thyroid-related QoL is available. The purpose of the present study was to test the cross-cultural validity of the newly developed thyroid...... = 148), Denmark (n = 902) and Sweden (n = 187). Translated versions were compared pairwise to the English version by examining uniform and nonuniform DIF, i.e., whether patients from different countries respond differently to a particular item, although they have identical level of the concept measured...... scale scores, most of which could be explained by sample differences not controlled for. CONCLUSION: The ThyPRO has good cross-cultural validity with only minor cross-cultural invariance and is recommended for use in international multicenter studies....
Bradford W. Fenton
Full Text Available Chronic pelvic pain affects multiple aspects of a patient’s physical, social, and emotional functioning. Latent class analysis (LCA of Patient Reported Outcome Measures Information System (PROMIS domains has the potential to improve clinical insight into these patients’ pain. Based on the 11 PROMIS domains applied to n=613 patients referred for evaluation in a chronic pelvic pain specialty center, exploratory factor analysis (EFA was used to identify unidimensional superdomains. Latent profile analysis (LPA was performed to identify the number of homogeneous classes present and to further define the pain classification system. The EFA combined the 11 PROMIS domains into four unidimensional superdomains of biopsychosocial dysfunction: Pain, Negative Affect, Fatigue, and Social Function. Based on multiple fit criteria, a latent class model revealed four distinct classes of CPP: No dysfunction (3.2%; Low Dysfunction (17.8%; Moderate Dysfunction (53.2%; and High Dysfunction (25.8%. This study is the first description of a novel approach to the complex disease process such as chronic pelvic pain and was validated by demographic, medical, and psychosocial variables. In addition to an essentially normal class, three classes of increasing biopsychosocial dysfunction were identified. The LCA approach has the potential for application to other complex multifactorial disease processes.
Poku, Edith; Duncan, Rosie; Keetharuth, Anju; Essat, Munira; Phillips, Patrick; Woods, Helen Buckley; Palfreyman, Simon; Jones, Georgina; Kaltenthaler, Eva; Michaels, Jonathan
Peripheral arterial disease (PAD) is generally associated with considerable morbidity and reduced quality of life. Patient-reported outcome measures (PROMs) provide important information about the burden of disease and impact of treatment in affected patients. The objective of the review was to identify and appraise studies reporting the psychometric evaluation of PROMs administered to a specified population of patients with PAD with a view to recommending suitable PROMs. A systematic review of peer-reviewed English language articles was undertaken to identify primary studies reporting psychometric properties of PROMs in English-speaking patients with various stages of PAD. Comprehensive searches were completed up until January 2015. Study selection, data extraction and quality assessment were undertaken independently by at least two researchers. Findings were presented as tabular and narrative summaries based on accepted guidance. Psychometric evaluation of 6 generic and 7 condition-specific PROMs reported in 14 studies contributed data to the review. The frequently reported measure was the SF-36 (n = 11 studies); others included the Walking Impairment Questionnaire (n = 8 studies), EQ-5D (n = 5 studies) and the Vascular Quality of Life Questionnaire (n = 3 studies). Studies included a diverse PAD population and varied in methodology, including approach to validation of PROMs. Various PROMs have been validated in patients with PAD but no study provided evidence of a full psychometric evaluation in the patient population. Careful selection is required to identify reliable and valid PROMs to use in clinical and research settings.
Takeuchi, Hiroyoshi; Fervaha, Gagan; Remington, Gary
This study aimed to assess patient's capacity to perform a patient-reported outcome (PRO) measure (i.e., a self-rating scale) and examine its relationship with clinical characteristics including cognition. Fifty patients with schizophrenia were asked to rate the Subjective Well-being under Neuroleptics scale - Short form (SWNS) twice; the second rating was started immediately after they completed the first to minimize the gap between ratings. At the same time, the Positive and Negative Symptoms Scale (PANSS) and Brief Neurocognitive Assessment (BNA) were administered. The correlations between the two ratings for the SWNS total and each item scores were high (rs=0.94 and rs=0.60-0.84, respectively); however, for 16 (80%) of 20 items, 5 or more patients (i.e., ≥10%) demonstrated a>1 point score difference. There was no significant correlation between the SWNS total score difference and any clinical characteristics including age, education duration, illness duration, antipsychotic dose, psychopathology, and cognition. In contrast, the number of items with a>1 point score difference was significantly correlated with disorganized symptoms and overall severity (rs=0.29 for both), as well as working memory and global cognition (rs=-0.41 and rs=-0.40, respectively). These findings suggest that PROs should be interpreted with caution in patients with schizophrenia with prominent disorganization and cognitive impairment.
Kelsall, Jennett E; McCulley, Stephen J; Brock, Lisa; Akerlund, Malin T E; Macmillan, R Douglas
Oncoplastic breast conserving surgery (OBCS) allows women who may otherwise have mastectomy and immediate reconstruction (MxIR) the choice to conserve their breast yet avoid deformity. We compared the outcome of these options. Two cohorts meeting study criteria were identified from prospectively audited series of women undergoing OBCS or MxIR. After case matching for age, tumour size and date of surgery, stratification by breast size and controlling for radiotherapy; body image scale (BIS) scores of psychosocial function and patient reported outcome measures (PROMs) for breast appearance and return to function were analysed. A total of 567 women (286 treated by OBCS and 281 by MxIR) fulfilled inclusion criteria. Demographics were similar between the two unmatched cohorts, except for radiotherapy, age and tumour size (all p < 0.001). Overall, BIS score (p = 0.002), self-rated breast appearance, return to work and function (all p < 0.001) significantly favoured OBCS. Case-matched women with larger breasts treated by OBCS reported better BIS scores (mean 3.30 vs. 5.37, p = 0.011) and self-rated breast appearance score (p < 0.001) than MxIR, whereas no significant difference was observed for smaller breasts. BIS and appearance favoured OBCS, regardless of whether radiotherapy would have been avoided if treated by MxIR. OBCS offers suitable women the option to avoid MxIR while providing faster recovery. Better psychosocial and self-rated satisfaction with breast appearance is achieved for OBCS in all groups, regardless of the need for radiotherapy, apart from those women with smaller breasts for whom the results are comparable. Copyright © 2017 British Association of Plastic, Reconstructive and Aesthetic Surgeons. Published by Elsevier Ltd. All rights reserved.
Robb, Kathryn A; Lo, Siu Hing; Power, Emily; Kralj-Hans, Ines; Edwards, Robert; Vance, Maggie; von Wagner, Christian; Atkin, Wendy; Wardle, Jane
Flexible sigmoidoscopy (FS) screening for colorectal cancer will be introduced into the National Cancer Screening Programmes in England in 2013. Patient-reported outcome measures (PROMs) from trial participants indicate high acceptability and no adverse physical or psychological consequences, but this may not generalize to routine screening in the community. This study examined PROMs in a community-based FS screening programme. Eligible adults aged 58-59 (n = 2016) registered at 34 London general practices were mailed a National Health Service-endorsed invitation to attend FS screening. Pain and side-effects were assessed in a 'morning-after' questionnaire, and satisfaction was assessed in a three-month follow-up questionnaire. Anxiety, self-rated health and colorectal symptoms were assessed at prescreening and follow-up. In total, 1020 people attended screening and were included in the current analyses, of whom 913 (90%) returned the morning-after questionnaire, and 674 (66%) the follow-up questionnaire. The prescreening questionnaire had been completed by 751 (74%) of those who attended. The majority (87%) of respondents reported no pain or mild pain, and the most frequent side-effect (wind) was only experienced more than mildly by 16%. Satisfaction was extremely high, with 98% glad they had the test; 97% would encourage a friend to have it. From prescreening to follow-up there were no changes in anxiety or self-rated health, and the number of colorectal symptoms declined. Satisfaction and changes in wellbeing were not moderated by gender, deprivation, ethnicity or screening outcome. PROMs indicate high acceptability of FS screening in 58-59 year olds, with no adverse effects on colorectal symptoms, health status or psychological wellbeing.
when discriminating between depressive severity levels (0.84, followed by emotional regulation (0.80, social integration (0.78, physical functioning and self-control (0.77, and mental functioning (0.73. Total SWN-K and its five subscales showed a significant linear trend against CDSS severity levels (P < 0.001.Conclusion: The presence of moderate to severe depressive symptoms was relatively high, and correlated inversely with patients’ subjective well-being. Routine assessment of patient-reported measures in patients with schizophrenia might reduce potential discrepancy between patient and physician assessment, increase therapeutic alliance, and improve outcome.Keywords: schizophrenia, subjective well-being, patient-reported outcome, depressive symptoms
Katz, Patricia; Pedro, Sofia; Michaud, Kaleb
The Patient-Reported Outcomes Measurement Information System (PROMIS) was developed to improve measurement of patient-reported outcomes. We examined performance of the 29-item PROMIS Profile (PROMIS-29) in persons with rheumatoid arthritis (RA), osteoarthritis (OA), fibromyalgia (FM), and systemic lupus erythematosus (SLE). Participants in the National Data Bank for Rheumatic Diseases completed the PROMIS-29, which includes 4-item forms for 7 PROMIS domains. Scales were scored and converted to T scores. Distributions of scale scores were examined, convergent and known-groups validity was tested, and differences in scores from online versus paper questionnaires were examined. Sample sizes were 4,346 for RA, 727 for OA, 241 for FM, and 240 for SLE. Participants were predominantly female, with a mean disease duration ≥20 years, and were ages ∼60 years. Large ceiling effects occurred for some PROMIS-29 scales. Correlations of PROMIS-29 scores with scales measuring similar constructs ranged from high to moderate for RA, OA, and SLE; correlations for FM were markedly lower for some scales. Consistent patterns of worsening PROMIS-29 scores with increasing disease severity or declining health status were observed. Differences in scores obtained by online versus paper questionnaires ranged from 0.3 to 2.2 points. Results provide guarded support for using the PROMIS-29 in these conditions. The PROMIS-29 4-item static forms appear to identify differences among levels of health and to measure constructs similar to those measured by legacy questionnaires. However, large ceiling effects suggest that measurement may be more precise at the "bad" ends of the scales, which may limit responsiveness, and differences by mode of administration appear to exist. © 2016, American College of Rheumatology.
Sprangers, M.A.G.; Sloan, J.A.; Barsevick, A.; Chauhan, C.; Dueck, A.C.; Raat, H.; Shi, Q.; van Noorden, C.J.F.
Objectives There is emerging evidence for a genetic basis of patient-reported quality-of-life (QOL) outcomes that can ultimately be incorporated into clinical research and practice. Objectives are (1) to provide arguments for the timeliness of investigating the genetic basis of QOL given the
Berg, Selina Kikkenborg; Svanholm, Jette; Lauberg, Astrid
as the time scale will be used to investigate associations between patient reported outcomes at baseline and morbidity/mortality, labour market affiliation and healthcare utilisation after 1 year. Ethics and dissemination: The study complies with the Declaration of Helsinki. The study has been approved...
A.P. Conijn (Anne P.); W. Jonkers (Wilma); E.V. Rouwet (Ellen); A. Vahl (Anco); J.A. Reekers (Jim); M.J. Koelemay
textabstractPurpose: The minimally important difference (MID) represents the smallest change in score on patient-reported outcome measures that is relevant to patients. The aim of this study was to introduce the MID for the Vascular Quality of Life Questionnaire (VascuQol) and the walking impairment
Wees, P.J. van der; Wammes, J.J.G.; Akkermans, R.P.; Koetsenruijter, J.; Westert, G.P.; Kampen, A. van; Hannink, G.J.; Waal Malefijt, M.C. de; Schreurs, B.W.
BACKGROUND: Patient-Reported Outcome (PRO) measurement is a method for measuring perceptions of patients on their health and quality of life. The aim of this paper is to present the results of PRO measurements in total hip and knee replacement as routinely collected during 20 years of surgery in a
Paulsen, Aksel; Overgaard, Søren; Lauritsen, Jens Martin
The clinical and scientific usage of patient-reported outcome measures is increasing in the health services. Often paper forms are used. Manual double entry of data is defined as the definitive gold standard for transferring data to an electronic format, but the process is laborious. Automated...
Jones, Jordan T; Carle, Adam C; Wootton, Janet; Liberio, Brianna; Lee, Jiha; Schanberg, Laura E; Ying, Jun; Morgan DeWitt, Esi; Brunner, Hermine I
To validate the pediatric Patient-Reported Outcomes Measurement Information System short forms (PROMIS-SFs) in childhood-onset systemic lupus erythematosus (SLE) in a clinical setting. At 3 study visits, childhood-onset SLE patients completed the PROMIS-SFs (anger, anxiety, depressive symptoms, fatigue, physical function-mobility, physical function-upper extremity, pain interference, and peer relationships) using the PROMIS assessment center, and health-related quality of life (HRQoL) legacy measures (Pediatric Quality of Life Inventory, Childhood Health Assessment Questionnaire, Simple Measure of Impact of Lupus Erythematosus in Youngsters [SMILEY], and visual analog scales [VAS] of pain and well-being). Physicians rated childhood-onset SLE activity on a VAS and completed the Systemic Lupus Erythematosus Disease Activity Index 2000. Using a global rating scale of change (GRC) between study visits, physicians rated change of childhood-onset SLE activity (GRC-MD1: better/same/worse) and change of patient overall health (GRC-MD2: better/same/worse). Questionnaire scores were compared in support of validity and responsiveness to change (external standards: GRC-MD1, GRC-MD2). In this population-based cohort (n = 100) with a mean age of 15.8 years (range 10-20 years), the PROMIS-SFs were completed in less than 5 minutes in a clinical setting. The PROMIS-SF scores correlated at least moderately (Pearson's r ≥ 0.5) with those of legacy HRQoL measures, except for the SMILEY. Measures of childhood-onset SLE activity did not correlate with the PROMIS-SFs. Responsiveness to change of the PROMIS-SFs was supported by path, mixed-model, and correlation analyses. To assess HRQoL in childhood-onset SLE, the PROMIS-SFs demonstrated feasibility, internal consistency, construct validity, and responsiveness to change in a clinical setting. © 2016, American College of Rheumatology.
Full Text Available To evaluate psychometric characteristics of a questionnaire (the Northwestern Ego-integrity Scale (NEIS on ego-integrity (the experience of wholeness and meaning in life, even in spite of negative experiences and despair (the experience of regret about the life one has led, and feelings of sadness, failure and hopelessness among cancer patients.Cancer patients (n = 164 completed patient reported outcome measures on ego-integrity and despair (NEIS, psychological distress, anxiety and depression (Hospital Anxiety and Depression Scale (HADS, and quality of life (EORTC QLQ-C30 (cancer survivors, n = 57 or EORTC QLQ-C15-PAL (advanced cancer patients, n = 107. Confirmatory Factor Analysis was used to assess construct validity. Cronbach's alpha was used to assess internal consistency. Convergent validity was tested based on a priori defined hypotheses: a higher level of ego-integrity was expected to be related to a higher level of quality of life, and lower levels of distress, depression and anxiety; a higher level of despair was expected to be related to a lower level of quality of life, and higher levels of distress, depression and anxiety.The majority of all items (94.5% of the NEIS were completed by patients and single item missing rate was below 2%. The two subscales, labeled as Ego-integrity (5 items and Despair (4 items had acceptable internal consistency (Cronbach's alpha .72 and .61, respectively. The Ego-integrity subscale was not significantly associated with quality of life, distress, anxiety, or depression. The Despair subscale correlated significantly (p <.001 with quality of life (r = -.29, distress (r = .44, anxiety (r = .47 and depression (r = .32.The NEIS has good psychometric characteristics to assess ego-integrity and despair among cancer patients.
Kochar, Bharati; Martin, Christopher F; Kappelman, Michael D; Spiegel, Brennan M; Chen, Wenli; Sandler, Robert S; Long, Millie D
Patient reported outcomes (PROs) are important treatment endpoints in inflammatory bowel diseases (IBD). We evaluated the gastrointestinal (GI) PRO Measurement Information System (PROMIS) in IBD subjects. Crohn's and Colitis Foundation of America's Partners is an Internet-based cohort of IBD subjects. Participants complete surveys, including demographics, disease characteristics, PROMIS domains, disease activity (short Crohn's disease activity index or simple clinical colitis activity index) and quality of life (QoL) indices. In a nested cross-sectional study, we used univariate and bivariate analyses to assess associations between 8 GI-PROMIS domains (reflux, swallowing, diarrhea, nausea, belly pain, gas, incontinence, and constipation) and QoL and disease activity indices. The study included 2,378 Crohn's Disease (CD) and 1,455 ulcerative colitis (UC) respondents with a median age of 41 years. Median disease duration was 11 years for CD subjects and 8 years for UC subjects; 57% of CD subjects and 42% of UC subjects were in remission. Among symptomatic CD subjects, those with active CD reported significantly worse symptoms on all 8 domains than those in remission. The same was observed for UC subjects with the exception of disrupted swallowing. IBD subjects with worse QoL reported significantly worse symptoms on all 8 domains compared to those with better QoL. In IBD subjects experiencing GI symptoms, GI-PROMIS domains were strongly associated with disease activity and QoL indices. GI-PROMIS holds potential as PRO measures in IBD and correlates with other validated indices in this population.Am J Gastroenterol advance online publication, 29 August 2017; doi:10.1038/ajg.2017.240.
Full Text Available Jean Endicott,1 Henrik Svedsäter,2 Julie C Locklear21Department of Psychiatry, Columbia University, New York, NY; 2AstraZeneca Pharmaceuticals, Wilmington, DE, USABackground: We evaluated the effects of once-daily extended-release quetiapine fumarate (quetiapine XR on patient-reported outcomes in generalized anxiety disorder (GAD.Methods: This is a report of a pooled analysis from three acute 8-week, randomized, placebo-controlled, fixed-dose (50, 150, 300 mg/day studies and a 52-week maintenance flexible dose (50–300 mg/day study of quetiapine XR monotherapy in patients with GAD. Quality of Life Enjoyment and Satisfaction Questionnaire Short Form (Q-LES-Q-SF percent maximum total scores (items 1–14, item 15 ("satisfaction with medication", item 16 ("overall life satisfaction", and Pittsburgh Sleep Quality Index (PSQI global scores are reported. Sheehan Disability Scale (SDS total scores were also assessed (maintenance study only.Results: The acute studies showed significant improvements at week 8 in Q-LES-Q-SF percent maximum total score with quetiapine XR 150 mg/day (P < 0.001 and item 16 with quetiapine XR 50 (P < 0.05 and 150 mg/day (P < 0.001 versus placebo; PSQI global scores significantly improved with quetiapine XR 50, 150, and 300 mg/day versus placebo (P < 0.001. The maintenance study showed significant benefits versus placebo with quetiapine XR 50–300 mg/day in Q-LES-Q-SF percent total score, item 15 and item 16 scores, PSQI global score, and SDS total score.Conclusion: Quetiapine XR 150 mg/day (acute studies and 50–300 mg/day (maintenance study improved quality of life, overall functioning, and sleep quality in patients with GAD.Keywords: atypical antipsychotic, anxiety disorders, quality of life, sleep quality, functioning, randomized studies
Kim, Man S; Koh, In J; Choi, Young J; Lee, Jong Y; In, Yong
The purpose of this study was to compare the patient-reported outcomes regarding joint awareness, function, and satisfaction after unicompartmental knee arthroplasty (UKA) and total knee arthroplasty (TKA). We identified all patients who underwent a UKA or TKA at our institution between September 2011 and March 2014, with a minimum follow-up of 2 years. Propensity score matching was performed for age, gender, body mass index, operation side, and the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) score. One hundred UKAs to 100 TKAs were matched. Each knee was evaluated according to the WOMAC score, Forgotten Joint Score (FJS), High Flexion Knee Score (HFKS) and patient's satisfaction at postoperative 2 years. There was no significant difference in WOMAC score at postoperative 2 years between UKA and TKA groups. However, the FJS of the UKA group was significantly higher than that of the TKA group (67.3 ± 19.8 and 60.6 ± 16.6, respectively; P = .011). The HFKS was also significantly higher in the UKA group compared with the TKA group (34.4 ± 6.4 and 31.3 ± 5.2, respectively; P < .001). Eighty-six percent of all patients who underwent UKA were satisfied compared with 71% of those who underwent TKA (P = .027). Patients who underwent UKA had higher FJS, HFKS, and satisfaction rate when compared with patients who underwent TKA, indicating that UKA facilitated less knee awareness and better function and satisfaction than TKA. Copyright © 2016 Elsevier Inc. All rights reserved.
Wolfe, Joanne; Orellana, Liliana; Ullrich, Christina; Cook, E. Francis; Kang, Tammy I.; Rosenberg, Abby; Geyer, Russ; Feudtner, Chris; Dussel, Veronica
Purpose Thousands of children are living with advanced cancer; yet patient-reported outcomes (PROs) have rarely been used to describe their experiences. We aimed to describe symptom distress in 104 children age 2 years or older with advanced cancer enrolled onto the Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQUEST) Study (multisite clinical trial evaluating an electronic PRO system). Methods Symptom data were collected using age- and respondent-adapted versions of the PediQUEST Memorial Symptom Assessment Scale (PQ-MSAS) at most once per week. Clinical and treatment data were obtained from medical records. Individual symptom scores were dichotomized into high/low distress. Determinants of PQ-MSAS scores were explored using linear mixed-effects models. Results During 9 months of follow-up, PQ-MSAS was administered 920 times: 459 times in teens (99% self-report), 249 times in children ages 7 to 12 years (96% child/parent report), and 212 times in those ages 2 to 6 years (parent reports). Common symptoms included pain (48%), fatigue (46%), drowsiness (39%), and irritability (37%); most scores indicated high distress. Among the 73 PQ-MSAS surveys administered in the last 12 weeks of life, pain was highly prevalent (62%; 58% with high distress). Being female, having a brain tumor, experiencing recent disease progression, and receiving moderate- or high-intensity cancer-directed therapy in the prior 10 days were associated with worse PQ-MSAS scores. In the final 12 weeks of life, receiving mild cancer-directed therapy was associated with improved psychological PQ-MSAS scores. Conclusion Children with advanced cancer experience high symptom distress. Strategies to promote intensive symptom management are indicated, especially with disease progression or administration of intensive treatments. PMID:25918277
Epis, Oscar Massimiliano; Casu, Cinzia; Belloli, Laura; Schito, Emanuela; Filippini, Davide; Muscarà, Marina; Gentile, Maria Giovanna; Venerelli, Chiara; Sonnati, Massimo; Schiavetti, Irene; Bruschi, Eleonora
Background In the management of chronic disease, new models for telemonitoring of patients combined with the choice of electronic patient-reported outcomes (ePRO) are being encouraged, with a clear improvement of both patients’ and parents’ quality of life. An Italian study demonstrated that ePRO were welcome in patients with rheumatoid arthritis (RA), with excellent matching data. Objective The aim of this study is to evaluate the level of agreement between electronic and paper-and-pencil questionnaire responses. Methods This is an observational prospective study. Patients were randomly assigned to first complete the questionnaire by paper and pencil and then by tablet or in the opposite order. The questionnaire consisted of 3 independent self-assessment visual rating scales (Visual Analog Scale, Global Health score, Patient Global Assessment of Disease Activity) commonly used in different adult patients, including those with rheumatic diseases. Results A total of 185 consecutive RA patients were admitted to hospital and were enrolled and completed the questionnaire both on paper and on electronic versions. For all the evaluated items, the intrarater degree of agreement between 2 approaches was found to be excellent (intraclass correlation coefficient>0.75, P<.001). Conclusions An electronic questionnaire is uploaded in a dedicated Web-based tool that could implement a telemonitoring system aimed at improving the follow-up of RA patients. High intrarater reliability between paper and electronic methods of data collection encourage the use of a new digital app with consequent benefit for the overall health care system. PMID:27852561
Full Text Available Abstract Background This retrospective study evaluated the impact of disease progression and of specific sites of metastasis on patient reported outcomes (PROs that assess symptom burden and health related quality of life (HRQoL in women with metastatic breast cancer (mBC. Methods HER-2 negative mBC patients (n = 102 were enrolled from 7 U.S. community oncology practices. Demographic, disease and treatment characteristics were abstracted from electronic medical records and linked to archived Patient Care Monitor (PCM assessments. The PCM is a self-report measure of symptom burden and HRQoL administered as part of routine care in participating practices. Linear mixed models were used to examine change in PCM scores over time. Results Mean age was 57 years, with 72% of patients Caucasian, and 25% African American. Median time from mBC diagnosis to first disease progression was 8.8 months. Metastasis to bone (60%, lung (28% and liver (26% predominated at initial metastatic diagnosis. Results showed that PCM items assessing fatigue, physical pain and trouble sleeping were sensitive to either general effects of disease progression or to effects associated with specific sites of metastasis. Progression of disease was also associated with modest but significant worsening of General Physical Symptoms, Treatment Side Effects, Acute Distress and Impaired Performance index scores. In addition, there were marked detrimental effects of liver metastasis on Treatment Side Effects, and of brain metastasis on Acute Distress. Conclusions Disease progression has a detrimental impact on cancer-related symptoms. Delaying disease progression may have a positive impact on patients' HRQoL.
Segmental mobility, disc height and patient-reported outcomes after surgery for degenerative disc disease: a prospective randomised trial comparing disc replacement and multidisciplinary rehabilitation.
Johnsen, L G; Brinckmann, P; Hellum, C; Rossvoll, I; Leivseth, G
This prospective multicentre study was undertaken to determine segmental movement, disc height and sagittal alignment after total disc replacement (TDR) in the lumbosacral spine and to assess the correlation of biomechanical properties to clinical outcomes.A total of 173 patients with degenerative disc disease and low back pain for more than one year were randomised to receive either TDR or multidisciplinary rehabilitation (MDR). Segmental movement in the sagittal plane and disc height were measured using distortion compensated roentgen analysis (DCRA) comparing radiographs in active flexion and extension. Correlation analysis between the range of movement or disc height and patient-reported outcomes was performed in both groups. After two years, no significant change in movement in the sagittal plane was found in segments with TDR or between the two treatment groups. It remained the same or increased slightly in untreated segments in the TDR group and in this group there was a significant increase in disc height in the operated segments. There was no correlation between segmental movement or disc height and patient-reported outcomes in either group.In this study, insertion of an intervertebral disc prosthesis TDR did not increase movement in the sagittal plane and segmental movement did not correlate with patient-reported outcomes. This suggests that in the lumbar spine the movement preserving properties of TDR are not major determinants of clinical outcomes.
Matthys, Heinrich; Lizogub, Victor G; Funk, Petra; Malek, Fathi A
Health-related quality of life (HRQL) and patient-reported outcome (PRO) have become important outcome parameters for the evaluation of medical treatment within clinical trials and, furthermore, to evaluate efficiency in clinical practice. We therefore report further exploratory results of an already reported dose-finding study with EPs 7630 tablets, now focussing on HRQL and PRO. A total of 406 adults with acute bronchitis were randomly assigned to one of four parallel treatment groups (placebo, 30 mg, 60 mg or 90 mg EPs 7630 daily). HRQL and PRO were assessed by questionnaires as secondary outcome measures at each study visit or daily in the patient's diary. At day 7, the patient-reported outcome measures were significantly more improved in all the three EPs 7630 groups compared to placebo (EQ-5D and EQ VAS, SF-12: physical score, impact of patient's sickness, duration of activity limitation, patient-reported treatment outcome, satisfaction with treatment). In conclusion, a statistically significant and clinically relevant improvement of HRQL/PRO compared to placebo was shown in all the three EPs 7630 groups.
何庆勇; 王阶; 张允岭; 王师菡; 杨戈
Development of clinical outcome rating scale based on patient-reported outcomes in traditional Chinese medicine has become the current the most active research areas in effect evaluation.But a key step of development of clinical outcome rating scale based on patient-reported outcomes is the item screening.Expert score method, distribution of entry method,discrete Trend method, factor analysis, cluster analysis, cronbach's a coefficient, stepwise regression, stepwise discriminant method, correlation coefficient, t-test, resolution coefficient method are systematic described in this paper.In order to screen items more comprehensively and objectively.%研制患者报告的临床结局评价量表已成为当前中医药疗效评价研究的最活跃领域之一.而研制患者报告的临床结局评价量表的关键一步是条目的筛选.本文较系统的叙述了专家重要评分法、条目分布考察法、离散趋势法、因子分析法、聚类分析法、Conbach's a系数法、逐步回归法、逐步判别法、相关系数法、t检验法、分辨力系数法等11种方法在患者报告的临床结局评价量表条目筛选中的应用方法,以求更好的全面而客观的筛选患者报告的临床结局评价量表的条目.
Seror, Raphaele; Ravaud, Philippe; Mariette, Xavier; Bootsma, Hendrika; Theander, Elke; Hansen, Arne; Ramos-Casals, Manel; Doerner, Thomas; Bombardieri, Stefano; Hachulla, Eric; Brun, Johan G.; Kruize, Aike A.; Praprotnik, Sonja; Tomsic, Matija; Gottenberg, Jacques-Eric; Devauchelle, Valerie; Devita, Salvatore; Vollenweider, Cristina; Mandl, Thomas; Tzioufas, Athanasios; Carsons, Steven; Saraux, Alain; Sutcliffe, Nurhan; Vitali, Claudio; Bowman, Simon J.
Objectives To develop a score for assessment of patients' symptoms in primary Sjogren's syndrome (SS): the EULAR SS Patient Reported Index (ESSPRI). Methods Dryness, pain, somatic and mental fatigue were identified as the main symptoms of patients with primary SS, in studies developing the Profile o
Shah, Hiral Anil; Dritsaki, Melina; Pink, Joshua; Petrou, Stavros
The aim of this study was to assess the psychometric properties of the EQ-5D-3 L, the SF-12 v2 and its preference based derivative the SF-6D, and the St Georges Respiratory Questionnaire (SGRQ), in patients diagnosed with Acute Respiratory Distress Syndrome (ARDS). Data from the Oscillation in ARDS (OSCAR) randomised unblinded clinical trial of 795 patients diagnosed with ARDS provided the foundation of this secondary psychometric analysis. The three source patient reported outcome measures (PROMs) (EQ-5D-3 L, SF-12 and SGRQ) were collected at both 6 and 12 months post randomisation. All measures were tested for acceptability, reliability, internal consistency, validity and responsiveness. Data from responders at 6 months was used to test for acceptability, reliability, known groups validity and internal responsiveness. Data from patients who responded at both 6 and 12 months was used to test for convergent validity and external responsiveness. Rates of response at both 6 and 12 months post randomisation were 89.88 % for the EQ-5D-3 L, 77.38 % for the SF-6D, 71.43 % for both the physical and mental components of the SF-12 and 38.10 % for the SGRQ. All measures had a Cronbach's Alpha statistic higher than 0.7. For known group's validity, there was no difference in mean summary or utility scores between known groups for all PROMs with minimal effect sizes. All three source measures showed strong convergent and discriminant validity. There was consistent evidence that the SF-6D is an empirically valid and efficient alternative to the EQ-5D-3 L. The EQ-5D-3 L and SGRQ were more responsive compared to the SF-12 and SF-6D with the EQ-5D-3 L generating greater effect sizes than the SGRQ. The PROMs explored in this study displayed varying psychometric properties in the context of ARDS. Further research should focus on shortening the SGRQ whilst still maintaining its psychometric properties and mapping between the SGRQ and preference-based measures for future application
Potter, Lori P; Mathias, Susan D; Raut, Monika; Kianifard, Farid; Tavakkol, Amir
Background This research was conducted to confirm the validity and reliability and to assess the responsiveness and clinical meaningfulness of the OnyCOE-t™, a questionnaire specifically designed to measure patient-reported outcomes (PRO) associated with toenail onychomycosis. Methods 504 patients with toenail onychomycosis randomized to receive 12 weeks of terbinafine 250 mg/day with or without target toenail debridement in the IRON-CLAD® trial completed the OnyCOE-t™ at baseline, weeks 6, 12, 24, and 48. The OnyCOE-t™ is composed of 6 multi-item scales and 1 single-item scale. These include a 7-item Toenail Symptom assessment, which comprises both Symptom Frequency and Symptom Bothersomeness scales; an 8-item Appearance Problems scale; a 7-item Physical Activities Problems scale; a 1-item Overall Problem scale; a 7-item Stigma scale; and a 3-item Treatment Satisfaction scale. In total, 33 toenail onychomycosis-specific items are included in the OnyCOE-t™. Clinical data, in particular the percent clearing of mycotic involvement in the target toenail, and OnyCOE-t™ responses were used to evaluate the questionnaire's reliability, validity, responsiveness, and the minimally clinical important difference (MCID). Results The OnyCOE-t™ was shown to be reliable and valid. Construct validity and known groups validity were acceptable. Internal consistency reliability of multi-item scales was demonstrated by Cronbach's alpha > .84. Responsiveness was good, with the Treatment Satisfaction, Symptom Frequency, Overall Problem, and Appearance Problem scales demonstrating the most responsiveness (Guyatt's statistic of 1.72, 1.31, 1.13, and 1.11, respectively). MCID was evaluated for three different clinical measures, and indicated that approximately an 8.5-point change (on a 0 to 100 scale) was clinically meaningful based on a 25% improvement in target nail clearing. Conclusion The OnyCOE-t™ questionnaire is a unique, toenail-specific PRO questionnaire that can be
Full Text Available Abstract Background This research was conducted to confirm the validity and reliability and to assess the responsiveness and clinical meaningfulness of the OnyCOE-t™, a questionnaire specifically designed to measure patient-reported outcomes (PRO associated with toenail onychomycosis. Methods 504 patients with toenail onychomycosis randomized to receive 12 weeks of terbinafine 250 mg/day with or without target toenail debridement in the IRON-CLAD® trial completed the OnyCOE-t™ at baseline, weeks 6, 12, 24, and 48. The OnyCOE-t™ is composed of 6 multi-item scales and 1 single-item scale. These include a 7-item Toenail Symptom assessment, which comprises both Symptom Frequency and Symptom Bothersomeness scales; an 8-item Appearance Problems scale; a 7-item Physical Activities Problems scale; a 1-item Overall Problem scale; a 7-item Stigma scale; and a 3-item Treatment Satisfaction scale. In total, 33 toenail onychomycosis-specific items are included in the OnyCOE-t™. Clinical data, in particular the percent clearing of mycotic involvement in the target toenail, and OnyCOE-t™ responses were used to evaluate the questionnaire's reliability, validity, responsiveness, and the minimally clinical important difference (MCID. Results The OnyCOE-t™ was shown to be reliable and valid. Construct validity and known groups validity were acceptable. Internal consistency reliability of multi-item scales was demonstrated by Cronbach's alpha > .84. Responsiveness was good, with the Treatment Satisfaction, Symptom Frequency, Overall Problem, and Appearance Problem scales demonstrating the most responsiveness (Guyatt's statistic of 1.72, 1.31, 1.13, and 1.11, respectively. MCID was evaluated for three different clinical measures, and indicated that approximately an 8.5-point change (on a 0 to 100 scale was clinically meaningful based on a 25% improvement in target nail clearing. Conclusion The OnyCOE-t™ questionnaire is a unique, toenail-specific PRO
Ray, Robbie; Goudie, Ewan B; Jenkins, Paul; Gaston, Paul
Resurfacing hip replacement has demonstrated good survival and outcomes for cohorts of younger male patients, but few controlled studies exist. In this study we compared patient reported outcome measures and satisfaction scores at one year following resurfacing hip replacement in 69 male patients with two control groups of equal numbers undergoing cemented total hip replacement: aged-matched patients and disease matched patients. At one year we found no difference in improvement in patient reported outcome measures between patients undergoing resurfacing hip replacement and disease matched patients, whereas patients undergoing resurfacing hip replacement had a statistically significant improvement in Oxford Hip Score compared to the age-matched controls (pResurfacing hip replacement and total hip replacement both confer increase in patient reported outcome scores and high patient satisfaction at one year. The results of this study will allow better counselling of patients and help inform treatment decisions.
Pirro, Ortensia; Mestak, Ondrej; Vindigni, Vincenzo; Sukop, Andrej; Hromadkova, Veronika; Nguyenova, Alena; Vitova, Lenka; Bassetto, Franco
Background: The demand for reconstructive breast procedures of various types has accelerated in recent years. Coupled with increased patient expectations, it has fostered the development of oncoplastic and reconstructive techniques in breast surgery. In the setting of postmastectomy reconstruction, patient satisfaction and quality of life are the most significant outcome variables when evaluating surgical success. The aim of this study was to evaluate the quality of life after implant breast ...
Full Text Available Objectives. To develop a prioritised list based on responsiveness for extracting patient-reported outcomes (PROs measuring pain and disability for performing meta-analyses in knee osteoarthritis (OA. Methods. A systematic search was conducted in 20 highest impact factor general and rheumatology journals chosen a priori. Eligible studies were randomised controlled trials, using two or more PROs measuring pain and/or disability. Results. A literature search identified 402 publications and 38 trials were included, resulting in 54 randomised comparisons. Thirty-five trials had sufficient data on pain and 15 trials on disability. The WOMAC “pain” and “function” subscales were the most responsive composite scores. The following list was developed. Pain: (1 WOMAC “pain” subscale, (2 pain during activity (VAS, (3 pain during walking (VAS, (4 general knee pain (VAS, (5 pain at rest (VAS, (6 other composite pain scales, and (7 other single item measures. Disability: (1 WOMAC “function” subscale, (2 SF-36 “physical function” subscale, (3 SF-36 (Physical composite score, and (4 Other composite disability scores. Conclusions. As choosing the PRO most favourable for the intervention from individual trials can lead to biased estimates, using a prioritised list as developed in this study is recommended to reduce risk of biased selection of PROs in meta-analyses.
Lamberts, Mark P.; Den Oudsten, Brenda L.; Keus, Frederik; De Vries, Jolanda; van Laarhoven, Cornelis J. H. M.; Westert, Gert P.; Drenth, Joost P. H.; Roukema, Jan A.
Up to 41 % of patients report pain after cholecystectomy and in most studies follow-up for these symptoms did not exceed 5 years. The episodic nature of abdominal pain associated with symptomatic cholelithiasis warrants long-term follow-up studies. We assessed which patient and surgical factors were
Chotai, Silky; Parker, Scott L; Sivaganesan, Ahilan; Sielatycki, J Alex; Asher, Anthony L; McGirt, Matthew J; Devin, Clinton J
OBJECT There is a paradigm shift toward rewarding providers for quality rather than volume. Complications appear to occur at a fairly consistent frequency in large aggregate data sets. Understanding how complications affect long-term patient-reported outcomes (PROs) following degenerative lumbar surgery is vital. The authors hypothesized that 90-day complications would adversely affect long-term PROs. METHODS Nine hundred six consecutive patients undergoing elective surgery for degenerative lumbar disease over a period of 4 years were enrolled into a prospective longitudinal registry. The following PROs were recorded at baseline and 12-month follow-up: Oswestry Disability Index (ODI) score, numeric rating scales for back and leg pain, quality of life (EQ-5D scores), general physical and mental health (SF-12 Physical Component Summary [PCS] and Mental Component Summary [MCS] scores) and responses to the North American Spine Society (NASS) satisfaction questionnaire. Previously published minimum clinically important difference (MCID) threshold were used to define meaningful improvement. Complications were divided into major (surgicalsite infection, hardware failure, new neurological deficit, pulmonary embolism, hematoma and myocardial infarction) and minor (urinary tract infection, pneumonia, and deep venous thrombosis). RESULTS Complications developed within 90 days of surgery in 13% (118) of the patients (major in 12%  and minor in 8% ). The mean improvement in ODI scores, EQ-5D scores, SF-12 PCS scores, and satisfaction at 3 months after surgery was significantly less in the patients with complications than in those who did not have major complications (ODI: 13.5 ± 21.2 vs 21.7 ± 19, lumbar spine surgery have significant impact on the short-term PROs. Patients with complications, however, do eventually achieve clinically meaningful outcomes and report satisfaction equivalent to those without major complications. This information allows a physician to
Drewes, Christina; Sagberg, Lisa Millgård; Jakola, Asgeir Store; Gulati, Sasha; Solheim, Ole
Published outcome reports in neurosurgical literature frequently rely on data from retrospective review of hospital records at discharge, but the sensitivity and specificity of retrospective assessments of surgical morbidity is not known. The aim of this study was to elucidate the sensitivity and specificity of retrospective assessment of morbidity after intracranial tumor surgery by comparing it to patient-reported outcomes at 30 days. In 191 patients who underwent surgery for the treatment of intracranial tumors, we evaluated newly acquired neurological deficits within the motor, language, and cognitive domains. Traditional retrospective discharge data were collected by review of hospital records. Patient-reported data were obtained by structured phone interviews at 30 days after surgery. Data on perioperative medical and surgical complications were obtained from both hospital records and patient interviews conducted 30 days postoperatively. Sensitivity values for retrospective review of hospital records as compared with patient-reported outcomes were 0.52 for motor deficits, 0.4 for language deficits, and 0.07 for cognitive deficits. According to medical records, 158 patients were discharged with no new or worsened deficits, but only 117 (74%) of these patients confirmed this at 30 days after surgery. Specificity values were high (0.97-0.99), indicating that new deficits were unlikely to be found by retrospective review of hospital records at discharge when the patients did not report any at 30 days. Major perioperative complications were all identified through retrospective review of hospital records. Retrospective assessment of medical records at discharge from hospital may greatly underestimate the incidence of new neurological deficits after brain tumor surgery when compared with patient-reported outcomes after 30 days.
Steven M. McPhail
Full Text Available Background. This paper aimed to identify condition-specific patient-reported outcome measures used in clinical trials among people with wrist osteoarthritis and summarise empirical peer-reviewed evidence supporting their reliability, validity, and responsiveness to change. Methods. A systematic review of randomised controlled trials among people with wrist osteoarthritis was undertaken. Studies reporting reliability, validity, or responsiveness were identified using a systematic reverse citation trail audit procedure. Psychometric properties of the instruments were examined against predefined criteria and summarised. Results. Thirteen clinical trials met inclusion criteria. The most common patient-reported outcome was the disabilities of the arm, shoulder, and hand questionnaire (DASH. The DASH, the Michigan Hand Outcomes Questionnaire (MHQ, the Patient Evaluation Measure (PEM, and the Patient-Reported Wrist Evaluation (PRWE had evidence supporting their reliability, validity, and responsiveness. A post-hoc review of excluded studies revealed the AUSCAN Osteoarthritis Hand Index as another suitable instrument that had favourable reliability, validity, and responsiveness. Conclusions. The DASH, MHQ, and AUSCAN Osteoarthritis Hand Index instruments were supported by the most favourable empirical evidence for validity, reliability, and responsiveness. The PEM and PRWE also had favourable empirical evidence reported for these elements. Further psychometric testing of these instruments among people with wrist osteoarthritis is warranted.
de Bock, Élodie; Hardouin, Jean-Benoit; Blanchin, Myriam; Le Neel, Tanguy; Kubis, Gildas; Bonnaud-Antignac, Angélique; Dantan, Étienne; Sébille, Véronique
The objective was to compare classical test theory and Rasch-family models derived from item response theory for the analysis of longitudinal patient-reported outcomes data with possibly informative intermittent missing items. A simulation study was performed in order to assess and compare the performance of classical test theory and Rasch model in terms of bias, control of the type I error and power of the test of time effect. The type I error was controlled for classical test theory and Rasch model whether data were complete or some items were missing. Both methods were unbiased and displayed similar power with complete data. When items were missing, Rasch model remained unbiased and displayed higher power than classical test theory. Rasch model performed better than the classical test theory approach regarding the analysis of longitudinal patient-reported outcomes with possibly informative intermittent missing items mainly for power. This study highlights the interest of Rasch-based models in clinical research and epidemiology for the analysis of incomplete patient-reported outcomes data.
Pirro, Ortensia; Vindigni, Vincenzo; Sukop, Andrej; Hromadkova, Veronika; Nguyenova, Alena; Vitova, Lenka; Bassetto, Franco
Background: The demand for reconstructive breast procedures of various types has accelerated in recent years. Coupled with increased patient expectations, it has fostered the development of oncoplastic and reconstructive techniques in breast surgery. In the setting of postmastectomy reconstruction, patient satisfaction and quality of life are the most significant outcome variables when evaluating surgical success. The aim of this study was to evaluate the quality of life after implant breast reconstruction compared with autologous breast reconstruction. Materials and Methods: A cross-sectional study design was used. A total of 65 women who had completed postmastectomy implant-based or autologous reconstruction in the participating center were asked to complete the BREAST-Q (Reconstruction Module). Results: Data analysis demonstrated that women with autologous breast reconstruction were significantly more satisfied with their breasts (P = 0.0003) and with the overall outcome (P = 0.0001) compared with women with implant breast reconstruction. All other BREAST-Q parameters that were considered and observed were not significantly different between the 2 patient groups. Conclusions: Through statistical analysis, our results showed that patients who underwent autologous tissue reconstruction had better satisfaction with the reconstructed breast and the outcome, while both techniques appear to equally improve psychosocial well-being, sexual well-being, and chest satisfaction. PMID:28203513
Pirro, Ortensia; Mestak, Ondrej; Vindigni, Vincenzo; Sukop, Andrej; Hromadkova, Veronika; Nguyenova, Alena; Vitova, Lenka; Bassetto, Franco
The demand for reconstructive breast procedures of various types has accelerated in recent years. Coupled with increased patient expectations, it has fostered the development of oncoplastic and reconstructive techniques in breast surgery. In the setting of postmastectomy reconstruction, patient satisfaction and quality of life are the most significant outcome variables when evaluating surgical success. The aim of this study was to evaluate the quality of life after implant breast reconstruction compared with autologous breast reconstruction. A cross-sectional study design was used. A total of 65 women who had completed postmastectomy implant-based or autologous reconstruction in the participating center were asked to complete the BREAST-Q (Reconstruction Module). Data analysis demonstrated that women with autologous breast reconstruction were significantly more satisfied with their breasts (P = 0.0003) and with the overall outcome (P = 0.0001) compared with women with implant breast reconstruction. All other BREAST-Q parameters that were considered and observed were not significantly different between the 2 patient groups. Through statistical analysis, our results showed that patients who underwent autologous tissue reconstruction had better satisfaction with the reconstructed breast and the outcome, while both techniques appear to equally improve psychosocial well-being, sexual well-being, and chest satisfaction.
Maria J Santana
Full Text Available Maria J Santana1, David Feeny2, Justin Weinkauf1, Roland Nador1, Ali Kapasi1, Kathleen Jackson1, Marianne Schafenacker1, Dalyce Zuk1, Dale Lien11Lung Transplant Program, University of Alberta Hospital, Edmonton, Alberta, Canada; 2The Center for Health Research, Kaiser Permanente Northwest, Portland, OR, USAObjective: To assess the use of patient-reported outcome (PROs measures in the routine clinical care of lung–heart transplant patients. We assessed whether the addition of PROs in routine clinical care affected the duration of the consultation and patient’s and clinician’s views.Method: Consecutive lung–heart transplant patients visiting the outpatient clinic, University of Alberta Hospital, completed the Chronic Respiratory Questionnaire (CRQ and the Health Utilities Index (HUI on touchscreen computers. Information on the patient’s responses was made available to the members of the transplant team prior to the encounter with the patient. The duration of clinical encounters was noted. At the end of every visit, clinicians completed a questionnaire on the usefulness of having PRO information available. After 6 months patients completed a survey of their experiences.Results: The final patient sample consisted of 172 patients with a mean (SD age of 52 (13.3 years old; 47% were female; 68% were organ recipients and 32% candidates. The transplant team, comprising four pulmunologists, two nurses, and one pharmacist had an average of 9 years of practical experience in pulmunology. The mean duration of patient–clinician encounters in minutes was 15.15 (4.52. Ninety-eight percent of patients indicated that they would be happy to complete the CRQ and HUI at every clinic visit. Ninety-one percent of the assessments completed by clinicians showed complete satisfaction with the use of PROs in routine practice. Further, the clinicians developed guidelines for the use of PRO information in clinical practice.Conclusions: The incorporation of PRO
Jeanne A. Teresi
Full Text Available This is the first study of the measurement equivalence of the Patient Reported Outcomes Measurement Information System® (PROMIS® Anxiety short forms in a large ethnically diverse sample. The psychometric properties and differential item functioning (DIF were examined across different racial/ethnic, educational, age, gender and language groups. Methods: These data are from individuals selected from cancer registries in the United States. For the analyses of race/ethnicity the reference group was non-Hispanic Whites (n = 2,263, the studied groups were non-Hispanic Blacks (n = 1,117, Hispanics (n = 1,043 and Asians/Pacific Islanders (n = 907. Within the Hispanic subsample, there were 335 interviews conducted in Spanish and 703 in English. The 11 anxiety items were from the PROMIS emotional disturbance item bank. DIF hypotheses were generated by content experts who rated whether or not they expected DIF to be present, and the direction of the DIF with respect to several comparison groups. The primary method used for DIF detection was the Wald test for examination of group differences in item response theory (IRT item parameters accompanied by magnitude measures. Expected item scores were examined as measures of magnitude. The method used for quantification of the difference in the average expected item scores was the non-compensatory DIF (NCDIF index. DIF impact was examined using expected scale score functions. Additionally, precision and reliabilities were examined using several methods. Results: Although not hypothesized to show DIF for Asians/Pacific Islanders, every item evidenced DIF by at least one method. Two items showed DIF of higher magnitude for Asians/Pacific Islanders vs. Whites: “Many situations made me worry” and “I felt anxious”. However, the magnitude of DIF was small and the NCDIF statistics were not above threshold. The impact of DIF was negligible. For education, six items were identified with consistent DIF across
Gilbert, Alexandra, E-mail: email@example.com [Leeds Institute of Cancer & Pathology, University of Leeds, Leeds (United Kingdom); Ziegler, Lucy; Martland, Maisie [Leeds Institute of Cancer & Pathology, University of Leeds, Leeds (United Kingdom); Davidson, Susan [The Christie Hospital, Manchester (United Kingdom); Efficace, Fabio [Italian Group for Adult Hematologic Diseases, Rome (Italy); Sebag-Montefiore, David; Velikova, Galina [Leeds Institute of Cancer & Pathology, University of Leeds, Leeds (United Kingdom)
The use of multimodal treatments for rectal cancer has improved cancer-related outcomes but makes monitoring toxicity challenging. Optimizing future radiation therapy regimens requires collection and publication of detailed toxicity data. This review evaluated the quality of toxicity information provided in randomized controlled trials (RCTs) of radiation therapy in rectal cancer and focused on the difference between clinician-reported and patient-reported toxicity. Medline, EMBASE, and the Cochrane Library were searched (January 1995-July 2013) for RCTs reporting late toxicity in patients treated with regimens including preoperative (chemo)radiation therapy. Data on toxicity measures and information on toxicity reported were extracted using Quantitative Analyses of Normal Tissue Effects in the Clinic recommendations. International Society for Quality of Life Research standards on patient-reported outcomes (PROs) were used to evaluate the quality of patient-reported toxicity. Twenty-one RCT publications met inclusion criteria out of 4144 articles screened. All PRO studies reported higher rates of toxicity symptoms than clinician-reported studies and reported on a wider range and milder symptoms. No clinician-reported study published data on sexual dysfunction. Of the clinician-reported studies, 55% grouped toxicity data related to an organ system together (eg “Bowel”), and 45% presented data only on more-severe (grade ≥3) toxicity. In comparison, all toxicity grades were reported in 79% of PRO publications, and all studies (100%) presented individual symptom toxicity data (eg bowel urgency). However, PRO reporting quality was variable. Only 43% of PRO studies presented baseline data, 28% did not use any psychometrically validated instruments, and only 29% of studies described statistical methods for managing missing data. Analysis of these trials highlights the lack of reporting standards for adverse events and reveals the differences between clinician and
Vaz Fragoso, Carlos A; Murphy, Terrence E; Agogo, George O; Allore, Heather G; McAvay, Gail J
Background Prior work suggests that asthma–COPD overlap syndrome (ACOS) has a greater health burden than asthma alone or COPD alone. In the current study, we have further evaluated the health burden of ACOS in a nationally representative sample of the US population, focusing on patient-reported outcomes and health care utilization and on comparisons with asthma alone and COPD alone. Patient-reported outcomes are especially meaningful, as these include functional activities that are highly valued by patients and are the basis for patient-centered care. Methods Using data from the Medical Expenditure Panel Survey (MEPS), we evaluated patient-reported outcomes and health care utilization among participants who were aged 40–85 years and had self-reported, physician-diagnosed asthma or COPD. MEPS administered five rounds of interviews, at baseline and approximately every 6 months over 2.5 years. Patient-reported outcomes included activities of daily living (ADLs), mobility, social/recreational activities, disability days in bed, and health status (Short Form 12, Version 2). Health care utilization included outpatient and emergency department (ED) visits, and hospitalization. Results Of 3,486 participants with asthma or COPD, 1,585 (45.4%) had asthma alone, 1,294 (37.1%) had COPD alone, and 607 (17.4%) had ACOS. Relative to asthma alone, ACOS was significantly associated with higher odds of prevalent disability in ADLs and limitations in mobility and social/recreational activities (adjusted odds ratios [adjORs]: 1.91–3.98), as well as with higher odds of incident limitations in mobility and social/recreational activities, disability days in bed, and respiratory-based outpatient and ED visits, and hospitalization (adjORs: 1.86–2.35). In addition, ACOS had significantly worse physical and mental health scores than asthma alone (P-values social/recreational activities (adjORs: 1.68–2.06), as well as with higher odds of incident disability days in bed and
Alves, Flávio Sérgio Marques; Pinto, Rogério de Melo Costa; Mendonça, Tânia Maria Silva; Silva, Carlos Henrique Martins da
The items bank of the Fatigue domain is part of an American system developed for evaluation of results reported by patients, called Patient-Reported-Outcomes Measurement Information System (PROMIS). This study aimed to translate and cross-culturally adapt this item bank for the Brazilian population, as a promising new tool for evaluating health-related quality of life. The items in this bank were translated using rigorous translation and back-translation protocols. The translated version was pre-tested in twenty Brazilians with a brief cognitive and retrospective interview in order to test the items' conceptual, cultural, and semantic equivalences. In the translation and back-translation process, only three of the 82 items had to be reworded due to the culturally inadequate content. In the pretest, only four items needed to be reworded, but without conceptual and semantic alterations. The results showed that the translated version of this item bank is conceptually, culturally, and semantically equivalent to the original version.
Bennett, Casey; Bragg, April; Luellen, Jason; Van Regenmorter, Christina; Lockman, Jennifer; Reiserer, Randall; 10.1109/HISB.2011.20
The CDOI outcome measure - a patient-reported outcome (PRO) instrument utilizing direct client feedback - was implemented in a large, real-world behavioral healthcare setting in order to evaluate previous findings from smaller controlled studies. PROs provide an alternative window into treatment effectiveness based on client perception and facilitate detection of problems/symptoms for which there is no discernible measure (e.g. pain). The principal focus of the study was to evaluate the utility of the CDOI for predictive modeling of outcomes in a live clinical setting. Implementation factors were also addressed within the framework of the Theory of Planned Behavior by linking adoption rates to implementation practices and clinician perceptions. The results showed that the CDOI does contain significant capacity to predict outcome delta over time based on baseline and early change scores in a large, real-world clinical setting, as suggested in previous research. The implementation analysis revealed a number of ...
Lucas, Steven M; Kim, Tae-Kyung; Ghani, Khurshid R; Miller, David C; Linsell, Susan; Starr, Jay; Peabody, James O; Hurley, Patrick; Montie, James; Cher, Michael L
To report on the establishment of a unified, electronic patient-reported outcome (PRO) infrastructure and pilot results from the first 5 practices enrolled in the web-based collection system developed by the Michigan Urological Surgery Improvement Collaborative. Eligible patients were those undergoing radical prostatectomy of 5 academic and community practices. PRO was obtained using a validated 21-item web-based questionnaire, regarding urinary function, erection function, and sexual interest and satisfaction. Data were collected preoperatively, at 3 months, and 6 months postoperatively. Patients were provided a link via email to complete the surveys. Perioperative and PRO data were analyzed as reports for individual patients and summary performance reports for individual surgeons. Among 773 eligible patients, 688 (89%) were enrolled preoperatively. Survey completion rate was 88%, 84%, and 90% preoperatively, at 3 months, and 6 months. Electronic completion rates preoperatively, at 3 months, and 6 months were 70%, 70%, and 68%, respectively. Mean urinary function scores were 18.3, 14.3, and 16.6 (good function ≥ 17), whereas mean erection scores were 18.7, 7.3, and 9.1 (good erection score ≥ 22) before surgery, at 3 months, and 6 months. Variation was noted for erectile function among the practices. Collection of electronic PRO via this unified, web-based format was successful and provided results that reflect expected recovery and identify opportunities for improvement. This will be extended to more practices statewide to improve outcomes after radical prostatectomy. Copyright © 2017 Elsevier Inc. All rights reserved.
Cheung, Peter P; Lahiri, Manjari; March, Lyn; Gossec, Laure
Patient-reported outcomes (PROs) such as the Europe-developed Rheumatoid Arthritis Impact of Disease (RAID) are important to assess patients' quality of life. Their interpretation may be culture-dependent. To evaluate the potential utility of RAID in multiethnic Asian rheumatoid arthritis (RA) patients. Cross-sectional study of English-speaking RA patients in a Singapore tertiary center. Validity of RAID (scored between 0 and 10 with higher score indicating worse status) was assessed by Spearman's correlation with patient global assessment (PGA), DAS28 and short form 12 (SF-12). Consistency was assessed by Cronbach's alpha and test-retest reliability by intra-class correlation coefficient (ICC) 7 days after (n = 20). Feasibility was assessed by % of missing data. Eighty-two patients were analyzed: median age 53 years (Q1:Q3 44.7; 60.7), disease duration 4.2 years (1.4; 8.8), 66 (81%) women and 54 (66%) Chinese. Although RA was moderately active (median DAS28, 3.2 (2.5; 4.3)), RAID score was very low (median, 1.9 (0.6; 3.7)) with 44 (53.7%) patients having RAID score between 0 and 2. RAID was strongly correlated with PGA (r = 0.75), and moderately with other outcomes (DAS28 r = 0.46, SF12 physical r = -0.45 and SF12 mental r = -0.52, p impact of RA in Asia. Multiethnic Asian patients may underestimate the impact of their disease compared to European patients.
Tucker, Carole A; Cieza, Alarcos; Riley, Anne W; Stucki, Gerold; Lai, Jin Shei; Bedirhan Ustun, T; Kostanjsek, Nenad; Riley, William; Cella, David; Forrest, Christopher B
The Patient Reported Outcomes Measurement Information System (PROMIS (®) ) is a US National Institutes of Health initiative that has produced self-report outcome measures, using a framework of physical, mental, and social health defined by the World Health Organization in 1948 (WHO, in Preamble to the Constitution of the World Health Organization as adopted by the International Health Conference, New York, 1948). The World Health Organization's International Classification of Functioning, Disability and Health (ICF) is a comprehensive classification system of health and health-related domains that was put forward in 2001. The purpose of this report is to compare and contrast PROMIS and ICF conceptual frameworks to support mapping of PROMIS instruments to the ICF classification system . We assessed the objectives and the classification schema of the PROMIS and ICF frameworks, followed by content analysis to determine whether PROMIS domain and sub-domain level health concepts can be linked to the ICF classification. Both PROMIS and ICF are relevant to all individuals, irrespective of the presence of health conditions, person characteristics, or environmental factors in which persons live. PROMIS measures are intended to assess a person's experiences of his or her health, functional status, and well-being in multiple domains across physical, mental, and social dimensions. The ICF comprehensively describes human functioning from a biological, individual, and social perspective. The ICF supports classification of health and health-related states such as functioning, but is not a specific measure or assessment of health, per se. PROMIS domains and sub-domain concepts can be meaningfully mapped to ICF concepts. Theoretical and conceptual similarities support the use of PROMIS instruments to operationalize self-reported measurement for many body function, activity and participation ICF concepts, as well as several environmental factor concepts. Differences observed in
and devices better tailored to patients’ needs that could enhance clinical outcomes and reduce MS costs. Understanding the factors underlying satisfaction and compliance with treatment and patients’ preference for certain therapies could help in the development of strategies that can improve adherence.Keywords: multiple sclerosis, adherence, satisfaction, delivery devices, costs
The patient reported outcomes following initial treatment and long term evaluation of survivorship registry: scope, rationale and design of an infrastructure for the study of physical and psychosocial outcomes in cancer survivorship cohorts
van de Poll-Franse, L.V.; Horevoorts, N.; van Eenbergen, M.; Denollet, J.; Roukema, J.A.; Aaronson, N.K.; Vingerhoets, A.; Coebergh, J.W.; de Vries, J.; Essink-Bot, M.L.; Mols, F.
‘Patient Reported Outcomes Following Initial treatment and Long term Evaluation of Survivorship (PROFILES)’ is a registry for the study of the physical and psychosocial impact of cancer and its treatment from a dynamic, growing population-based cohort of both short and long-term cancer survivors. PR
Mayo, Benjamin C; Massel, Dustin H; Bohl, Daniel D; Narain, Ankur S; Hijji, Fady Y; Long, William W; Modi, Krishna D; Basques, Bryce A; Yacob, Alem; Singh, Kern
OBJECTIVE Prior studies have correlated preoperative depression and poor mental health status with inferior patient-reported outcomes following lumbar spinal procedures. However, literature regarding the effect of mental health on outcomes following cervical spinal surgery is limited. As such, the purpose of this study is to test for the association of preoperative SF-12 Mental Component Summary (MCS) scores with improvements in Neck Disability Index (NDI), SF-12 Physical Component Summary (PCS), and neck and arm pain following anterior cervical discectomy and fusion (ACDF). METHODS A prospectively maintained surgical database of patients who underwent a primary 1- or 2-level ACDF during 2014-2015 was reviewed. Patients were excluded if they did not have complete patient-reported outcome data for the preoperative or 6-week, 12-week, or 6-month postoperative visits. At baseline, preoperative SF-12 MCS score was assessed for association with preoperative NDI, neck visual analog scale (VAS) score, arm VAS score, and SF-12 PCS score. The preoperative MCS score was then tested for association with changes in NDI, neck VAS, arm VAS, and SF-12 PCS scores from the preoperative visit to postoperative visits. These tests were conducted using multivariate regression controlling for baseline characteristics as well as for the preoperative score for the patient-reported outcome being assessed. RESULTS A total of 52 patients were included in the analysis. At baseline, a higher preoperative MCS score was negatively associated with a lower preoperative NDI (coefficient: -0.74, p 0.05 for each). The percentage of patients achieving a minimum clinically important difference at 6 months did not differ between the bottom and top MCS score halves (p > 0.05 for each). CONCLUSIONS The results of this study suggest that better preoperative mental health status is associated with lower perceived preoperative disability but is not associated with severity of preoperative neck or arm pain
Lamberts, Mark P; Den Oudsten, Brenda L; Keus, Frederik; De Vries, Jolanda; van Laarhoven, Cornelis J H M; Westert, Gert P; Drenth, Joost P H; Roukema, Jan A
Up to 41% of patients report pain after cholecystectomy and in most studies follow-up for these symptoms did not exceed 5 years. The episodic nature of abdominal pain associated with symptomatic cholelithiasis warrants long-term follow-up studies. We assessed which patient and surgical factors were associated with absence of pain and patient-reported success of surgery after ≥ 5 years of follow-up. Patients of ≥ 18 years of age with symptomatic cholelithiasis, classified as ASA I or II, who had previously returned a preoperative questionnaire were sent a questionnaire consisting of the gastrointestinal quality of life index (GIQLI) and patient ratings of current versus presurgical abdominal symptoms and of surgery result. Logistic regression analysis was performed to determine associations. Questionnaires were sent to 197 patients and returned by 126 (64.0%) patients (73.8 % female, mean age at surgery 47.5 ± 12.2 years) at a mean of 10.0 ± 1.0 years after cholecystectomy. Absence of abdominal pain was reported by 60.3% of the patients. Patients classified as ASA II as opposed to ASA I were less likely to report absence of pain (OR 0.41, 95% CI 0.17-0.99). A positive rating of long-term postsurgical versus presurgical abdominal symptoms was given by 89.7% of the patients and 90.5% considered the cholecystectomy result to be good. No variables were significantly associated with these latter two outcome measures. We found a high patient-reported surgery success rate after >5 years of follow-up after cholecystectomy despite residual abdominal pain in some of these patients. None of the patient and surgery-related characteristics were consistently associated with all three outcome measures. This discrepancy between patient' outcomes highlights the need for realistic expectations prior to cholecystectomy.
H. Felix Fischer
Full Text Available Abstract Background Recently, a growing number of Item-Response Theory (IRT models has been published, which allow estimation of a common latent variable from data derived by different Patient Reported Outcomes (PROs. When using data from different PROs, direct estimation of the latent variable has some advantages over the use of sum score conversion tables. It requires substantial proficiency in the field of psychometrics to fit such models using contemporary IRT software. We developed a web application ( http://www.common-metrics.org , which allows estimation of latent variable scores more easily using IRT models calibrating different measures on instrument independent scales. Results Currently, the application allows estimation using six different IRT models for Depression, Anxiety, and Physical Function. Based on published item parameters, users of the application can directly estimate latent trait estimates using expected a posteriori (EAP for sum scores as well as for specific response patterns, Bayes modal (MAP, Weighted likelihood estimation (WLE and Maximum likelihood (ML methods and under three different prior distributions. The obtained estimates can be downloaded and analyzed using standard statistical software. Conclusions This application enhances the usability of IRT modeling for researchers by allowing comparison of the latent trait estimates over different PROs, such as the Patient Health Questionnaire Depression (PHQ-9 and Anxiety (GAD-7 scales, the Center of Epidemiologic Studies Depression Scale (CES-D, the Beck Depression Inventory (BDI, PROMIS Anxiety and Depression Short Forms and others. Advantages of this approach include comparability of data derived with different measures and tolerance against missing values. The validity of the underlying models needs to be investigated in the future.
Fischer, H Felix; Rose, Matthias
Recently, a growing number of Item-Response Theory (IRT) models has been published, which allow estimation of a common latent variable from data derived by different Patient Reported Outcomes (PROs). When using data from different PROs, direct estimation of the latent variable has some advantages over the use of sum score conversion tables. It requires substantial proficiency in the field of psychometrics to fit such models using contemporary IRT software. We developed a web application ( http://www.common-metrics.org ), which allows estimation of latent variable scores more easily using IRT models calibrating different measures on instrument independent scales. Currently, the application allows estimation using six different IRT models for Depression, Anxiety, and Physical Function. Based on published item parameters, users of the application can directly estimate latent trait estimates using expected a posteriori (EAP) for sum scores as well as for specific response patterns, Bayes modal (MAP), Weighted likelihood estimation (WLE) and Maximum likelihood (ML) methods and under three different prior distributions. The obtained estimates can be downloaded and analyzed using standard statistical software. This application enhances the usability of IRT modeling for researchers by allowing comparison of the latent trait estimates over different PROs, such as the Patient Health Questionnaire Depression (PHQ-9) and Anxiety (GAD-7) scales, the Center of Epidemiologic Studies Depression Scale (CES-D), the Beck Depression Inventory (BDI), PROMIS Anxiety and Depression Short Forms and others. Advantages of this approach include comparability of data derived with different measures and tolerance against missing values. The validity of the underlying models needs to be investigated in the future.
Full Text Available Abstract Background Patient reported outcome measures (PROMs are self-report measures of health status increasingly promoted for use in healthcare quality improvement. However people with low literacy skills or learning disabilities may find PROMs hard to complete. Our study investigated stakeholder views on the accessibility and use of PROMs to develop suggestions for more inclusive practice. Methods Taking PROMs recommended for chronic obstructive pulmonary disease (COPD as an example, we conducted 8 interviews with people with low literacy skills and/or learning disabilities, and 4 focus groups with 20 health professionals and people with COPD. Discussions covered the format and delivery of PROMs using the EQ-5D and St George Respiratory Questionnaire as prompts. Thematic framework analysis focused on three main themes: Accessibility, Ease of Use, and Contextual factors. Results Accessibility included issues concerning the questionnaire format, and suggestions for improvement included larger font sizes and more white space. Ease of Use included discussion about PROMs’ administration. While health professionals suggested PROMs could be completed in waiting rooms, patients preferred settings with more privacy and where they could access help from people they know. Contextual Factors included other challenges and wider issues associated with completing PROMs. While health professionals highlighted difficulties created by the system in managing patients with low literacy/learning disabilities, patient participants stressed that understanding the purpose of PROMs was important to reduce intimidation. Conclusions Adjusting PROMs’ format, giving an explicit choice of where patients can complete them, and clearly conveying PROMs’ purpose and benefit to patients may help to prevent inequality when using PROMs in health services.
Santana, Maria J; Feeny, David; Weinkauf, Justin; Nador, Roland; Kapasi, Ali; Jackson, Kathleen; Schafenacker, Marianne; Zuk, Dalyce; Lien, Dale
Objective: To assess the use of patient-reported outcome (PROs) measures in the routine clinical care of lung–heart transplant patients. We assessed whether the addition of PROs in routine clinical care affected the duration of the consultation and patient’s and clinician’s views. Method: Consecutive lung–heart transplant patients visiting the outpatient clinic, University of Alberta Hospital, completed the Chronic Respiratory Questionnaire (CRQ) and the Health Utilities Index (HUI) on touchscreen computers. Information on the patient’s responses was made available to the members of the transplant team prior to the encounter with the patient. The duration of clinical encounters was noted. At the end of every visit, clinicians completed a questionnaire on the usefulness of having PRO information available. After 6 months patients completed a survey of their experiences. Results: The final patient sample consisted of 172 patients with a mean (SD) age of 52 (13.3) years old; 47% were female; 68% were organ recipients and 32% candidates. The transplant team, comprising four pulmunologists, two nurses, and one pharmacist had an average of 9 years of practical experience in pulmunology. The mean duration of patient–clinician encounters in minutes was 15.15 (4.52). Ninety-eight percent of patients indicated that they would be happy to complete the CRQ and HUI at every clinic visit. Ninety-one percent of the assessments completed by clinicians showed complete satisfaction with the use of PROs in routine practice. Further, the clinicians developed guidelines for the use of PRO information in clinical practice. Conclusions: The incorporation of PRO measures in the routine clinical care of lung–heart transplant patients resulted in a reduction of the duration of patient–clinician encounters. The experience was well accepted by patients and clinicians. We conclude that the routine use of PROs in lung–heart transplant patients has become standard practice
Pilkonis, Paul A; Yu, Lan; Dodds, Nathan E; Johnston, Kelly L; Maihoefer, Catherine C; Lawrence, Suzanne M
The Patient-Reported Outcomes Measurement Information System (PROMIS) is an NIH Roadmap initiative devoted to developing better measurement tools for assessing constructs relevant to the clinical investigation and treatment of all diseases-constructs such as pain, fatigue, emotional distress, sleep, physical functioning, and social participation. Following creation of item banks for these constructs, our priority has been to validate them, most often in short-term observational studies. We report here on a three-month prospective observational study with depressed outpatients in the early stages of a new treatment episode (with assessments at intake, one-month follow-up, and three-month follow-up). The protocol was designed to compare the psychometric properties of the PROMIS depression item bank (administered as a computerized adaptive test, CAT) with two legacy self-report instruments: the Center for Epidemiological Studies Depression scale (CESD; Radloff, 1977) and the Patient Health Questionnaire (PHQ-9; Spitzer et al., 1999). PROMIS depression demonstrated strong convergent validity with the CESD and the PHQ-9 (with correlations in a range from .72 to .84 across all time points), as well as responsiveness to change when characterizing symptom severity in a clinical outpatient sample. Identification of patients as "recovered" varied across the measures, with the PHQ-9 being the most conservative. The use of calibrations based on models from item response theory (IRT) provides advantages for PROMIS depression both psychometrically (creating the possibility of adaptive testing, providing a broader effective range of measurement, and generating greater precision) and practically (these psychometric advantages can be achieved with fewer items-a median of 4 items administered by CAT-resulting in less patient burden).
Vallance, Jeff K; Buman, Matthew P; Lynch, Brigid M; Boyle, Terry
The purpose of this study was to examine potential effects of reallocating time between sleep, sedentary and active behaviours on fatigue symptoms and quality of life in a sample of non-Hodgkin lymphoma survivors. Non-Hodgkin lymphoma survivors identified from the Western Australian Cancer Registry (N = 149) (response rate = 36%; median age = 64 years) wore an Actigraph® GT3X+ accelerometer for 7 days and completed the Fatigue Scale, the Functional Assessment of Cancer Therapy-General and the Pittsburgh Sleep Quality Index. We used isotemporal substitution methods in linear regression models to examine the potential effects of reallocating time between sleep, sedentary and activity behaviours on fatigue and quality of life. Data collection was conducted in Western Australia in 2013. Significant differences were observed for fatigue symptoms when 30 min per day of bouted moderate-to-vigorous physical activity (10 min) was reallocated from 30 min per day of sleep (5.7 points, 95% CI = 1.8, 9.7), sedentary time bouts (20 min) (5.7 points, 95% CI = 1.6, 9.7), sedentary time non-bouts (5.1 points, 95% CI = 1.0, 9.3) or light intensity activity (5.5 points, 95% CI = 1.5, 9.5). Isotemporal substitution effects of reallocating sedentary time, sleep and light physical activity with bouted physical activity was significantly associated with fatigue, but not quality of life. Findings from the present study may aid in the development and delivery of health behaviour interventions that are more likely to influence the health outcome of interest.
El Miedany, Yasser; El Gaafary, Maha; Youssef, Sally; Ahmed, Ihab
Objectives. To assess the validity, reliability, and responsiveness to change of a patient self-reported questionnaire combining the Widespread Pain Index and the Symptom Severity Score as well as construct outcome measures and comorbidities assessment in fibromyalgia patients. Methods. The PROMs-FM was conceptualized based on frameworks used by the WHO Quality of Life tool and the PROMIS. Initially, cognitive interviews were conducted to identify item pool of questions. Item selection and reduction were achieved based on patients as well as an interdisciplinary group of specialists. Rasch and internal consistency reliability analyses were implemented. The questionnaire included the modified ACR criteria main items (Symptom Severity Score and Widespread Pain Index), in addition to assessment of functional disability, quality of life (QoL), review of the systems, and comorbidities. Every patient completed HAQ and EQ-5D questionnaires. Results. A total of 146 fibromyalgia patients completed the questionnaire. The PROMs-FM questionnaire was reliable as demonstrated by a high standardized alpha (0.886-0.982). Content construct assessment of the functional disability and QoL revealed significant correlation (p < 0.01) with both HAQ and EQ-5D. Changes in functional disability and QoL showed significant (p < 0.01) variation with diseases activity status in response to therapy. There was higher prevalence of autonomic symptoms, CVS risk, sexual dysfunction, and falling. Conclusions. The developed PROMs-FM questionnaire is a reliable and valid instrument for assessment of fibromyalgia patients. A phased treatment regimen depending on the severity of FMS as well as preferences and comorbidities of the patient is the best approach to tailored patient management.
Edward J Mills
Full Text Available BACKGROUND: Adherence to highly active antiretroviral therapy (HAART medication is the greatest patient-enabled predictor of treatment success and mortality for those who have access to drugs. We systematically reviewed the literature to determine patient-reported barriers and facilitators to adhering to antiretroviral therapy. METHODS AND FINDINGS: We examined both developed and developing nations. We searched the following databases: AMED (inception to June 2005, Campbell Collaboration (inception to June 2005, CinAhl (inception to June 2005, Cochrane Library (inception to June 2005, Embase (inception to June 2005, ERIC (inception to June 2005, MedLine (inception to June 2005, and NHS EED (inception to June 2005. We retrieved studies conducted in both developed and developing nation settings that examined barriers and facilitators addressing adherence. Both qualitative and quantitative studies were included. We independently, in duplicate, extracted data reported in qualitative studies addressing adherence. We then examined all quantitative studies addressing barriers and facilitators noted from the qualitative studies. In order to place the findings of the qualitative studies in a generalizable context, we meta-analyzed the surveys to determine a best estimate of the overall prevalence of issues. We included 37 qualitative studies and 47 studies using a quantitative methodology (surveys. Seventy-two studies (35 qualitative were conducted in developed nations, while the remaining 12 (two qualitative were conducted in developing nations. Important barriers reported in both economic settings included fear of disclosure, concomitant substance abuse, forgetfulness, suspicions of treatment, regimens that are too complicated, number of pills required, decreased quality of life, work and family responsibilities, falling asleep, and access to medication. Important facilitators reported by patients in developed nation settings included having a sense of
Rosenlund, Signe; Holsgaard-Larsen, Anders; Overgaard, Søren;
BACKGROUND: The Gait Deviation Index summarizes overall gait 'quality', based on kinematic data from a 3-dimensional gait analysis. However, it is unknown which clinical outcomes may affect the Gait Deviation Index in patients with primary hip osteoarthritis. The aim of this study......) and with severe primary hip osteoarthritis underwent 3-dimensional gait analysis. Mean Gait Deviation Index, pain after walking and maximal isometric hip muscle strength (flexor, extensor, and abductor) were recorded. All patients completed the 'Physical Function Short-form of the Hip disability...... was to investigate associations between Gait Deviation Index as a measure of gait 'quality' and hip muscle strength and between Gait Deviation Index and patient-reported outcomes in patients with primary hip osteoarthritis. METHOD: Forty-seven patients (34 males), aged 61.1 ± 6.7 years, with BMI 27.3 ± 3.4 (kg/m2...
Rosenlund, Signe; Holsgaard-Larsen, Anders; Overgaard, Søren
was to investigate associations between Gait Deviation Index as a measure of gait 'quality' and hip muscle strength and between Gait Deviation Index and patient-reported outcomes in patients with primary hip osteoarthritis. METHOD: Forty-seven patients (34 males), aged 61.1 ± 6.7 years, with BMI 27.3 ± 3.4 (kg/m2......BACKGROUND: The Gait Deviation Index summarizes overall gait 'quality', based on kinematic data from a 3-dimensional gait analysis. However, it is unknown which clinical outcomes may affect the Gait Deviation Index in patients with primary hip osteoarthritis. The aim of this study......) and with severe primary hip osteoarthritis underwent 3-dimensional gait analysis. Mean Gait Deviation Index, pain after walking and maximal isometric hip muscle strength (flexor, extensor, and abductor) were recorded. All patients completed the 'Physical Function Short-form of the Hip disability...
刘砚燕; 姚静静; 陈如男; 袁长蓉
Patient-reported outcomes (PROs) are defined as a measurement of any aspect of a patient's health status that comes directly from the patient.And it has been used in the measurement of health-related quality of life,being endpoints of clinical trials,monitoring of adverse event and approval of new drugs and so on.Research on patient-reported outcomes tools has been the focus at home and abroad.The Patient Reported Outcomes Measurement Information System (PROMIS) is funded by NIH,has been developing by using modern test theory,advanced administration and scoring technology.In this article,it summarized research process,content and development tendency in future of PROMIS,to provide some suggestions for domestic related research of PROs.%患者报告结局是指直接来自患者的有关患者健康状态的任何方面的信息,广泛应用于健康相关生活质量、临床试验、不良事件监测及批准药物等.有关患者报告结局工具的研究一直是国内外研究热点,PROMIS由美国健康国立医学中心牵头,运用现代测量理论、先进的测评方式及计分方法研制开发.本文将对PROMIS的研制过程、研究内容及未来发展趋势加以总结,以期对我国患者报告结局的相关研究提供借鉴.
Vaz Fragoso CA
Full Text Available Carlos A Vaz Fragoso,1,2 Terrence E Murphy,1 George O Agogo,1 Heather G Allore,1,3 Gail J McAvay1 1Department of Medicine, Yale School of Medicine, New Haven, 2Veterans Affairs Clinical Epidemiology Research Center, West Haven, 3Department of Biostatistics, Yale School of Public Health, New Haven, CT, USA Background: Prior work suggests that asthma–COPD overlap syndrome (ACOS has a greater health burden than asthma alone or COPD alone. In the current study, we have further evaluated the health burden of ACOS in a nationally representative sample of the US population, focusing on patient-reported outcomes and health care utilization and on comparisons with asthma alone and COPD alone. Patient-reported outcomes are especially meaningful, as these include functional activities that are highly valued by patients and are the basis for patient-centered care.Methods: Using data from the Medical Expenditure Panel Survey (MEPS, we evaluated patient-reported outcomes and health care utilization among participants who were aged 40–85 years and had self-reported, physician-diagnosed asthma or COPD. MEPS administered five rounds of interviews, at baseline and approximately every 6 months over 2.5 years. Patient-reported outcomes included activities of daily living (ADLs, mobility, social/recreational activities, disability days in bed, and health status (Short Form 12, Version 2. Health care utilization included outpatient and emergency department (ED visits, and hospitalization.Results: Of 3,486 participants with asthma or COPD, 1,585 (45.4% had asthma alone, 1,294 (37.1% had COPD alone, and 607 (17.4% had ACOS. Relative to asthma alone, ACOS was significantly associated with higher odds of prevalent disability in ADLs and limitations in mobility and social/recreational activities (adjusted odds ratios [adjORs]: 1.91–3.98, as well as with higher odds of incident limitations in mobility and social/recreational activities, disability days in bed, and
Nissen, Nina; Douw, Karla; Overgaard, Søren
The purpose of this study was to investigate patientreported outcome in terms of satisfaction in two study groups that had undergone hip resurfacing arthro-plasty (HRA) or total hip replacement (THR). The procedure consists of placing a hollow, mushroom-shaped metal cap over the femoral head while...
Nissen, Tina Koerner; Douw, Karla; Overgaard, Søren
The purpose of this study was to investigate patientreported outcome in terms of satisfaction in two study groups that had undergone hip resurfacing arthro-plasty (HRA) or total hip replacement (THR). The procedure consists of placing a hollow, mushroom-shaped metal cap over the femoral head while...
Full Text Available Kei Ogawa,1 Shinji Fujikoshi,2 William Montgomery,3 Levent Alev1 1Medical Science, 2Statistical Science, Eli Lilly Japan K.K., Kobe, Japan; 3Global Patient Outcomes and Real World Evidence, Eli Lilly Australia Pty Ltd, West Ryde, NSW, Australia Objective: We assessed whether quality of life (QoL improvement in duloxetine-treated patients with diabetic peripheral neuropathic pain (DPNP correlates with the extent of pain relief.Methods: Pooled data from three multicountry, double-blind, 12-week, placebo-controlled trials of duloxetine-treated (duloxetine 60 mg once daily; total number =335 patients with DPNP were analyzed. Based on improvement in 24-hour average pain scores, patients were stratified into four groups. Improvement in QoL, which was measured as the change from baseline in two patient-reported health outcome measures (Short Form [SF]-36 and five-dimension version of the EuroQol Questionnaire [EQ-5D], was evaluated and compared among the four groups. Pearson’s correlation coefficient was calculated to assess the correlation between improvement in pain scores and improvement in QoL.Results: The group with more pain improvement generally showed greater mean change from baseline in all of the SF-36 scale scores and on the EQ-5D index. Pearson’s correlation coefficients ranged from 0.114 to 0.401 for the SF-36 scale scores (P<0.05, and it was 0.271 for the EQ-5D (P<0.001.Conclusion: Improvement in pain scores was positively correlated with improvement in QoL and patient-reported outcomes in duloxetine-treated patients. Keywords: diabetic peripheral neuropathic pain, duloxetine, efficacy, function, quality of life
Fiore, Julio F; Figueiredo, Sabrina; Balvardi, Saba; Lee, Lawrence; Nauche, Bénédicte; Landry, Tara; Mayo, Nancy E; Feldman, Liane S
To appraise the level of evidence supporting the measurement properties of patient-reported outcome measures (PROMs) in the context of postoperative recovery after abdominal surgery. There is growing interest in using PROMs to support value-based care in abdominal surgery; however, to draw valid conclusions regarding patient-reported outcomes data, PROMs with robust measurement properties are required. Eight databases (MEDLINE, EMBASE, Biosis, PsycINFO, The Cochrane Library, CINAHL, Scopus, Web of Science) were searched for studies focused on the measurement properties of PROMs in the context of recovery after abdominal surgery. The methodological quality of individual studies was evaluated using the consensus-based COSMIN checklist. Evidence supporting the measurement properties of each PROM was synthetized according to standardized criteria and compared against the International Society of Quality of Life Research minimum standards for the selection of PROMs for outcomes research. We identified 35 studies evaluating 22 PROMs [12 focused on nonspecific surgical populations (55%), 4 focused on abdominal surgery (18%), and 6 generic PROMs (27%)]. The great majority of the studies (74%) received only poor or fair quality ratings. Measurement properties of PROMs were predominantly supported by limited or unknown evidence. None of the PROMs fulfilled International Society of Quality of Life Research's minimum standards, hindering specific recommendations. There is very limited evidence supporting the measurement properties of existing PROMs used in the context of recovery after abdominal surgery. This precludes the use of these PROMs to support value-based surgical care. Further research is required to bridge this major knowledge gap. International Prospective Register of Systematic Reviews (PROSPERO): CRD42014014349.
Full Text Available Background: Efforts have been made to measure integration in health care delivery, but few existing instruments have adopted a patient perspective, and none is sufficiently generic and brief for administration at scale. We sought to develop a brief and generic patient-reported measure of integration in health care delivery.Methods: Drawing on both existing conceptualisations of integrated care and research on patients’ perspectives, we chose to focus on four distinct domains of integration: information sharing,consistent advice, mutual respect and role clarity. We formulated candidate items and conducted cognitive interviews with end users to further develop and refine the items. We then pilot-tested the measure.Results: Four rounds of cognitive interviews were conducted (n = 14 and resulted in a four-item measure that was both relevant and understandable to end users. The pilot administration of the measure (n = 15 further confirmed the relevance and interpretability of items and demonstrated that the measure could be completed in less than one minute.Conclusions: This new measure, IntegRATE, represents a patient-reported measure of integration in health care delivery that is conducive to use in both routine performance monitoring and research. The psychometric properties of the measure will be assessed in the next stage of development.
Makino, Takahiro; Kaito, Takashi; Fujiwara, Hiroyasu; Honda, Hirotsugu; Sakai, Yusuke; Takenaka, Shota; Yoshikawa, Hideki; Yonenobu, Kazuo
A retrospective review of prospectively collected data. This study aimed to identify risk factors for poor patient-reported quality of life (QOL) outcomes, based on five categories (pain-related disorders, lumbar spine dysfunction, gait disturbance, social life dysfunction, and psychological disorders) of the Japanese Orthopaedic Association Back Pain Evaluation Questionnaire (JOABPEQ), after posterior lumbar interbody fusion (PLIF) at the two-year follow-up. Many studies have been reported on patient QOL outcomes after lumbar surgery; however, few reports have focused on risk factors for poor postoperative QOL outcomes in terms of the various disabilities and dysfunctions after PLIF. One hundred consecutive patients (39 men and 61 women; mean age 69.6 [44-84] years) who underwent single- or two-level PLIF for degenerative spondylolisthesis and/or foraminal stenosis with a two-year follow-up were included. The effectiveness of surgery in each category of the JOABPEQ was evaluated. Demographic and clinical data and radiographic parameters were reviewed. Risk factors for poor postoperative QOL outcomes in each category of the JOABPEQ were investigated by multivariate logistic regression analysis. Older age and spinopelvic malalignment (preoperative high pelvic tilt or postoperative decrease in lumbar lordosis [= postoperative increase in the mismatch between pelvic incidence and lumbar lordosis]) were risk factors for poor postoperative QOL outcomes in all categories of the JOABPEQ, except for lumbar spine dysfunction. In contrast, increase in number of PLIF segments, non-union, and radiographic adjacent segment degeneration (ASD) were risk factors for poor postoperative QOL outcomes in lumbar spine dysfunction and gait disturbance. The risk factors for poor QOL outcomes after PLIF differed among the five categories of the JOABPEQ. In particular, surgery-related factors (e.g., increase in number of PLIF segments, non-union, and radiographic ASD) had a negative impact
Hans, Parminder K; Gray, Carolyn Steele; Gill, Ashlinder; Tiessen, James
Aim This qualitative study investigates how the Electronic Patient-Reported Outcome (ePRO) mobile application and portal system, designed to capture patient-reported measures to support self-management, affected primary care provider workflows. The Canadian health system is facing an ageing population that is living with chronic disease. Disruptive innovations like mobile health technologies can help to support health system transformation needed to better meet the multifaceted needs of the complex care patient. However, there are challenges with implementing these technologies in primary care settings, in particular the effect on primary care provider workflows. Over a six-week period interdisciplinary primary care providers (n=6) and their complex care patients (n=12), used the ePRO mobile application and portal to collaboratively goal-set, manage care plans, and support self-management using patient-reported measures. Secondary thematic analysis of focus groups, training sessions, and issue tracker reports captured user experiences at a Toronto area Family Health Team from October 2014 to January 2015. Findings Key issues raised by providers included: liability concerns associated with remote monitoring, increased documentation activities due to a lack of interoperability between the app and the electronic patient record, increased provider anxiety with regard to the potential for the app to disrupt and infringe upon appointment time, and increased demands for patient engagement. Primary care providers reported the app helped to focus care plans and to begin a collaborative conversation on goal-setting. However, throughout our investigation we found a high level of provider resistance evidenced by consistent attempts to shift the app towards fitting with existing workflows rather than adapting much of their behaviour. As health systems seek innovative and disruptive models to better serve this complex patient population, provider change resistance will need to
Andikyan, Vaagn; Rezk, Youssef; Einstein, M Heather; Gualtiere, Gina; Leitao, Mario M; Sonoda, Yukio; Abu-Rustum, Nadeem R; Barakat, Richard R; Basch, Ethan M; Chi, Dennis S
The purposes of this study are to evaluate the feasibility of capturing patient-reported outcomes (PROs) electronically and to identify the most common distressing symptoms in women recovering from major gynecologic cancer surgery. This was a prospective, single-arm pilot study. Eligible participants included those scheduled for a laparotomy for presumed or known gynecologic malignancy. Patients completed a Web-based "STAR" (Symptom Tracking and Reporting for Patients) questionnaire once preoperatively and weekly during the 6-week postoperative period. The questionnaire consisted of the patient adaptation of the NCI CTCAE 3.0 and EORTC QLQ-C30 3.0. When a patient submitted a response that was concerning, an automated email alert was sent to the clinician. The patient's assessment of STAR's usefulness was measured via an exit survey. Forty-nine patients completed the study. The procedures included the following: hysterectomy±staging (67%), resection of tumor (22%), salpingo-oophorectomy (6%), and other (4%). Most patients (82%) completed at least 4 sessions in STAR. The CTC generated 43 alerts. These alerts resulted in 25 telephone contacts with patients, 2 ER referrals, one new appointment, and one pharmaceutical prescription. The 3 most common patient-reported symptoms generating an alert were as follows: poor performance status (19%), nausea (18%), and fatigue (17%). Most patients found STAR useful (80%) and would recommend it to others (85%). Application of a Web-based, electronic STAR system is feasible in the postoperative period, highly accepted by patients, and warrants further study. Poor performance status, nausea, and fatigue were the most common distressing patient-reported symptoms. Copyright © 2012 Elsevier Inc. All rights reserved.
What is the value of the routine use of patient-reported outcome measures toward improvement of patient outcomes, processes of care, and health service outcomes in cancer care? A systematic review of controlled trials.
Kotronoulas, Grigorios; Kearney, Nora; Maguire, Roma; Harrow, Alison; Di Domenico, David; Croy, Suzanne; MacGillivray, Stephen
The systematic use of patient-reported outcome measures (PROMs) has been advocated as an effective way to standardize cancer practice. Yet, the question of whether PROMs can lead to actual improvements in the quality of patient care remains under debate. This review examined whether inclusion of PROM in routine clinical practice is associated with improvements in patient outcomes, processes of care, and health service outcomes during active anticancer treatment. A systematic review of five electronic databases (Medline, EMBASE, CINAHL [Cumulative Index to Nursing and Allied Health Literature], PsycINFO, and Psychology and Behavioral Sciences Collection [PBSC]) was conducted from database inception to May 2012 to locate randomized and nonrandomized controlled trials of patients receiving active anticancer treatment or supportive care irrespective of type of cancer. Based on prespecified eligibility criteria, we included 26 articles that reported on 24 unique controlled trials. Wide variability in the design and use of interventions delivered, outcomes evaluated, and cancer- and modality-specific context was apparent. Health service outcomes were only scarcely included as end points. Overall, the number of statistically significant findings were limited and PROMs' intervention effect sizes were predominantly small-to-moderate. The routine use of PROMs increases the frequency of discussion of patient outcomes during consultations. In some studies, PROMs are associated with improved symptom control, increased supportive care measures, and patient satisfaction. Additional effort is required to ensure patient adherence, as well as additional support to clinicians who will respond to patient concerns and issues, with clear system guidelines in place to guide their responses. More research is required to support PROM cost-benefit in terms of patient safety, clinician burden, and health services usage.
Morena, Marion; Jaussent, Audrey; Chalabi, Lotfi; Leray-Moragues, Hélène; Chenine, Leila; Debure, Alain; Thibaudin, Damien; Azzouz, Lynda; Patrier, Laure; Maurice, Francois; Nicoud, Philippe; Durand, Claude; Seigneuric, Bruno; Dupuy, Anne-Marie; Picot, Marie-Christine; Cristol, Jean-Paul; Canaud, Bernard
Large cohort studies suggest that high convective volumes associated with online hemodiafiltration may reduce the risk of mortality/morbidity compared to optimal high-flux hemodialysis. By contrast, intradialytic tolerance is not well studied. The aim of the FRENCHIE (French Convective versus Hemodialysis in Elderly) study was to compare high-flux hemodialysis and online hemodiafiltration in terms of intradialytic tolerance. In this prospective, open-label randomized controlled trial, 381 elderly chronic hemodialysis patients (over age 65) were randomly assigned in a one-to-one ratio to either high-flux hemodialysis or online hemodiafiltration. The primary outcome was intradialytic tolerance (day 30-day 120). Secondary outcomes included health-related quality of life, cardiovascular risk biomarkers, morbidity, and mortality. During the observational period for intradialytic tolerance, 85% and 84% of patients in high-flux hemodialysis and online hemodiafiltration arms, respectively, experienced at least one adverse event without significant difference between groups. As exploratory analysis, intradialytic tolerance was also studied, considering the sessions as a statistical unit according to treatment actually received. Over a total of 11,981 sessions, 2,935 were complicated by the occurrence of at least one adverse event, with a significantly lower occurrence in online hemodiafiltration with fewer episodes of intradialytic symptomatic hypotension and muscle cramps. By contrast, health-related quality of life, morbidity, and mortality were not different in both groups. An improvement in the control of metabolic bone disease biomarkers and β2-microglobulin level without change in serum albumin concentration was observed with online hemodiafiltration. Thus, overall outcomes favor online hemodiafiltration over high-flux hemodialysis in the elderly.
Imam, Mohamed A; Barke, Samuel; Stafford, Giles H; Parkin, David; Field, Richard E
Patient reported outcome measures (PROMs) are used to gauge clinical performance. The PROMs outcome programme at our centre achieves a preoperative data capture rate of 99%. This falls to 90.6%, 89%, 83% and 79% at the six-week, six-month, one-year and two-year time points, respectively. The study aims were to determine factors associated with patients who did not respond to outcome questionnaires following total hip replacement (THR), and the potential implications this may have when assessing patients following THRs. During the first year of the PROMs programme, 1,322 patients underwent unilateral primary THR at our institution. Of these, 1,311 completed preoperative questionnaires. Thirty-eight patients (2.9%) died within two years of surgery and have been excluded. For the remaining 1,273 patients, we identified those who did not return postoperative questionnaires at each of our review time points. Younger age, lower baseline EQ5D and Oxford Hip scores (OHS) were significantly associated with non-response (prespond to subsequent questionnaires. A significant association between non-response and deprivation (presponded. This phenomenon may apply to studies where those categorised as "lost to follow-up" represent a subset of patients who have disengaged due to poor outcome or satisfaction.
Jeanne A. Teresi
Full Text Available The articles in this two-part series of Psychological Test and Assessment Modeling describe the psychometric performance and measurement equivalence of the Patient Reported Outcomes Measurement Information System® (PROMIS® short form measures in ethnically, socio-demographically diverse groups of cancer patients. Measures in eight health-related quality of life domains were evaluated: fatigue, depression, anxiety, cognition, pain, sleep, and physical and social function. State-of-the-art latent variable methods, most based on item response theory, and described in two methods overview articles in this series were used to examine differential item functioning (DIF. Findings were generally supportive of the performance of the PROMIS measures. Although use of powerful methods and large samples resulted in the identification of many items with DIF, practi-cally none were identified with high magnitude. The aggregate level impact of DIF was small, and minimal individual impact was detected. Some methodological challenges were encountered in-volving positively and negatively worded items, but most were resolved through modest item removal. Sensitivity analyses showed minimal impact of model assumption violation on the results presented. A cautionary note is the observance of a few instances of individual-level impact of DIF in the analyses of depression, anxiety, and pain, and one instance of aggregate level impact just below threshold in the analyses of physical function. Although this sample of over 5,000 individuals was diverse, ethnically, a limitation was the lack of ability to examine language groups other than Spanish and English and specific ethnic subgroups within Hispanic, Asian/Pacific Islander, and Black subsamples. Extensive qualitative and quantitative analyses were performed in the development of PROMIS item banks. These sets of analyses, performed by several teams of psychometricians, statisticians, and qualitative experts, were the
Nixon, Annabel; Kerr, Cicely; Breheny, Katie; Wild, Diane
Despite collection of patient reported outcome (PRO) data in clinical trials of antiepileptic drugs (AEDs), PRO results are not being routinely reported on European Medicines Agency (EMA) and Food and Drug Administration (FDA) product labels. This review aimed to evaluate epilepsy-specific PRO instruments against FDA regulatory standards for supporting label claims. Structured literature searches were conducted in Embase and Medline databases to identify epilepsy-specific PRO instruments. Only instruments that could potentially be impacted by pharmacological treatment, were completed by adults and had evidence of some validation work were selected for review. A total of 26 PROs were reviewed based on criteria developed from the FDA regulatory standards. The ability to meet these criteria was classified as either full, partial or no evidence, whereby partial reflected some evidence but not enough to comprehensively address the FDA regulatory standards. Most instruments provided partial evidence of content validity. Input from clinicians and literature was common although few involved patients in both item generation and cognitive debriefing. Construct validity was predominantly compromised by no evidence of a-priori hypotheses of expected relationships. Evidence for test-retest reliability and internal consistency was available for most PROs although few included complete results regarding all subscales and some failed to reach recommended thresholds. The ability to detect change and interpretation of change were not investigated in most instruments and no PROs had published evidence of a conceptual framework. The study concludes that none of the 26 have the full evidence required by the FDA to support a label claim, and all require further research to support their use as an endpoint. The Subjective Handicap of Epilepsy (SHE) and the Neurological Disorders Depression Inventory for Epilepsy (NDDI-E) have the fewest gaps that would need to be addressed through
Bryce B. Reeve
Full Text Available Measurement equivalence across differing socio-demographic groups is essential for valid assessment. This is one of two issues of Psychological Test and Assessment Modeling that contains articles describing methods and substantive findings related to establishing measurement equivalence in self-reported health, mental health and social functioning measures. The articles in this two part series describe analyses of items assessing eight domains: fatigue, depression, anxiety, sleep, pain, physical function, cognitive concerns and social function. Additionally, two overview articles describe the methods and sample characteristics of the data set used in these analyses. An additional article describes the important topic of assessing magnitude and impact of differential item functioning. These articles provide the first strong evidence supporting the measurement equivalence of the Patient Reported Outcomes Measurement Information System® (PROMIS® short form measures in ethnically, socio-demographically diverse groups, and is a beginning step in meeting the international call for further study of their performance in such groups.
Ageberg, Eva; Nilsdotter, Anna; Kosek, Eva
The benefits of exercise in mild and moderate knee or hip osteoarthritis (OA) are apparent, but the evidence in severe OA is less clear. We recently reported that neuromuscular training was well tolerated and feasible in patients with severe primary hip or knee OA. The aims of this controlled bef...... before-and-after study were to compare baseline status to an age-matched population-based reference group and to examine the effects of neuromuscular training on patient-reported outcomes and physical function in patients with severe primary OA of the hip or knee.......The benefits of exercise in mild and moderate knee or hip osteoarthritis (OA) are apparent, but the evidence in severe OA is less clear. We recently reported that neuromuscular training was well tolerated and feasible in patients with severe primary hip or knee OA. The aims of this controlled...
Castro, Natália Fontes Caputo de; Rezende, Carlos Henrique Alves de; Mendonça, Tânia Maria da Silva; Silva, Carlos Henrique Martins da; Pinto, Rogério de Melo Costa
The Patient-Reported Outcome Measurement Information System (PROMIS), structured in Itens Banks, provides a new tool for evaluating results that apply to various chronic diseases through advanced statistical techniques (TRI) and computerized adaptive testing (CAT). The aim of this study was to culturally adapt the Items Banks of Anxiety and Depression of PROMIS to the Portuguese language. The process followed the recommendations of PROMIS through the advanced translation, reconciliation, back-translation, FACIT review, independent review, finalization, pre-test, and incorporation of the results from the pre-test. The translated version was pre-tested in ten patients, and items 3, 46, and 53 of the Bank of Anxiety and item 46 of the bank of Depression had to be changed. Changes affected equivalence of meaning, and the final version was consistent with the Brazilian population's linguistic and cultural skills. In conclusion, for the Brazilian population the translated version proved semantically and conceptually equivalent to the original.
Rosenlund, Signe; Broeng, Leif; Jensen, Carsten
BACKGROUND: Total hip replacement provides pain relief and improves physical function and quality of life in patients with end-stage hip osteoarthritis. The incidence of hip replacement operations is expected to increase due to the growing elderly population. Overall, the posterior approach...... and lateral approach are the two most commonly used approaches for hip replacement operations. The posterior approach is associated with an increased risk of revision due to dislocations, and some studies have shown that the lateral approach is associated with reduced patient-reported outcomes, including...... is a prospective, double blinded, parallel-group controlled trial with balanced randomisation [1: 1]. Patients with hip osteoarthritis scheduled for hip replacement surgery, aged 45-70 years, will be consecutively recruited and randomised into two groups. Group A will receive hip replacement using the posterior...
Kaat, Aaron J; Schalet, Benjamin D; Rutsohn, Joshua; Jensen, Roxanne E; Cella, David
Measuring patient-reported outcomes (PROs) is becoming an integral component of quality improvement initiatives, clinical care, and research studies in cancer, including comparative effectiveness research. However, the number of PROs limits comparability across studies. Herein, the authors attempted to link the Functional Assessment of Cancer Therapy-General Physical Well-Being (FACT-G PWB) subscale with the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function (PF) calibrated item bank. The also sought to augment a subset of the conceptually most similar FACT-G PWB items with PROMIS PF items to improve the linking. Baseline data from 5506 participants in the Measuring Your Health (MY-Health) study were used to identify the optimal items for linking FACT-G PWB with PROMIS PF. A mixed methods approach identified the optimal items for creating the 5-item FACT/PROMIS-PF5 scale. Both the linked and augmented relationships were cross-validated using the follow-up MY-Health data. A 5-item FACT-G PWB item subset was found to be optimal for linking with PROMIS PF. In addition, a 2-item subset, including only items that were conceptually very similar to the PROMIS item bank content, were augmented with 3 PROMIS PF items. This new FACT/PROMIS-PF5 provided superior score recovery. The PROMIS PF metric allows for the evaluation of the extent to which similar questionnaires can be linked and therefore expressed on the same metric. These results allow for the aggregation of existing data and provide an optimal measure for future studies wishing to use the FACT yet also report on the PROMIS PF metric. Cancer 2017. © 2017 American Cancer Society. © 2017 American Cancer Society.
Richard D O'Connor
Full Text Available Richard D O'ConnorSharp Rees-Stealy Medical Group, San Diego, CA, USAAbstract: In the United States, budesonide/formoterol pressurized metered-dose inhaler (pMDI is approved for treatment of asthma in patients aged ≥12 years whose asthma is not adequately controlled with an inhaled corticosteroid (ICS or whose disease severity clearly warrants treatment with an ICS and a long-acting β2-adrenergic agonist. This article reviews studies of budesonide/formoterol pMDI in patients with persistent asthma, with a particular focus on patient-reported outcomes (eg, perceived onset of effect, patient satisfaction with treatment, health-related quality of life [HRQL], global assessments, sleep quality and quantity, as these measures reflect patient perceptions of asthma control and disease burden. A search of PubMed and respiratory meetings was performed to identify relevant studies. In two pivotal budesonide/formoterol pMDI studies in adolescents and adults, greater efficacy and similar tolerability were shown with budesonide/formoterol pMDI 160/9 µg and 320/9 µg twice daily versus its monocomponents or placebo. In those studies, improvements in HRQL, patient satisfaction, global assessments of asthma control, and quality of sleep also favored budesonide/formoterol pMDI compared with one or both of its monocomponents or placebo. Budesonide/formoterol pMDI has a rapid onset of effect (within 15 minutes that patients can feel, an attribute that may have benefits for treatment adherence. In summary, budesonide/formoterol pMDI is effective and well tolerated and has additional therapeutic benefits that may be important from the patient’s perspective.Keywords: budesonide, formoterol, patient-reported outcomes, efficacy, tolerability, onset of effect
The Patient Reported Outcomes Following Initial treatment and Long term Evaluation of Survivorship registry: scope, rationale and design of an infrastructure for the study of physical and psychosocial outcomes in cancer survivorship cohorts.
van de Poll-Franse, Lonneke V; Horevoorts, Nicole; van Eenbergen, Mies; Denollet, Johan; Roukema, Jan Anne; Aaronson, Neil K; Vingerhoets, Ad; Coebergh, Jan Willem; de Vries, Jolanda; Essink-Bot, Marie-Louise; Mols, Floortje
'Patient Reported Outcomes Following Initial treatment and Long term Evaluation of Survivorship (PROFILES)' is a registry for the study of the physical and psychosocial impact of cancer and its treatment from a dynamic, growing population-based cohort of both short and long-term cancer survivors. PROFILES contains a large web-based component and are linked directly to clinical data from the population-based Eindhoven cancer registry. This paper describes the rationale and design of PROFILES. The primary aims of studies that use the PROFILES registry are: (1) psychosocial risk and outcome assessment to identify patients at high risk for poor physical and mental health outcomes, (2) to analyse mediating mechanisms to better understand the biological and behavioural factors associated with cancer treatment outcomes, and (3) to evaluate physical and psychosocial care needs of cancer survivors. PROFILES is a tool that enables data collection management; from inviting patients to participation in studies, to collecting patient-reported outcomes data via web-based or mailed questionnaires and linking these data with clinical data. The availability of a control cohort of approximately 2000 persons from the general population who complete the same basic questionnaire annually will provide the opportunity to estimate the unique impact of cancer, beyond that of normal ageing and comorbidities. Raw data from the PROFILES registry will be available for non-commercial scientific research, subject to study question, privacy and confidentiality restrictions, and registration (www.profilesregistry.nl).
Deodhar, Atul A; Dougados, Maxime; Baeten, Dominique L; Cheng-Chung Wei, James; Geusens, Piet; Readie, Aimee; Richards, Hanno B; Martin, Ruvie; Porter, Brian
To evaluate the effect of secukinumab (interleukin-17A inhibitor) on patient-reported outcomes in patients with active ankylosing spondylitis (AS). In this phase III study, 371 patients were randomized (1:1:1) to receive intravenous (IV) secukinumab 10 mg/kg at baseline and weeks 2 and 4 followed by subcutaneous (SC) secukinumab 150 mg every 4 weeks (IV→150 mg group), or SC secukinumab 75 mg every 4 weeks (IV→75 mg group), or placebo. Patient-reported outcomes included the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), BASDAI criteria for 50% improvement (BASDAI 50), Short Form 36 (SF-36) physical component summary (PCS) score and mental component summary (MCS) score, Ankylosing Spondylitis Quality of Life (ASQoL) questionnaire, Bath Ankylosing Spondylitis Functional Index (BASFI), EuroQol 5-domain (EQ-5D) questionnaire, Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), and Work Productivity and Activity Impairment-General Health questionnaire (WPAI-GH). At week 16, secukinumab IV→150 mg or IV→75 mg was associated with statistically and clinically significant improvements from baseline versus placebo in the BASDAI (-2.3 for both regimens versus -0.6; P < 0.0001 and P < 0.001, respectively), SF-36 PCS (5.6 for both regimens versus 1.0; P < 0.0001 and P < 0.001, respectively), and ASQoL (-3.6 for both regimens versus -1.0; P < 0.0001 and P < 0.001, respectively). Clinically significant improvements in the SF-36 MCS, BASFI, EQ-5D, and BASDAI 50 were observed with both secukinumab groups versus placebo at week 16; improvements were also observed in the FACIT-F and WPAI-GH. All improvements were sustained through week 52. Our findings indicate that secukinumab provides significant and sustained improvements in patient-reported disease activity and health-related quality of life, and reduces functional impairment, fatigue, and impact of disease on work productivity in patients with active AS. © 2016 The
Chan, A.; Cameron, M.C.; Garden, B.; Boers-Doets, C.B.; Schindler, K.; Epstein, J.B.; Choi, J.; Beamer, L.; Roeland, E.; Russi, E.G.; Bensadoun, R.J.; Teo, Y.L.; Chan, R.J.; Shih, V.; Bryce, J.; Raber-Durlacher, J.; Gerber, P.A.; Freytes, C.O.; Rapoport, B.; LeBoeuf, N.; Sibaud, V.; Lacouture, M.E.
Purpose Dermatologic adverse events (dAEs) in cancer treatment are frequent with the use of targeted therapies. These dAEs have been shown to have significant impact on health-related quality of life (HRQoL). While standardized assessment tools have been developed for physicians to assess severity o
McKenna Stephen P
Full Text Available Abstract Background The PRIMUS is a Multiple Sclerosis (MS-specific suite of outcome measures including assessments of QoL (PRIMUS QoL, scored 0-22 and activity limitations (PRIMUS Activities, scored 0-30. The U-FIS is a measure of fatigue impact (scored 0-66. These measures have been fully validated previously using an MS sample with mixed diagnoses. The aim of the present study was to validate the measures further in a specifically Relapse Remitting MS (RRMS sample and to provide preliminary evidence of the responder definitions (RD; also known as minimal important difference for these instruments. Methods Data were derived from a multi-country efficacy trial of MS patients with assessments at baseline and 12 months. Baseline data were used to assess the internal reliability and validity of the measures. Both anchor-based and distribution-based approaches were employed for estimating RD. Anchor-based estimates were based on published RD values for the EQ-5D and were assessed for those improving and deteriorating separately. Distribution-based estimates were based on standard error of measurement (SEM, change score equivalent to 0.30, and change score equivalent to 0.50, effect sizes (ES. Results The sample included 911 RRMS patients (67.3% female, age mean (SD 36.2 (8.4 years, duration of MS mean (SD 4.8 (5.2 years. Results showed that the PRIMUS and U-FIS had good internal consistency. Appropriate correlations were observed with comparator instruments and both measures were able to distinguish between participants based on Expanded Disability Status Scale scores and time since diagnosis. The anchor-based and distribution-based RD estimates were: PRIMUS Activities range = 1.2-2.3, PRIMUS QoL range = 1.0-2.2, and U-FIS range = 2.4-7.0. Conclusions The results show that the PRIMUS and U-FIS are valid instruments for use with RRMS patients. The analyses provide preliminary information on how to interpret scores on the scales. These data will be
Rinkel, Rico N.; Verdonck-de Leeuw, Irma M.; Doornaert, Patricia; Buter, Jan; de Bree, Remco; Langendijk, Johannes A.; Aaronson, Neil K.; Leemans, C. Rene
The objective of this study is to assess swallowing and speech outcome after chemoradiation therapy for head and neck cancer, based on the patient-reported outcome measures Swallowing Quality of Life Questionnaire (SWAL-QOL) and Speech Handicap Index (SHI), both provided with cut-off scores. This is
Rinkel, R.N.; Verdonck-de Leeuw, I.M.; Doornaert, P.; Buter, J.; de Bree, R.; Langendijk, J.A.; Aaronson, N.K.; Leemans, C.R.
The objective of this study is to assess swallowing and speech outcome after chemoradiation therapy for head and neck cancer, based on the patient-reported outcome measures Swallowing Quality of Life Questionnaire (SWAL-QOL) and Speech Handicap Index (SHI), both provided with cut-off scores. This is
Merkel, Peter A; Aydin, Sibel Z; Boers, Maarten; Cornell, Christina; Direskeneli, Haner; Gebhart, Don; Hatemi, Gulen; Luqmani, Raashid; Matteson, Eric L; Milman, Nataliya; Robson, Joanna; Seo, Philip; Tomasson, Gunnar
The conduct of randomized controlled trials for vasculitis, especially for the antineutrophil cytoplasmic antibody-associated vasculitides [AAV, granulomatosis with polyangiitis (Wegener's) and microscopic polyangiitis], has been greatly advanced by the development, use, and acceptance of validated outcome measures. Trials have subsequently provided the opportunity to validate and refine reliable, valid outcome measures for these multisystemic and relapsing rare diseases. The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group was formed in 2004 to foster development of validated and widely accepted outcomes in vasculitis using data-driven analyses, a dedication to building consensus, and adherence to, and guidance by, the principles of the OMERACT approach. This work led to the endorsement by OMERACT of the core set of domains and associated outcome measures for AAV. Next steps for the study of existing outcome tools in AAV include better definition of response criteria through development of more data-driven weighting of the elements of activity and damage assessment. The Working Group is now also embarking on a series of linked projects to develop validated patient-reported outcomes for use in clinical research in vasculitis. Additionally, the Working Group is studying how current methods of disease assessment and plans for new outcomes can be informed by the conceptual framework of the International Classification of Function of the World Health Organization. The success of the Group's work in AAV has also led to a formal process for developing outcomes for the large vessel vasculitides (Takayasu arteritis and giant cell arteritis) and Behçet disease.
Patients’ attitudes and perceptions of two health-related quality-of-life questionnaires used to collect patient-reported outcome measures in the English National Health Service: A qualitative study of patients undergoing cardiac interventions
Bashir M Matata
Full Text Available Objectives: To explore patients’ views on the EuroQol-5D and Coronary Revascularisation Outcome Questionnaire, tools currently used for collecting patient-reported outcome measures in the English National Health Service. The key questions were as follows: (1 whether patients consider them sensitive enough to detect change in their health after cardiovascular disease interventions and (2 whether they consider the health-related quality-of-life questions as meaningful. Methods: Data were collected on patients’ views using focus groups. We held four focus groups selecting participants on the basis of their baseline and follow-up EuroQol-5D scores. Data were analysed using framework analysis and grounded theory. Results: Focus group participants confirmed that they had derived substantial health benefits from their cardiac interventions despite the lack of measurable effects on the EuroQol-5D scores. Participants felt that the EuroQol-5D questionnaire was limited because of the following reasons: Their health fluctuates from day to day. They had difficulty assessing their general health status on the visual analogue scale. They felt that the Coronary Revascularisation Outcome Questionnaire was limited because of the following reasons: They did not understand the clinical terms used. The impact of tiredness on their quality of life was not captured. They were unable to distinguish between the effects of their heart condition and other health issues. Additionally, neither questionnaire considers the adjustments people have made to their domestic arrangements to improve their health-related quality of life. Conclusion: This study provides evidence that the two questionnaires do not capture some aspects of health that patients consider important. Furthermore, the presence of co-morbidities masks the symptoms relating to the heart disease and the effect of their cardiac interventions. Future work on patient-reported outcome measures should consider
Jackson, Leanne K; Deng, Jie; Ridner, Sheila H; Gilbert, Jill; Dietrich, Mary S; Murphy, Barbara A
We describe development and preliminary testing of Vanderbilt Head and Neck Symptom Survey-Recurrent/Metastatic (VHNSS-RM) to assess residual symptoms, tumor-related symptoms, and side effects from therapy. Items were identified through patient and provider interviews. Card sort selected high-yield and high-impact items. The VHNSS-RM was administered to 50 patients with recurrent/metastatic head and neck cancer (RMHNC). The VHNSS-RM includes 12 unique symptoms (diet change, tongue movement affecting speech/swallowing, face/neck swelling, neck/jaw cramping, bad breath, drooling, wound drainage/pain/odor, nasal congestion/drainage, eyes watering, face/tongue/ear/scalp numbness, headaches, and confusion) and 7 unique psychosocial issues (burden to family/friends, lost independence, fear, embarrassment, mood swings, stress, and boredom). The VHNSS-RM contains 35 physical and 12 psychosocial issues. The VHNSS-RM is feasible and not overly burdensome. Nineteen unique items may improve palliation to patients with RMHNC. © The Author(s) 2015.
Modi, A; Ebeling, P R; Lee, M S; Min, Y K; Mithal, A; Yang, X; Baidya, S; Sen, S; Sajjan, S
The purpose of this study was to describe the impact of gastrointestinal events on patient-reported outcomes and health care resource use among Asia-Pacific women with postmenopausal osteoporosis. The results of this study show that gastrointestinal events decreased adherence, treatment satisfaction, and quality of life in Asia-Pacific women with postmenopausal osteoporosis. This study aimed to describe the impact of gastrointestinal (GI) events on patient-reported outcomes and health care resource use among Asia-Pacific women with postmenopausal osteoporosis. The MUSIC OS-AP study included an observational cohort study of postmenopausal women with osteoporosis. Women were classified as untreated or treated, with treated patients further classified as new or experienced users. Adherence was measured by the Adherence Evaluation of Osteoporosis treatment (ADEOS) questionnaire, treatment satisfaction by the Osteoporosis Patient Satisfaction Questionnaire (OPSAT) while general health-related and osteoporosis-specific quality of life were measured by the European Quality of Life-5 Dimensions (EQ-5D) questionnaire and the Osteoporosis Assessment Questionnaire (OPAQ), respectively. The association of GI events with these outcomes was determined by covariate-adjusted regression analysis of least squares mean differences in the scores of treated patients with and without GI events. Resource utilization was measured as the number of physician visits over the past 3 months, and multivariate regression analysis was used to assess the association of GI events with the likelihood of a visit. The GI event profile, quality of life scores, and resource use were numerically similar in untreated and treated women. The rate of adherence among treated women was higher in experienced than in new users. As indicated by mean scores, experienced users had better quality of life and slightly higher treatment satisfaction and fewer physician visits than new users. Except for adherence in
刘为民; 崔学军; 何丽云; 刘保延; 罗文舒; 白文静; 毛文超; 方积乾
Patient Reported Outcome (PRO) is an outcome used in the measurement and evaluation of a disease. PRO is directly from patients' perception of their own health condition. It includes symptoms, physiology, psychology and treatment satisfaction. This article introduced the current condition of PRO study and its application in traditional Chinese medicine (TCM). It emphasized on Evaluating the Measurement of Patient -Reported Outcomes (EMPRO). The EMPRO contains 39 items in eight key attributes, which are the conceptual and measurement model, reliability, validity, responsiveness, interpretability, burden, alternative modes of administration, and cross -cultural and linguistic adaptations. This study showed that the EMPRO has high reliability and validity. It has already been used in the assessment of the SF-36 Health Survey, the Nottingham Health Profile, the COOP/WONCA Charts, the EuroQol-5D, and the EORTC-QLQ-C30.%患者报告结局是一类直接以患者对其自身健康状况的感受来测量与评价疾病及其后果的结局指标,广泛用于临床各科,本文介绍了PRO目前研究情况,PRO在中医药领域的应用,应该如何评估,并重点说明用于PRO评估的EMPRO及其应用情况,EMPRO包含了8个方面的内容:概念和测量模型、信度、效度、反应性、可解释性、负担、其他的操作方式和文化和语言调适.研究表明,EMPRO具有较高的信度、效度和可靠性,已经用于评估SF-36、诺丁汉健康量表、COOP/WONCA量表、欧洲五维健康量表和EORTC-QLQ-C30生存质量问卷等量表.
Tyring, Stephen; Solomon, James A; Staedtler, Gerald; Lott, Jason P; Nkulikiyinka, Richard; Shakery, Kaweh
Patient-reported treatment outcomes are important for evaluating the impact of drug therapies on patient experience. A randomized, double-blind, vehicle-controlled, parallel-group, multicenter, phase 3 study was conducted in 961 participants to assess patient perception of efficacy, utility, and effect on quality of life (QOL) of an azelaic acid (AzA) 15% foam formulation for the treatment of papulopustular rosacea (PPR). Secondary end points included patient-reported global assessment of treatment response, global assessment of tolerability, and opinion on cosmetic acceptability and practicability of product use. Quality of life assessments included the Dermatology Quality of Life Index (DLQI) and Rosacea Quality of Life Index (RosaQOL). Self-reported global assessment of treatment response favored AzA foam over vehicle foam (Pfoam group reporting excellent or good improvement versus 44.7% in the vehicle foam group. Tolerability was rated excellent or good in 67.8% of the AzA foam group versus 78.2% of the vehicle foam group. Mean overall DLQI scores at end of treatment (EoT) were improved (P=.018) in favor of the AzA foam group compared with the vehicle foam group. Both treatment groups showed improvements in RosaQOL. Treatment with AzA foam was associated with improved QOL and meaningful reductions in the patient-perceived burden of PPR, which correlates with earlier reported primary end points of this study and supports the inclusion of patient perspectives in studies evaluating the effects of topical dermatologic treatments.
Issa, Kimona; Pierce, Todd P; Harwin, Steven F; Scillia, Anthony J; McInerney, Vincent K; Mont, Michael A
It is estimated that 3%-6% of orthopedic patients, many of whom may undergo lower extremity total joint arthroplasty, are infected with hepatitis C. The purpose of this study was to assess the outcomes of patients with hepatitis C who undergo total hip arthroplasty (THA) in comparison with a matched control cohort in terms of (1) patient-reported outcomes, (2) implant survivorship, and (3) complications. Fifty-four hips in 49 hepatitis C-infected patients who underwent a primary THA between 2002 and 2011 were reviewed. This included 10 women and 39 men who had a mean age of 57 years and a mean 6.5-year follow-up. These patients were matched to 163 THAs (148 patients) who did not have this disease and underwent a THA during the same period. We compared implant survivorship, complication rates, Harris hip scores, and University of California, Los Angeles, activity scores. Radiographs were evaluated for loosening, fracture, malalignment, and osteolysis. The implant survivorship in the hepatitis C-infected patients and comparison group was 96.2% and 98.7%, respectively. The risk of revision surgery in the hepatitis C cohort was 3-fold higher than the comparison group; however, this difference was not significant (P = .26). The hepatitis C-infected cohort had a higher risk of surgical complications (odds ratio = 6.5; P = .034). There were no differences in postoperative Harris hip scores or University of California, Los Angeles, activity scores between the cohorts. Hepatitis C patients can achieve good implant survivorship and clinical outcomes after THA. However, these patients may be at an increased risk for surgical complications and revision rate. Copyright © 2017 Elsevier Inc. All rights reserved.
Burwinkle Tasha M
Full Text Available Abstract Background Attention-Deficit/Hyperactivity Disorder (ADHD is the most common chronic mental health condition in children and adolescents. The application of health-related quality of life (HRQOL as a pediatric population health measure may facilitate risk assessment and resource allocation, the identification of health disparities, and the determination of health outcomes from interventions and policy decisions for children and adolescents with ADHD at the local community, state, and national health level. Methods An analysis from an existing statewide database to determine the feasibility, reliability, and validity of the 23-item PedsQL™ 4.0 (Pediatric Quality of Life Inventory™ Generic Core Scales as a patient-reported outcome (PRO measure of pediatric population health for children and adolescents with ADHD. The PedsQL™ 4.0 Generic Core Scales (Physical, Emotional, Social, School Functioning were completed by families through a statewide mail survey to evaluate the HRQOL of new enrollees in the State of California State's Children's Health Insurance Program (SCHIP. Seventy-two children ages 5–16 self-reported their HRQOL. Results The PedsQL™ 4.0 evidenced minimal missing responses, achieved excellent reliability for the Total Scale Score (α = 0.92 child self-report, 0.92 parent proxy-report, and distinguished between healthy children and children with ADHD. Children with ADHD self-reported severely impaired psychosocial functioning, comparable to children with newly-diagnosed cancer and children with cerebral palsy. Conclusion The results suggest that population health monitoring may identify children with ADHD at risk for adverse HRQOL. The implications of measuring pediatric HRQOL for evaluating the population health outcomes of children with ADHD internationally are discussed.
Sherman, Karen J.; Heagerty, Patrick J.; Mock, Charles; Jarvik, Jeffrey G.
Background Among older adults, it is not clear how different types or amounts of physical therapy may be associated with improvements in back pain and function. Objective The study objective was to investigate the association between types or amounts of physical therapist services and 1-year outcomes among older adults with back pain. Design This was a prospective cohort study. Methods A total of 3,771 older adults who were enrolled in a cohort study and who had a new primary care visit for back pain participated. Physical therapy use was ascertained from electronic health records. The following patient-reported outcomes were collected over 12 months: back-related disability (Roland-Morris Disability Questionnaire) and back and leg pain intensity (11-point numerical rating scale). Marginal structural models were used to estimate average effects of different amounts of physical therapy use on disability and pain for all types of physical therapy and for active, passive, and manual physical therapy. Results A total of 1,285 participants (34.1%) received some physical therapy. There was no statistically significant gradient in relationships between physical therapy use and back-related disability score. The use of passive or manual therapy was not consistently associated with pain outcomes. Higher amounts of active physical therapy were associated with decreased back and leg pain and increased odds of clinically meaningful improvements in back and leg pain relative to results obtained with no active physical therapy. Limitations The fact that few participants had high amounts of physical therapy use limited precision and the ability to test for nonlinear relationships for the amount of use. Conclusions Higher amounts of active physical therapy were most consistently related to the greatest improvements in pain intensity; however, as with all observational studies, the results must be interpreted with caution. PMID:25278334
Stefanovic, Stefan; Wallwiener, Markus; Karic, Uros; Domschke, Christoph; Katic, Luka; Taran, Florin-Andrei; Pesic, Aleksandra; Hartkopf, Andreas; Hadji, Peyman; Teufel, Martin; Schuetz, Florian; Sohn, Christof; Fasching, Peter; Schneeweiss, Andreas; Brucker, Sara
The capture of adequate treatment outcomes and quality of life (QOL) of advanced breast cancer patients in clinical routine represents a great challenge. Patient-reported outcomes (PROs) are data elements directly reported by patients about experiences with care, including symptoms, functional status, or quality of life. There is growing interest in the medical community for the evaluation and implementation of PROs of adverse events (PRO-AEs). Recent interest in PROs in health care has evolved in the context of patient centeredness. Our primary objective was to identify trials that had implemented PRO-AEs in the breast cancer treatment setting, thereby demonstrating its feasibility. We aimed to identify published studies that used patient reports to assess AEs during and after breast cancer treatment, to identify clinician underreported and modifiable AEs that are important to patients, and to analyze the feasibility and usefulness of PRO instrument implementation in everyday oncological practice with special attention given to electronic-based PRO instruments. We conducted a systematic search of PubMed for studies that used PRO instruments to assess AEs of breast cancer treatment in the metastatic and adjuvant settings. Two authors independently reviewed the search results and decided which studies fully met the predefined inclusion criteria. The search yielded 606 publications. The two reviewers found that 9 studies met the inclusion criteria. Three AEs were identified as important to patients but inadequately reported by health care providers, namely hot flushes, vaginal dryness, and weight gain. PROs and PRO-AEs are the consequence of contemporary concepts of patient-centered medicine and the growing feasibility, utility, and implications of collecting data using modern technology. Furthermore, the willingness of patients to utilize innovative applications for their own health has been increasing in parallel to the enhanced impact of the World Wide Web
Aleem, Ilyas S; Duncan, Jonathan; Ahmed, Amin M; Zarrabian, Mohammad; Eck, Jason; Rhee, John; Clarke, Michelle; Currier, Bradford L; Nassr, Ahmad
Prospective cohort study. To characterize the accuracy of patient recollection of preoperative symptoms after lumbar spine surgery. Although patient-reported outcomes have become important in the evaluation of spine surgery patients, the accuracy of patient recall remains unknown. Patients undergoing lumbar decompression with or without fusion were enrolled. Back and leg Numeric Pain Scores and Oswestry Disability Indices were recorded preoperatively. Patients were asked to recall their preoperative status at a minimum of 1 year after surgery. Actual and recalled scores were compared using paired t tests and relations were quantified using Pearson correlation coefficients. Multivariable linear regression was used to identify factors that affected recollection. Sixty-two patients with a mean age of 66.1 years were included. Compared to their preoperative scores, patients showed significant improvement in back pain (mean difference [MD] = -3.2, 95% CI -4.0 to -2.4), leg pain (MD -3.3, 95% CI -4.3 to -2.2), and disability (MD -25.0%, 95% CI -28.7 to -19.6). Patient recollection of preoperative status was significantly more severe than actual for back pain (MD +2.3, 95% CI 1.5-3.2), leg pain (MD +1.8, 95% CI 0.9-2.7), and disability (MD +9.6%, 95% CI 5.6-14.0). No significant correlation between actual and recalled scores with regards to back (r = 0.18) or leg (r = 0.24) pain and only moderate correlation with disability (r = 0.44) were seen. This was maintained across age, sex, and time between date of surgery and recollection. More than 40% of patients switched their predominant symptom from back pain to leg pain or leg pain to back pain on recall. Relying on patient recollection does not provide an accurate measure of preoperative status after lumbar spine surgery. Recall bias indicates the importance of obtaining true baseline scores and patient-reported outcomes prospectively and not retrospectively. 2.
Carol M. Greco
Full Text Available CAM therapies are often dismissed as “no better than placebo;” however, this belief may be overcome through careful analysis of nonspecific factors in healing. To improve trial methodology, we propose that CAM (and conventional RCTs should evaluate and adjust for the effects of intrapersonal, interpersonal, and environmental factors on outcomes. However, measurement of these is challenging, and there are no brief, precise instruments that are suitable for widespread use in trials and clinical settings. This paper describes the methodology of a project to develop a set of patient-reported instruments that will quantify the nonspecific or “placebo” effects that are in fact specific and active ingredients in healing. The project uses the rigorous instrument-development methodology of the NIH-PROMIS initiative. The methods include (1 integration of patients’ and clinicians’ opinions with existing literature; (2 development of relevant items; (3 calibration of items on large samples; (4 classical test theory and modern psychometric methods to select the most useful items; (5 development of computerized adaptive tests (CATs that maximize information while minimizing patient burden; and (6 initial validation studies. The instruments will have the potential to revolutionize clinical trials in both CAM and conventional medicine through quantifying contextual factors that contribute to healing.
Kim, Elliott J; Chotai, Silky; Archer, Kristin R; Bydon, Mohamad; Asher, Anthony L; Devin, Clinton J
Retrospective analysis of prospectively collected data. The aim of this study was to determine whether 1-year patient-reported outcomes (PROs) can accurately assess effective care for patients undergoing surgery for degenerative lumbar spine disease. Prospective longitudinal PROs registries provide a means to accurately assess outcomes and determine the relative effectiveness of various spine treatments. Obtaining long-term PROs can be costly and challenging. Patients enrolled into a prospective registry who underwent lumbar spine surgery for degenerative disease were included. Baseline, 1-year, and 2-year Oswestry Disability Index (ODI) scores were captured. Previously published minimum clinically important difference (MCID) for ODI (14.9) was used. Multivariable linear regression model was created to derive model-estimated 2-year ODI scores. Absolute differences between 1-year and 2-year ODI were compared to absolute differences between 2-year and model-estimated 2-year ODI. Concordance rates in achieving MCID at 1-year and 2-year and predictive values were calculated. A total of 868 patients were analyzed. One-year ODI scores differed from 2-year scores by an absolute difference of 9.7 ± 8.9 points and predictive model-estimated 2-year scores differed from actual 2-year scores by 8.8 ± 7.3 points. The model-estimated 2-year ODI was significantly different than actual 1-year ODI in assessing actual 2-year ODI for all procedures (P = 0.001) except for primary (P = 0.932) and revision microdiscectomy (P = 0.978) and primary laminectomy (P = 0.267). The discordance rates of achieving or not achieving MCID for ODI ranged from 8% to 27%. Concordance rate was about 90% for primary and revision microdiscectomy. The positive and negative predictive value of 1-year ODI to predict 2-year ODI was 83% and 67% for all procedures and 92% and 67% for primary and 100% and 86% for revision microdiscectomy respectively. One-year disability outcomes can
Efficace, F; Cocks, K; Breccia, M; Sprangers, M; Meyers, C A; Vignetti, M; Baccarani, M; Mandelli, F
Health-related quality of life (HRQOL) and other patient-reported outcomes (PROs) might be crucial in comparing effectiveness of treatments as they could provide invaluable information to better inform clinical decision-making. This is particularly true in the era of targeted therapies (TT). A systematic review was undertaken on all studies with CML patients published from 1980 to 2010 and including a PRO evaluation. Out of 619 articles scrutinized, 15 met eligibility criteria and no study was published before 1995. Six dealt mainly with interferon-based therapies, 7 with bone marrow transplantation and only 2 evaluated PROs in the context of TT. No disease-specific, validated PRO instrument for these patients was found. The main evidence being that Imatinib provides clear advantage in terms of HRQOL over interferon-based treatments. There is lack of data concerning PROs in patients treated with current TT. Documenting HRQOL and side effects of CML treatments, from the patients' perspective is needed to evaluate overall treatment effectiveness and net clinical benefit of newer therapeutic strategies.
Goren, Amir; Carter, Chureen; Lee, Seina
The objective of this study is to compare health outcomes of patients using biologic therapies ustekinumab (UST) or adalimumab (ADA) for moderate-to-severe plaque psoriasis (PsO) and assess biologics non-adherence. Two phases of web-based survey data were collected, assessing adult patients with PsO from a Diplomat® Specialty Pharmacy US claims database (Diplomat Specialty Pharmacy; Flint, MI). Measures included demographics, treatment and health characteristics/behaviors, treatment satisfaction, health-related quality of life (HRQoL), and productivity. Pooled and stratified (by biologics experience) bivariate and multivariable analyses were conducted. UST (n = 262) versus ADA (n = 83) users more frequently had psoriasis cleared (40.5% versus 15.4%, respectively, with no visible signs), better HRQoL as per Dermatology Life Quality Index (DLQI) score = 0 (45.2% versus 19.2%), and higher current effectiveness satisfaction, all p patients (n = 68) had better (53.4% lower) DLQI scores, lower percent body surface affected (%BSA; 0.85 versus 1.43), more %BSA improvement (-1.60 versus -1.03), and lower activity impairment (90.4% lower), all p Non-adherence to UST (11.8%) versus ADA (32.5%) was lower, p patients reported higher clearing rates, better DLQI, and lower activity impairment.
Yu, M; Van Brunt, K; Varnado, O J; Boye, K S
We evaluated patient-reported outcome (PRO) measures from the Assessment of Weekly AdministRation of LY2189265 (dulaglutide) in Diabetes (AWARD) clinical trial programme for dulaglutide (1.5 mg and 0.75 mg) in patients with type 2 diabetes (T2D). The Impact of Weight on Self-Perception (IW-SP), Impact of Weight on Ability to Perform Physical Activities of Daily Living (APPADL), Impact of Weight on Quality of Life-Lite, EQ-5D, Diabetes Treatment Satisfaction Questionnaire (DTSQ), Diabetes Symptom Checklist-Revised and Adult Low Blood Sugar Survey were administered and analysed for changes from baseline in one or more AWARD studies. Significant within-group changes from baseline to the primary time point were observed for several PRO measures across all studies. Compared with insulin glargine, significantly greater improvements in the IW-SP score were observed with dulaglutide 1.5 mg and with both dulaglutide doses in the APPADL score. Both dulaglutide doses resulted in significantly greater improvement in DTSQ scores (all subscales) compared with exenatide. Dulaglutide 1.5 mg also resulted in significantly greater improvement on the DTSQ hyperglycaemia subscale compared with metformin. Overall, these PRO results suggest that dulaglutide is beneficial in the treatment of T2D.
Objective: We. would develop the Chronic Hepatitis B Patient Report Outcome Scale with Chinese Characteristics, and test the reliahility, validity and responsibility of the scale. Methods : We investigated 326 patients with chronic hepatitis B. After using several statistical methods,28 items were composed of the formal scale. Then the formal scale was tested about reliability, validity and responsibility. Results ,(. 1 )The Chronic Hepatitis B PRO Scale with Chinese Characteristics was developed including physical, psychological and social fields, and a total of 28 items. ( 2 ) It was proved that the PRO scale was reliable, effective, and sensitive by the reliability, validity and responsibility test. Conclusions : The PRO Scale of Chronic Hepatitis B with Chinese Characteristics has good reliability, validity and responsibility, it can be a good reflection of chronic hepatitis B patients physical, psychological and social fitness, it can be used to evaluate the clinical efficacy of chronic hepatitis B patients.%目的:研制具有中医特色的慢性乙型肝炎患者自评量表(Patient report outcome,PRO量表).方法:采用流行病学方法对326例慢乙肝患者进行调查,收集数据,通过一系列统计分析研制慢性乙型肝炎中医特色PRO量表,并对其进行科学性考核.结果:研制出具有较好信度、效度和反应度的慢性乙型肝炎中医特色PRO量表.结论:慢性乙型肝炎中医特色PRO量表能较好反映慢性乙肝患者的生理、心理和社会适应度,可以用来辅助评价慢乙肝患者的临床疗效.
Maria Cristina Lima e Silva
Full Text Available Background: Mental disorders often impair functioning in several areas of life and lead to unhappiness and suffering that may affect health-related quality of life (HRQoL. Satisfaction with participation is an indicator of HRQoL, and its measurement by patients reflects the impact of disease on their social, emotional and professional life. The Patient-Reported Outcomes Measurement Information System (PROMIS(r offers an item bank based on item response theory. This system provides efficient, reliable and valid self-report instruments of satisfaction with participation, a measure that is both scarce and useful in the assessment of mental disorder outcomes.Objective:To cross-culturally adapt the PROMIS(r satisfaction with participation item bank to Portuguese.Methods:Cross-cultural adaptation followed the Functional Assessment of Chronic Illness Therapy (FACIT multilingual translation method and was achieved through steps of forward and backward translations, review by bilingual experts (one of them a native of Portugal and pretesting in a group of 11 adult native Brazilians. Instrument adaptation followed a universal approach to translation, with harmonization across languages.Results: Equivalence of meaning was achieved. As two of the 26 translated items, which asked about leisure and social activities, were not understood by less educated participants, an explanation in parentheses was added to each item, and the problem was solved. All items were appropriate and did not cause embarrassment to the participants.Conclusions: The satisfaction with participation item bank is culturally and linguistically suitable to be used in Brazil. After the pretest is applied in Portugal and in other Portuguese-speaking countries, the same instrument will be ready to be used in multinational studies.
Conijn, Anne P., E-mail: firstname.lastname@example.org [Academic Medical Center, Departments of Vascular Surgery and Interventional Radiology (Netherlands); Jonkers, Wilma, E-mail: email@example.com [Achmea Insurances, Division of Health Care (Netherlands); Rouwet, Ellen V., E-mail: firstname.lastname@example.org [Erasmus Medical Center, Department of Vascular Surgery (Netherlands); Vahl, Anco C., E-mail: email@example.com [Onze Lieve Vrouwe Gasthuis, Department of Vascular Surgery (Netherlands); Reekers, Jim A., E-mail: firstname.lastname@example.org [Academic Medical Center, Department of Radiology (Netherlands); Koelemay, Mark J. W., E-mail: email@example.com [Academic Medical Center, Department of vascular surgery (Netherlands)
PurposeThe minimally important difference (MID) represents the smallest change in score on patient-reported outcome measures that is relevant to patients. The aim of this study was to introduce the MID for the Vascular Quality of Life Questionnaire (VascuQol) and the walking impairment questionnaire (WIQ) for patients with intermittent claudication (IC).MethodsIn this multicenter study, we recruited 294 patients with IC between July and October 2012. Patients completed the VascuQol, with scores ranging from 1 to 7 (worst to best), and the WIQ, with scores ranging from 0 to 1 (worst to best) at first visit and after 4 months follow-up. In addition, patients answered an anchor-question rating their health status compared to baseline, as being improved, unchanged, or deteriorated. The MID for improvement and deterioration was calculated by an anchor-based approach, and determined with the upper and lower limits of the 95 % confidence interval of the mean change of the group who had not changed according to the anchor-question.ResultsFor the MID analyses of the VascuQol and WIQ, 163 and 134 patients were included, respectively. The MID values for the VascuQol (mean baseline score 4.25) were 0.87 for improvement and 0.23 for deterioration. For the WIQ (mean baseline score 0.39), we found MID values of 0.11 and −0.03 for improvement and deterioration, respectively.ConclusionIn this study, we calculated the MID for the VascuQol and the WIQ. Applying these MID facilitates better interpretation of treatment outcomes and can help to set treatment goals for individual care.
Full Text Available Abstract Background Skin-sparing mastectomy (SSM and latissimus dorsi (LD flap immediate breast reconstruction (IBR is a tailored surgical procedure. The surgical and patient-reported outcome (PRO of SSM and LD IBR were assessed. Methods Retrospective data of 146 SSMs performed by a single surgeon was reviewed. Among patients included in the data, 65 patients underwent SSM and LD IBR without a prosthetic implant. A survey estimating the degree of patient satisfaction (poor, fair, good, and excellent as regards the cosmetic outcomes of surgery was performed. The patients were divided into two groups according to their degree of satisfaction (excellent group versus non- excellent group, and analysis was done to identify factors affecting the highest patient satisfaction. Results The mean age of the patients was 48.4 years, and pathological results were: infiltrating ductal carcinoma (n = 48, 73.8%, ductal carcinoma in situ (n = 15, 23.1%, and others (n = 2, 3.1%. One patient received postmastectomy radiotherapy. After a mean follow-up of 34 months, no local recurrence occurred. There was no skin necrosis or LD flap loss. Donor site morbidities were seroma (n = 8, 12.3%, scarring (n = 8, 12.3%, and back pain (n = 6, 9.2%. Fifty patients (76.9% were satisfied and 40% reported their degree of satisfaction as excellent. Breast symmetry (P P P = 0.021, and panel assessment score (P Conclusions Our SSM and LD IBR was safe, with no local recurrence and low morbidities, and produced a sufficiently high level of patient satisfaction. Achieving breast symmetry and nipple cosmesis would be the key to meeting the patient’s expectation.
Silva, Maria Cristina Lima E; Mendonça, Tânia Maria da Silva; da Silva, Carlos Henrique Martins; Pinto, Rogério de Melo Costa
Mental disorders often impair functioning in several areas of life and lead to unhappiness and suffering that may affect health-related quality of life (HRQoL). Satisfaction with participation is an indicator of HRQoL, and its measurement by patients reflects the impact of disease on their social, emotional and professional life. The Patient-Reported Outcomes Measurement Information System (PROMIS(r)) offers an item bank based on item response theory. This system provides efficient, reliable and valid self-report instruments of satisfaction with participation, a measure that is both scarce and useful in the assessment of mental disorder outcomes. To cross-culturally adapt the PROMIS(r) satisfaction with participation item bank to Portuguese. Cross-cultural adaptation followed the Functional Assessment of Chronic Illness Therapy (FACIT) multilingual translation method and was achieved through steps of forward and backward translations, review by bilingual experts (one of them a native of Portugal) and pretesting in a group of 11 adult native Brazilians. Instrument adaptation followed a universal approach to translation, with harmonization across languages. Equivalence of meaning was achieved. As two of the 26 translated items, which asked about leisure and social activities, were not understood by less educated participants, an explanation in parentheses was added to each item, and the problem was solved. All items were appropriate and did not cause embarrassment to the participants. The satisfaction with participation item bank is culturally and linguistically suitable to be used in Brazil. After the pretest is applied in Portugal and in other Portuguese-speaking countries, the same instrument will be ready to be used in multinational studies.
de Souza, Jonas A; Yap, Bonnie J; Wroblewski, Kristen; Blinder, Victoria; Araújo, Fabiana S; Hlubocky, Fay J; Nicholas, Lauren H; O'Connor, Jeremy M; Brockstein, Bruce; Ratain, Mark J; Daugherty, Christopher K; Cella, David
Cancer and its treatment lead to increased financial distress for patients. To the authors' knowledge, to date, no standardized patient-reported outcome measure has been validated to assess this distress. Patients with AJCC Stage IV solid tumors receiving chemotherapy for at least 2 months were recruited. Financial toxicity was measured by the COmprehensive Score for financial Toxicity (COST) measure. The authors collected data regarding patient characteristics, clinical trial participation, health care use, willingness to discuss costs, psychological distress (Brief Profile of Mood States [POMS]), and health-related quality of life (HRQOL) as measured by the Functional Assessment of Cancer Therapy: General (FACT-G) and the European Organization for Research and Treatment of Cancer (EORTC) QOL questionnaires. Test-retest reliability, internal consistency, and validity of the COST measure were assessed using standard-scale construction techniques. Associations between the resulting factors and other variables were assessed using multivariable analyses. A total of 375 patients with advanced cancer were approached, 233 of whom (62.1%) agreed to participate. The COST measure demonstrated high internal consistency and test-retest reliability. Factor analyses revealed a coherent, single, latent variable (financial toxicity). COST values were found to be correlated with income (correlation coefficient [r] = 0.28; Pfinancial toxicity were race (P = .04), employment status (Pfinancial distress (P = .49). The COST measure demonstrated reliability and validity in measuring financial toxicity. Its correlation with HRQOL indicates that financial toxicity is a clinically relevant patient-centered outcome. Cancer 2017;123:476-484. © 2016 American Cancer Society. © 2016 The Authors. Cancer published by Wiley Periodicals, Inc. on behalf of American Cancer Society.
Smolen, Josef S; Kremer, Joel M; Gaich, Carol L; DeLozier, Amy M; Schlichting, Douglas E; Xie, Li; Stoykov, Ivaylo; Rooney, Terence; Bird, Paul; Sánchez Bursón, Juan Miguel; Genovese, Mark C; Combe, Bernard
To assess baricitinib on patient-reported outcomes (PROs) in patients with moderately to severely active rheumatoid arthritis, who had insufficient response or intolerance to ≥1 tumour necrosis factor inhibitors (TNFis) or other biological disease-modifying antirheumatic drugs (bDMARDs). In this double-blind phase III study, patients were randomised to once-daily placebo or baricitinib 2 or 4 mg for 24 weeks. PROs included the Short Form-36, EuroQol 5-D, Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), Health Assessment Questionnaire-Disability Index (HAQ-DI), Patient's Global Assessment of Disease Activity (PtGA), patient's assessment of pain, duration of morning joint stiffness (MJS) and Work Productivity and Activity Impairment Questionnaire-Rheumatoid Arthritis. Treatment comparisons were performed with logistic regression for categorical measures or analysis of covariance for continuous variables. 527 patients were randomised (placebo, 176; baricitinib 2 mg, 174; baricitinib 4 mg, 177). Both baricitinib-treated groups showed statistically significant improvements versus placebo in most PROs. Improvements were generally more rapid and of greater magnitude for patients receiving baricitinib 4 mg than 2 mg and were maintained to week 24. At week 24, more baricitinib-treated patients versus placebo-treated patients reported normal physical functioning (HAQ-DI <0.5; p≤0.001), reductions in fatigue (FACIT-F ≥3.56; p≤0.05), improvements in PtGA (p≤0.001) and pain (p≤0.001) and reductions in duration of MJS (p<0.01). Baricitinib improved most PROs through 24 weeks compared with placebo in this study of treatment-refractory patients with previously inadequate responses to bDMARDs, including at least one TNFi. PRO results aligned with clinical efficacy data for baricitinib. NCT01721044; Results. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/.
Stockler, Martin R; Hilpert, Felix; Friedlander, Michael; King, Madeleine T; Wenzel, Lari; Lee, Chee Khoon; Joly, Florence; de Gregorio, Nikolaus; Arranz, José Angel; Mirza, Mansoor Raza; Sorio, Roberto; Freudensprung, Ulrich; Sneller, Vesna; Hales, Gill; Pujade-Lauraine, Eric
To determine the effects of bevacizumab on patient-reported outcomes (PROs; secondary end point) in the AURELIA trial. Patients with platinum-resistant ovarian cancer were randomly assigned to chemotherapy alone (CT) or with bevacizumab (BEV-CT). PROs were assessed using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Ovarian Cancer Module 28 (EORTC QLQ-OV28) and Functional Assessment of Cancer Therapy-Ovarian Cancer symptom index (FOSI) at baseline and every two or three cycles (8/9 weeks) until disease progression. The primary PRO hypothesis was that more patients receiving BEV-CT than CT would achieve at least a 15% (≥ 15-point) absolute improvement on the QLQ-OV28 abdominal/GI symptom subscale (items 31-36) at week 8/9. Patients with missing week 8/9 questionnaires were included as unimproved. Questionnaires from all assessments until disease progression were analyzed using mixed-model repeated-measures (MMRM) analysis. Sensitivity analyses were used to determine the effects of differing assumptions and methods for missing data. Baseline questionnaires were available from 89% of 361 randomly assigned patients. More BEV-CT than CT patients achieved a ≥ 15% improvement in abdominal/GI symptoms at week 8/9 (primary PRO end point, 21.9% v 9.3%; difference, 12.7%; 95% CI, 4.4 to 20.9; P = .002). MMRM analysis covering all time points also favored BEV-CT (difference, 6.4 points; 95% CI, 1.3 to 11.6; P = .015). More BEV-CT than CT patients achieved ≥ 15% improvement in FOSI at week 8/9 (12.2% v 3.1%; difference, 9.0%; 95% CI, 2.9% to 15.2%; P = .003). Sensitivity analyses gave similar results and conclusions. Bevacizumab increased the proportion of patients achieving a 15% improvement in patient-reported abdominal/GI symptoms during chemotherapy for platinum-resistant ovarian cancer.
Full Text Available Katharine Batt,1 Michael Recht,2 David L Cooper,3 Neeraj N Iyer,3 Christine L Kempton4 1Hematology and Oncology, Wake Forest School of Medicine, Winston-Salem, NC, 2The Hemophilia Center, Oregon Health & Science University, Portland, OR, 3Novo Nordisk Inc., Plainsboro, NJ, 4Departments of Pediatrics and Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA, USA Background: People with hemophilia (PWH experience frequent joint bleeding, resulting in pain and functional impairment. Generic and disease-specific patient-reported outcome (PRO instruments have been used in clinical studies, but rarely in the comprehensive hemophilia care setting. Objective: The objective of this study was to assess construct validity of PRO instruments measuring pain, functional impairment, and health-related quality of life in US PWH with a history of joint pain/bleeding. Methods: Adult male PWH completed 4 PRO instruments (EQ-5D-5L with visual analog scale, Brief Pain Inventory v2 Short Form [BPI], SF-36v2, Hemophilia Activities List [HAL] and underwent a musculoskeletal examination (Hemophilia Joint Health Score v2.1 [HJHS]. Construct validity between index and domain scores was evaluated by Pearson product-moment correlation coefficient. Results: A total of 381 PWH were enrolled. EQ-5D-5L Mobility correlated with BPI, SF-36v2, and HAL domains related to pain, physical function, and activity of the lower extremities. EQ-5D-5L Self-Care correlated only with HAL Self-Care. EQ-5D-5L Usual Activities correlated with BPI Pain Interference and domains within SF-36v2 and HAL related to pain and physical function/activities (particularly those involving the lower extremities. EQ-5D-5L Pain/Discomfort correlated with Bodily Pain and Physical Summary on SF-36v2, HAL Overall Activity, and all BPI pain domains. EQ-5D-5L Anxiety/Depression correlated with social/emotional/mental aspects of SF-36v2. On BPI, most pain domains correlated with Bodily
Laxy, Michael; Mielck, Andreas; Hunger, Matthias; Schunk, Michaela; Meisinger, Christa; Rückert, Ina-Maria; Rathmann, Wolfgang; Holle, Rolf
Little is known about the impact of diabetes self-management behavior (SMB) on long-term outcomes. We aimed to examine the association among patient-reported SMB, intermediate clinical outcomes, and mortality in patients with type 2 diabetes. Data were collected from 340 patients with type 2 diabetes of the KORA-A study (1997/1998) who were recruited from two previous population-based surveys (n = 161) and a myocardial infarction registry (n = 179) in southern Germany. Based on previous methodological work, a high level of SMB was defined as being compliant with at least four of six different self-care dimensions, comprising physical exercise, foot care, blood glucose self-monitoring, weight monitoring, having a diet plan, and keeping a diabetes diary. The vital status of the participants was observed until 2009. Multivariable linear, logistic, and Cox regression models were applied to assess the association with intermediate clinical outcomes at baseline and to predict mortality over the follow-up period, adjusted for sociodemographic, behavioral, and disease-related factors. In the cross-sectional perspective, a high level of SMB was weakly associated with a lower glycated hemoglobin A1c level (-0.44% [-4.8 mmol/mol] [95% CI -0.88 to 0.00]), but not with low-density lipoprotein cholesterol, systolic blood pressure, or the presence of microalbuminuria, peripheral arterial disease, or polyneuropathy. During a mean follow-up time of 11.6 years, 189 patients died. SMB was a preventive factor for all-cause (hazard ratio 0.61 [95% CI 0.40-0.91]) and cardiovascular mortality (0.65 [95% CI 0.41-1.03]). Although measuring SMB is difficult and the used operationalization might be limited, our results give some indication that a high level of SMB is associated with prolonged life expectancy in patients with type 2 diabetes and highlight the potential impact of the patients' active contribution on the long-term trajectory of the disease. We assume that the used proxy for SMB
Limbers Christine A
Full Text Available Abstract Background Fibromyalgia is a chronic health condition characterized by widespread musculoskeletal pain, multiple tender points on physical examination, generalized muscular aching, stiffness, fatigue, nonrestorative sleep pattern, cognitive dysfunction, and mood disturbance. Recently, the Outcome Measures in Rheumatoid Arthritis Clinical Trials (OMERACT Fibromyalgia Syndrome Workshop ranked and prioritized the domains that should be consistently measured in fibromyalgia clinical trials, specifically, pain, generic health-related quality of life, fatigue, sleep quality, and physical function. The focus of these deliberations was exclusively on adult patients, and to our knowledge, these domains have not been previously tested within a multidimensional framework in children and adolescents with fibromyalgia. Methods An analysis to determine the feasibility, reliability, and validity of the PedsQL™ 4.0 (Pediatric Quality of Life Inventory™ Generic Core Scales, PedsQL™ Multidimensional Fatigue Scale, and PedsQL™ Rheumatology Module Pain and Hurt Scale as patient-reported outcome (PRO measures for pediatric patients with fibromyalgia. The PedsQL™ Scales were completed by 59 families in a pediatric rheumatology clinic in a large children's hospital. Results The PedsQL™ evidenced minimal missing responses (0.53% patient self-report, 0.70% parent proxy-report, achieved excellent reliability for the Generic Core Scales Total Scale Score (α = 0.88 patient self-report, 0.87 parent proxy-report, the Multidimensional Fatigue Scale Total Scale Score (α = 0.94 patient self-report, 0.94 parent proxy-report, and acceptable reliability for the 4-item Rheumatology Module Pain and Hurt Scale (α = 0.68 patient self-report, 0.75 parent proxy-report. The PedsQL™ Generic Core Scales and Multidimensional Fatigue Scale significantly distinguished between pediatric patients with fibromyalgia and healthy children. Pediatric patients with
Shekhawat, Laxmi; Busheri, Laleh; Dixit, Santosh; Patel, Chaula; Dhar, Upendra; Koppiker, Chaitanyanand
Breast Cancer (BC) treatment leads to mutilation and destruction of breast shape with negative effects on body image and self-esteem.One of the main goals of reconstructive and oncoplastic breast surgery is to satisfy patients and improve their quality of life (QoL).Therefore, it is important to assess the patient experience post-surgery by means of patient-reported outcome measures (PROMs) that focus on the patient's perception of the surgery and surgical care, as well as psychosocial well-being and physical functioning. The objective of the current study was to identify predictors of patient satisfaction such as breast appearance including implant type in a selective sample of women who underwent breast reconstruction surgery using implants. Participants in this prospective study were women, (age 26-75 years) that were newly diagnosed with breast carcinoma. All consecutive patients who underwent breast reconstruction between January 2013 and October 2014 were asked to complete the BREAST-Q questionnaire 1 year after surgery. 120 patients underwent unilateral breast reconstruction using implant. While 38 patients underwent reconstruction with opposite breast reduction symmertization, 27 patients underwent therapeutic mammoplasty. The response rate for BREAST-Q questionnaire completion was 98 % with 147 out of 150 study participants completed the questionnaire. From the data collected from 147 patients, the responses could be distributed into 4 distinct groups based on the reconstruction outcomes namely "very much satisfied" (93 %) or "definitely and mostly satisfied" (94 %) or "satisfied" with the outcome (88 %) or "definitely agree on having reconstruction rather than the alternative of having no breast "(91 %).The results showed significant improvement in all four areas that were evaluated after surgery namely satisfaction with the appearance of the breasts, psychosocial, sexual and physical well-being. While the reconstruction surgery had an overall
Full Text Available Christine L Kempton,1 Michael Wang,2 Michael Recht,3 Anne Neff,4 Amy D Shapiro,5 Amit Soni,6 Roshni Kulkarni,7 Tyler W Buckner,2 Katharine Batt,8 Neeraj N Iyer,9 David L Cooper9 1Departments of Pediatrics and Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA, USA; 2Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO, USA; 3The Hemophilia Center, Oregon Health & Science University, Portland, OR, USA; 4Hematology and Medical Oncology, Cleveland Clinic, Cleveland, OH, USA; 5Indiana Hemophilia & Thrombosis Center, Indianapolis, IN, USA; 6Center for Inherited Blood Disorders and CHOC Children’s Hospital/UC Irvine, Orange, CA, USA; 7MSU Center for Bleeding and Clotting Disorders, Michigan State University, East Lansing, MI, USA; 8Hematology and Oncology, Wake Forest School of Medicine, Winston-Salem, NC, USA; 9Clinical, Medical and Regulatory Affairs, Novo Nordisk Inc., Plainsboro, NJ, USA Background: Hemophilia is marked by frequent joint bleeding, resulting in pain and functional impairment.Objective: This study aimed to assess the reliability of five patient-reported outcome (PRO instruments in people with hemophilia (PWH in a non-bleeding state.Methods: Adult male PWH of any severity and inhibitor status, with a history of joint pain or bleeding, completed a pain history and five PRO instruments (EQ-5D-5L, Brief Pain Inventory v2 [BPI], International Physical Activity Questionnaire [IPAQ], Short Form 36 Health Survey v2 [SF-36v2], and Hemophilia Activities List [HAL] during their routine comprehensive care visit. Patients were approached to complete the PRO instruments again at the end of their visit while in a similar non-bleeding state. Concordance of individual questionnaire items and correlation between domain scores were assessed using intra-class correlation coefficient (ICC.Results: Participants completing the retest (n=164 had a median age of 33.9 years. Median time for
JOSEPH P. EIMICKE
Full Text Available The aims of this paper are to present findings related to differential item functioning (DIF in the Patient Reported Outcome Measurement Information System (PROMIS depression item bank, and to discuss potential threats to the validity of results from studies of DIF. The 32 depression items studied were modified from several widely used instruments. DIF analyses of gender, age and education were performed using a sample of 735 individuals recruited by a survey polling firm. DIF hypotheses were generated by asking content experts to indicate whether or not they expected DIF to be present, and the direction of the DIF with respect to the studied comparison groups. Primary analyses were conducted using the graded item response model (for polytomous, ordered response category data with likelihood ratio tests of DIF, accompanied by magnitude measures. Sensitivity analyses were performed using other item response models and approaches to DIF detection. Despite some caveats, the items that are recommended for exclusion or for separate calibration were "I felt like crying" and "I had trouble enjoying things that I used to enjoy." The item, "I felt I had no energy," was also flagged as evidencing DIF, and recommended for additional review. On the one hand, false DIF detection (Type 1 error was controlled to the extent possible by ensuring model fit and purification. On the other hand, power for DIF detection might have been compromised by several factors, including sparse data and small sample sizes. Nonetheless, practical and not just statistical significance should be considered. In this case the overall magnitude and impact of DIF was small for the groups studied, although impact was relatively large for some individuals.
Abstract Background Nowadays, more and more clinical scales consisting in responses given by the patients to some items (Patient Reported Outcomes - PRO), are validated with models based on Item Response Theory, and more specifically, with a Rasch model. In the validation sample, presence of missing data is frequent. The aim of this paper is to compare sixteen methods for handling the missing data (mainly based on simple imputation) in the context of psychometric validation of PRO by a Rasch model. The main indexes used for validation by a Rasch model are compared. Methods A simulation study was performed allowing to consider several cases, notably the possibility for the missing values to be informative or not and the rate of missing data. Results Several imputations methods produce bias on psychometrical indexes (generally, the imputation methods artificially improve the psychometric qualities of the scale). In particular, this is the case with the method based on the Personal Mean Score (PMS) which is the most commonly used imputation method in practice. Conclusions Several imputation methods should be avoided, in particular PMS imputation. From a general point of view, it is important to use an imputation method that considers both the ability of the patient (measured for example by his\\/her score), and the difficulty of the item (measured for example by its rate of favourable responses). Another recommendation is to always consider the addition of a random process in the imputation method, because such a process allows reducing the bias. Last, the analysis realized without imputation of the missing data (available case analyses) is an interesting alternative to the simple imputation in this context.
Full Text Available Abstract Background Nowadays, more and more clinical scales consisting in responses given by the patients to some items (Patient Reported Outcomes - PRO, are validated with models based on Item Response Theory, and more specifically, with a Rasch model. In the validation sample, presence of missing data is frequent. The aim of this paper is to compare sixteen methods for handling the missing data (mainly based on simple imputation in the context of psychometric validation of PRO by a Rasch model. The main indexes used for validation by a Rasch model are compared. Methods A simulation study was performed allowing to consider several cases, notably the possibility for the missing values to be informative or not and the rate of missing data. Results Several imputations methods produce bias on psychometrical indexes (generally, the imputation methods artificially improve the psychometric qualities of the scale. In particular, this is the case with the method based on the Personal Mean Score (PMS which is the most commonly used imputation method in practice. Conclusions Several imputation methods should be avoided, in particular PMS imputation. From a general point of view, it is important to use an imputation method that considers both the ability of the patient (measured for example by his/her score, and the difficulty of the item (measured for example by its rate of favourable responses. Another recommendation is to always consider the addition of a random process in the imputation method, because such a process allows reducing the bias. Last, the analysis realized without imputation of the missing data (available case analyses is an interesting alternative to the simple imputation in this context.
Albrecht, Helmut; Vernon, Margaret; Solomon, Gail
Guaifenesin is a component of medicines used to improve symptoms associated with upper respiratory tract infections. Patient-reported outcome instruments are valuable for evaluating symptom improvements; however, a validated tool to assess efficacy of mucoactive drugs does not exist. We compared the efficacy of extended-release guaifenesin with placebo for treatment of symptoms of upper respiratory tract infection using subjective efficacy assessments in a pilot study and confirmed precision of assessments in a validation study. The pilot study was a randomized, double-blind study where patients were dosed with either 1200 mg extended-release guaifenesin (n = 188) or placebo (n = 190), every 12 hours for 7 days. Efficacy was assessed using subjective measures including the Daily Cough and Phlegm Diary, the Spontaneous Symptom Severity Assessment and the Wisconsin Upper Respiratory Symptom Survey. End-of-study assessments were completed by patients and investigator. The validation study consisted of two phases. In Phase I, subjects completed interviews to gather evidence to support the content validity of the Daily Cough and Phlegm Diary, the Spontaneous Symptom Severity Assessment and Patient's End-of-Treatment Assessment. Phase II examined the psychometric properties of assessments evaluated in Phase I of the validation study using data from the pilot study. Subjective measures of efficacy at Day 4 showed the most prominent difference between treatment groups, in favor of guaifenesin. The 8-symptom related questions (SUM8) in the Daily Cough and Phlegm Diary, analyzed as a composite score appeared to be the strongest candidate endpoint for further evaluation. Results from the interviews in Phase I supported the content of the assessments which were validated during Phase II. Treatments were well tolerated. Results from the clinical pilot and validation studies showed that the SUM8 diary scores were robust and reliable for use as efficacy endpoints in studies of
Tang, Qian Ying; Lai, Wei Hong; Tay, Shian Chao
There is a paucity of studies in published literature that examines the effect of hand dominance on the resolution of symptoms following a carpal tunnel release. The objective of this study is to examine the effect of hand dominance on the resolution of symptoms following surgical decompression in patients with severe and moderate carpal tunnel syndrome. Bilateral carpal tunnel release (total 90 open and 84 endoscopic) was performed on 87 patients (11 males, 76 females) presenting with bilateral severe or moderate carpal tunnel syndrome of equal severity. Patient-reported outcome of resolution of symptoms were recorded, with patients followed up until complete resolution of symptoms or last recorded consultation (mean follow-up duration 11.4 months, range 3.1 to 32.4 months). In patients with bilateral severe carpal tunnel syndrome, a larger proportion of non-dominant hand (75.4%) achieved complete resolution compared to dominant hand (72.1%), and did so at a statistically shorter time (mean: 52.3 days) than the dominant hand (mean: 81.0 days). However, there was no statistically significant difference between proportion of patients and time taken before complete resolution of symptoms between dominant and non-dominant hand in patients with bilateral moderate carpal tunnel syndrome. Symptoms in the non-dominant hand resolved faster after carpal tunnel release in patients with severe carpal tunnel syndrome. We postulate that greater daily activity by the dominant hand compared to the non-dominant hand may be a contributing factor to its slower rate of symptoms resolution post-surgically in patients with bilateral severe carpal tunnel syndrome. This effect of hand dominance is not evident in post-surgical patients with moderate carpal tunnel syndrome.
Coons, Stephen Joel; Eremenco, Sonya; Lundy, J Jason; O'Donohoe, Paul; O'Gorman, Hannah; Malizia, William
Patient-reported outcomes (PROs) are an important means of evaluating the treatment benefit of new medical products. It is recognized that PRO measures should be used when assessing concepts best known by the patient or best measured from the patient's perspective. As a result, there is growing emphasis on well defined and reliable PRO measures. In addition, advances in technology have significantly increased electronic PRO (ePRO) data collection capabilities and options in clinical trials. The movement from paper-based to ePRO data capture has enhanced the integrity and accuracy of clinical trial data and is encouraged by regulators. A primary distinction in the types of ePRO platforms is between telephone-based interactive voice response systems and screen-based systems. Handheld touchscreen-based devices have become the mainstay for remote (i.e., off-site, unsupervised) PRO data collection in clinical trials. The conventional approach is to provide study subjects with a handheld device with a device-based proprietary software program. However, an emerging alternative for clinical trials is called bring your own device (BYOD). Leveraging study subjects' own Internet-enabled mobile devices for remote PRO data collection (via a downloadable app or a Web-based data collection portal) has become possible due to the widespread use of personal smartphones and tablets. However, there are a number of scientific and operational issues that must be addressed before BYOD can be routinely considered as a practical alternative to conventional ePRO data collection methods. Nevertheless, the future for ePRO data collection is bright and the promise of BYOD opens a new chapter in its evolution.
Teresi, Jeanne A.; Ocepek-Welikson, Katja; Kleinman, Marjorie; Eimicke, Joseph P.; Crane, Paul K.; Jones, Richard N.; Lai, Jin-shei; Choi, Seung W.; Hays, Ron D.; Reeve, Bryce B.; Reise, Steven P.; Pilkonis, Paul A.; Cella, David
The aims of this paper are to present findings related to differential item functioning (DIF) in the Patient Reported Outcome Measurement Information System (PROMIS) depression item bank, and to discuss potential threats to the validity of results from studies of DIF. The 32 depression items studied were modified from several widely used instruments. DIF analyses of gender, age and education were performed using a sample of 735 individuals recruited by a survey polling firm. DIF hypotheses were generated by asking content experts to indicate whether or not they expected DIF to be present, and the direction of the DIF with respect to the studied comparison groups. Primary analyses were conducted using the graded item response model (for polytomous, ordered response category data) with likelihood ratio tests of DIF, accompanied by magnitude measures. Sensitivity analyses were performed using other item response models and approaches to DIF detection. Despite some caveats, the items that are recommended for exclusion or for separate calibration were “I felt like crying” and “I had trouble enjoying things that I used to enjoy.” The item, “I felt I had no energy,” was also flagged as evidencing DIF, and recommended for additional review. On the one hand, false DIF detection (Type 1 error) was controlled to the extent possible by ensuring model fit and purification. On the other hand, power for DIF detection might have been compromised by several factors, including sparse data and small sample sizes. Nonetheless, practical and not just statistical significance should be considered. In this case the overall magnitude and impact of DIF was small for the groups studied, although impact was relatively large for some individuals. PMID:20336180
Bradley, C; Gilbride, C J B
Insulin therapy becomes essential for many people with type 2 diabetes. After starting insulin, people with diabetes that is poorly controlled with oral agents typically report improved well-being and treatment satisfaction. However, healthcare professionals and people with type 2 diabetes are often reluctant to begin insulin treatment, citing concerns such as time/resources needed to educate patients, increased risks of hypoglycaemia and fear of injections, which lead them to focus on intensifying conventional oral therapy. Insulin glargine, which offers people with diabetes a once-a-day injection regimen with low risk of hypoglycaemia, is more likely to overcome such initial barriers than other more complex insulin regimens. Once-daily insulin glargine, in combination with modern glucose-dependent oral agents that do not need to be chased with food to prevent hypoglycaemia, does not require the fixed mealtimes and set amounts of carbohydrates necessary with twice-daily injection mixes and older sulphonylureas. We know that it is such dietary restrictions that cause the most damage to quality of life (QoL). To avoid damaging QoL unnecessarily and to ensure optimal satisfaction with treatment, it is important to evaluate the effects of treatment on QoL, treatment satisfaction and other patient-reported outcomes (PROs) using questionnaires validated for this purpose, such as the widely used Diabetes Treatment Satisfaction Questionnaire and the Audit of Diabetes-Dependent Quality of Life measure. A systematic electronic literature search identified reports of studies evaluating PROs associated with insulin glargine in comparison with other treatments. The studies show that insulin glargine is usually associated with greater improvements in treatment satisfaction and other PROs compared with intensifying oral therapy or alternative insulin regimens.
Hollen, Patricia J; Msaouel, Pavlos; Gralla, Richard J
Identifying key issues for patients is central to assessing treatment for cancer, especially when evaluating health-related quality of life (QL) and patient-reported outcomes (PROs). This study was conducted to provide enhanced content validity support by incorporating the views of a large number of patients with breast cancer. This methodological study used an anonymous, cross-sectional, electronic web-based survey of 1072 patients with a diagnosis of breast cancer. Patients ranked the importance of 21 issues on a 5-point scale. Issues included general, physical, functional, psychosocial, and summative items. Analysis was also performed by four key factors (age group, time since diagnosis, adjuvant treatment or not, and tumor extent). All of the top five issues rated as either "very important" or "important" were global issues-rather than symptoms-such as maintaining quality of life (ranked in these two highest categories by 99 % of patients), maintaining independence (97 %), and ability to perform normal activities (97 %). The abilities to concentrate and to be able to sleep (97 and 96 %, respectively) were ranked above specific breast cancer symptoms. Specific symptoms included within the top ten highest ranked items were fatigue, depression, anxiety, shortness of breath, and pain. This is the largest analysis of evidence-based data determining support for content validity for QL and PROs provided by patients with breast cancer. While symptoms are important to patients, the survey also demonstrates that PRO measures that only evaluate symptoms are not fully responding to patient-expressed needs. These results provide confidence in the content of quality of life measures for large groups of patients with breast cancer, including the new Breast Cancer Symptom Scale (BCSS) questionnaire.
Joswig, Holger; Stienen, Martin N; Smoll, Nicolas R; Corniola, Marco V; Chau, Ivan; Schaller, Karl; Hildebrandt, Gerhard; Gautschi, Oliver P
The Timed Up and Go (TUG) test, as a measure of objective functional impairment in lumbar degenerative disk disease (DDD), complements patient-reported outcome measures (PROMs) of subjective functional impairment. Prospective 2-center study on consecutive patients scheduled for surgical treatment for lumbar DDD who underwent an objective (TUG test) and subjective (PROMs) functional assessment before and 1 and 3 days after surgery. PROMs included the visual analog scale (VAS), Roland-Morris Disability Index (RMDI), Oswestry Disability Index (ODI), Euro-Qol (EQ-5D), and Short Form 12 (SF-12) questionnaires. On completion of each assessment, patients were asked whether they would prefer performing the TUG test or completing the PROMs questionnaires. A total of 109 of 125 patients (87.2%) completed the assessments. Preoperatively, patients were 2.18 times as likely to prefer the TUG test to the PROMs (odds ratio [OR], 2.18; 95% confidence interval [CI], 1.27-3.75). On postoperative days 1 and 3, patients were 5.79 (OR, 5.79; 95% CI, 3.23-10.37) and 6.33 times as likely to prefer the TUG test to the PROMs, respectively (OR, 6.33; 95% CI, 3.51-11.41). There were no statistical differences in baseline characteristics, TUG t scores, VAS, RMDI, ODI, SF-12, and EQ-5D index between patients preferring either the TUG test or the PROMs questionnaires. The TUG test is preferred over a battery of PROMs by 60%-70% of patients with lumbar DDD not only in the preoperative, but also in the postoperative, setting. High functional disability does not result in avoidance of the TUG test, and repeated assessments lead to higher preference. Copyright © 2016 Elsevier Inc. All rights reserved.
Volk, Gerd Fabian; Granitzka, Thordis; Kreysa, Helene; Klingner, Carsten M; Guntinas-Lichius, Orlando
Patients with facial palsy (FP) not only suffer from their facial movement disorder, but also from social and psychological disabilities. These can be assessed by patient-reported outcome measures (PROMs) like the quality-of-life Short-Form 36 Item Questionnaire (SF36) or FP-specific instruments like the Facial Clinimetric Evaluation Scale (FaCE) or the Facial Disability Index (FDI). Not much is known about factors influencing PROMs in patients with FP. We identified predictors for baseline SF36, FaCE, and FDI scoring in 256 patients with unilateral peripheral FP using univariate correlation and multivariate linear regression analyses. Mean age was 52 ± 18 years. 153 patients (60 %) were female. 90 patients (31 %) and 176 patients (69 %) were first seen 90 days after onset, respectively, i.e., with acute or chronic FP. House-Brackmann grading was 3.9 ± 1.4. FaCE subscores varied from 41 ± 28 to 71 ± 26, FDI scores from 65 ± 20 to 70 ± 22, and SF36 domains from 52 ± 20 to 80 ± 24. Older age, female gender, higher House-Brackmann grading, and initial assessment >90 days after onset were independent predictors for lower FaCE subscores and partly for lower FDI subscores (all p role (all p < 0.05). Specific PROMs reveal that older and female patients and patients with chronic FP suffer particularly from motor and non-motor disabilities related to FP. Comorbidity unrelated to the FP could additionally impact the quality of life of patients with FP.
Full Text Available Naoko Takahashi-Ando,1 Mark A Jones,2 Shigeki Fujisawa,3 Rokuro Hama4 1Department of Applied Chemistry, Faculty of Science and Engineering, Toyo University, Saitama, Japan; 2School of Population Health, The University of Queensland, Herston, Queensland, Australia; 3Fujisawa Dermatology Clinic, Tokyo, Japan; 4Japan Institute of Pharmacovigilance, Osaka, Japan Background: Topical corticosteroid (TCS treatment is widely prescribed for atopic dermatitis (AD. However, TCS treatment is associated with tachyphylaxis, and discontinuation after long-term use may cause exacerbation of symptoms. Some AD patients are reluctant to use TCS. Objective: To evaluate patient-reported short- and long-term outcomes after discontinuation of TCS treatment for AD. Methods: Questionnaires were distributed to adult AD patients (n=1,812 of doctors who did not recommend TCS as first-line therapy for patients who preferred to avoid TCS. Data collected included current TCS use, duration of TCS use, past discontinuation of TCS use, exacerbation of symptoms after discontinuation of TCS use, and limitations to daily activities because of AD. Results: Of 918 respondents, 97.7% had used TCS, of whom 92.3% had experienced discontinuation of TCS use. After discontinuation, 63.9% experienced their most severe AD symptoms ever. The severity of exacerbation of symptoms was significantly correlated with the length of TCS use (P<0.001. Although most respondents who experienced severe exacerbation after TCS discontinuation were not current TCS users, they generally had fewer current limitations to activities than when AD symptoms were at their worst. Conclusion: Adult Japanese AD patients who experience severe exacerbation of symptoms immediately after discontinuation of TCS use generally improve over time. We suggest caution regarding long-term TCS treatment in AD patients.Keywords: adverse effects of corticosteroids, symptom exacerbation, rosacea-like dermatitis
Lee, Yi Xuan; Kwan, Yu Heng; Png, Wan Yu; Lim, Ka Keat; Tan, Chuen Seng; Lui, Nai Lee; Chew, Eng Hui; Thumboo, Julian; Østbye, Truls; Fong, Warren
To determine if obesity is associated with poorer patient-reported outcomes (PROs) in patients with axial spondyloarthritis (axSpA), we conducted a cross-sectional study using data of the PRESPOND registry from a tertiary referral center in Singapore between 2011 and 2015. Demographics, clinical, and PRO variables were collected. Patients were divided into three categories: normal (BMI < 23 kg/m(2)), overweight (23 kg/m(2) ≤ BMI < 27.5 kg/m(2)) and obese (BMI ≥ 27.5 kg/m(2)), using Asian BMI classification. The dependent variables are Pain score, Bath Ankylosing Spondylitis Patient Global Score (BAS-G), Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), Bath Ankylosing Spondylitis Functional Index (BASFI), Health Assessment Questionnaire (HAQ), and Medical Outcomes Study Short Form 36 version 2 (SF-36). Multivariate regression analyses were performed with these dependent variables and obesity categories, adjusting for confounders. Among 194 patients with axSpA, 32% are overweight while 22% are obese. We found that obese patients had significant poorer pain (β: 11.87, 95%CI 2.13, 21.60) and BAS-G scores (β: 10.18, 95%CI 1.59, 18.76) when compared to normal BMI patients. However, obesity was not associated with BASDAI (β 0.50, 95%CI -0.22, 1.22), BASFI (β 0.08, 95%CI -0.66, 0.81), HAQ (β -0.07, 95%CI -0.21, 0.06), physical component summary (β -0.02, 95%CI -4.47, 4.44), and mental component summary (β -2.85, 95%CI -7.57, 1.88) of SF-36. Obesity was associated with pain score and BAS-G but not with BASDAI, BASFI, HAQ, and SF-36. Further study is needed to examine the causal relationship between obesity and poorer PROs.
Drummond, F J; Kinnear, H; Donnelly, C; O'Leary, E; O'Brien, K; Burns, R M; Gavin, A; Sharp, L
Objective To establish an international patient-reported outcomes (PROMs) study among prostate cancer survivors, up to 18 years postdiagnosis, in two countries with different healthcare systems and ethical frameworks. Design A cross-sectional, postal survey of prostate cancer survivors sampled and recruited via two population-based cancer registries. Healthcare professionals (HCPs) evaluated patients for eligibility to participate. Questionnaires contained validated instruments to assess health-related quality of life and psychological well-being, including QLQ-C30, QLQ-PR25, EQ-5D-5L, 21-question Depression, Anxiety and Stress Scale (DASS-21) and the Decisional Regret Scale. Setting Republic of Ireland (RoI) and Northern Ireland (NI). Primary outcome measures Registration completeness, predictors of eligibility and response, data missingness, unweighted and weighted PROMs. Results Prostate cancer registration was 80% (95% CI 75% to 84%) and 91% (95% CI 89% to 93%) complete 2 years postdiagnosis in NI and RoI, respectively. Of 12 322 survivors sampled from registries, 53% (n=6559) were classified as eligible following HCP screening. In the multivariate analysis, significant predictors of eligibility were: being ≤59 years of age at diagnosis (p<0.001), short-term survivor (<5 years postdiagnosis; p<0.001) and from RoI (p<0.001). 3348 completed the questionnaire, yielding a 54% adjusted response rate. 13% of men or their families called the study freephone with queries for assistance with questionnaire completion or to talk about their experience. Significant predictors of response in multivariate analysis were: being ≤59 years at diagnosis (p<0.001) and from RoI (p=0.016). Mean number of missing questions in validated instruments ranged from 0.12 (SD 0.71; EQ-5D-5L) to 3.72 (SD 6.30; QLQ-PR25). Weighted and unweighted mean EQ-5D-5L, QLQ-C30 and QLQ-PR25 scores were similar, as were the weighted and unweighted prevalences of depression, anxiety and
significantly lower risk. H1N1p vaccine was well tolerated by children under 5 years with side effects reported at a similar frequency to that found in the total population.Conclusions: Safety and tolerability data from influenza vaccine recipients including young children (via parents/carers can be effectively collected using an online questionnaire with a telephone option (PROBE. The influenza A (H1N1p vaccine was well tolerated, but was associated with more local short-term reactions than the seasonal influenza vaccine.Keywords: safety, influenza, vaccination, H1N1, patient reported outcomes, side effects
Ahmed, Khaled; Kyte, Derek; Keeley, Thomas; Efficace, Fabio; Armes, Jo; Brown, Julia M; Calman, Lynn; Copland, Chris; Gavin, Anna; Glaser, Adam; Greenfield, Diana M; Lanceley, Anne; Taylor, Rachel; Velikova, Galina; Brundage, Michael; Mercieca-Bebber, Rebecca; King, Madeleine T; Calvert, Melanie
Emerging evidence suggests that patient-reported outcome (PRO)-specific information may be omitted in trial protocols and that PRO results are poorly reported, limiting the use of PRO data to inform cancer care. This study aims to evaluate the standards of PRO-specific content in UK cancer trial protocols and their arising publications and to highlight examples of best-practice PRO protocol content and reporting where they occur. The objective of this study is to determine if these early findings are generalisable to UK cancer trials, and if so, how best we can bring about future improvements in clinical trials methodology to enhance the way PROs are assessed, managed and reported. Trials in which the primary end point is based on a PRO will have more complete PRO protocol and publication components than trials in which PROs are secondary end points. Completed National Institute for Health Research (NIHR) Portfolio Cancer clinical trials (all cancer specialities/age-groups) will be included if they contain a primary/secondary PRO end point. The NIHR portfolio includes cancer trials, supported by a range of funders, adjudged as high-quality clinical research studies. The sample will be drawn from studies completed between 31 December 2000 and 1 March 2014 (n=1141) to allow sufficient time for completion of the final trial report and publication. Two reviewers will then review the protocols and arising publications of included trials to: (1) determine the completeness of their PRO-specific protocol content; (2) determine the proportion and completeness of PRO reporting in UK Cancer trials and (3) model factors associated with PRO protocol and reporting completeness and with PRO reporting proportion. The study was approved by the ethics committee at University of Birmingham (ERN_15-0311). Trial findings will be disseminated via presentations at local, national and international conferences, peer-reviewed journals and social media including the CPROR twitter account
Full Text Available Abstract Background Guaifenesin is a component of medicines used to improve symptoms associated with upper respiratory tract infections. Patient-reported outcome instruments are valuable for evaluating symptom improvements; however, a validated tool to assess efficacy of mucoactive drugs does not exist. We compared the efficacy of extended-release guaifenesin with placebo for treatment of symptoms of upper respiratory tract infection using subjective efficacy assessments in a pilot study and confirmed precision of assessments in a validation study. Methods The pilot study was a randomized, double-blind study where patients were dosed with either 1200 mg extended-release guaifenesin (n = 188 or placebo (n = 190, every 12 hours for 7 days. Efficacy was assessed using subjective measures including the Daily Cough and Phlegm Diary, the Spontaneous Symptom Severity Assessment and the Wisconsin Upper Respiratory Symptom Survey. End-of-study assessments were completed by patients and investigator. The validation study consisted of two phases. In Phase I, subjects completed interviews to gather evidence to support the content validity of the Daily Cough and Phlegm Diary, the Spontaneous Symptom Severity Assessment and Patient’s End-of-Treatment Assessment. Phase II examined the psychometric properties of assessments evaluated in Phase I of the validation study using data from the pilot study. Results Subjective measures of efficacy at Day 4 showed the most prominent difference between treatment groups, in favor of guaifenesin. The 8-symptom related questions (SUM8 in the Daily Cough and Phlegm Diary, analyzed as a composite score appeared to be the strongest candidate endpoint for further evaluation. Results from the interviews in Phase I supported the content of the assessments which were validated during Phase II. Treatments were well tolerated. Conclusions Results from the clinical pilot and validation studies showed that the SUM8 diary
Ahmed, Khaled; Kyte, Derek; Keeley, Thomas; Efficace, Fabio; Armes, Jo; Brown, Julia M; Calman, Lynn; Copland, Chris; Gavin, Anna; Glaser, Adam; Greenfield, Diana M; Lanceley, Anne; Taylor, Rachel; Velikova, Galina; Brundage, Michael; Mercieca-Bebber, Rebecca; King, Madeleine T
Introduction Emerging evidence suggests that patient-reported outcome (PRO)-specific information may be omitted in trial protocols and that PRO results are poorly reported, limiting the use of PRO data to inform cancer care. This study aims to evaluate the standards of PRO-specific content in UK cancer trial protocols and their arising publications and to highlight examples of best-practice PRO protocol content and reporting where they occur. The objective of this study is to determine if these early findings are generalisable to UK cancer trials, and if so, how best we can bring about future improvements in clinical trials methodology to enhance the way PROs are assessed, managed and reported. Hypothesis: Trials in which the primary end point is based on a PRO will have more complete PRO protocol and publication components than trials in which PROs are secondary end points. Methods and analysis Completed National Institute for Health Research (NIHR) Portfolio Cancer clinical trials (all cancer specialities/age-groups) will be included if they contain a primary/secondary PRO end point. The NIHR portfolio includes cancer trials, supported by a range of funders, adjudged as high-quality clinical research studies. The sample will be drawn from studies completed between 31 December 2000 and 1 March 2014 (n=1141) to allow sufficient time for completion of the final trial report and publication. Two reviewers will then review the protocols and arising publications of included trials to: (1) determine the completeness of their PRO-specific protocol content; (2) determine the proportion and completeness of PRO reporting in UK Cancer trials and (3) model factors associated with PRO protocol and reporting completeness and with PRO reporting proportion. Ethics and dissemination The study was approved by the ethics committee at University of Birmingham (ERN_15-0311). Trial findings will be disseminated via presentations at local, national and international conferences, peer
Responsiveness and Minimally Important Differences for 4 Patient-Reported Outcomes Measurement Information System Short Forms: Physical Function, Pain Interference, Depression, and Anxiety in Knee Osteoarthritis.
Lee, Augustine C; Driban, Jeffrey B; Price, Lori Lyn; Harvey, William F; Rodday, Angie Mae; Wang, Chenchen
Patient-Reported Outcomes Measurement Information System (PROMIS) instruments can provide valid, interpretable measures of health status among adults with osteoarthritis (OA). However, their ability to detect meaningful change over time is unknown. We evaluated the responsiveness and minimally important differences (MIDs) for 4 PROMIS Short Forms: Physical Function, Pain Interference, Depression, and Anxiety. We analyzed adults with symptomatic knee OA from our randomized trial comparing Tai Chi and physical therapy. Using baseline and 12-week scores, responsiveness was evaluated according to consensus standards by testing 6 a priori hypotheses of the correlations between PROMIS and legacy change scores. Responsiveness was considered high if ≥5 hypotheses were confirmed, and moderate if 3 or 4 were confirmed. MIDs were evaluated according to prospective change for people achieving previously-established MID on legacy comparators. The lowest and highest MIDs meeting a priori quality criteria formed a MID range for each PROMIS Short Form. Among 165 predominantly female (70%) and white (57%) participants, mean age was 61 years and body mass index was 33. PROMIS Physical Function had 5 confirmed hypotheses and Pain Interference, Depression, and Anxiety had 3 or 4. MID ranges were: Depression = 3.0 to 3.1; Anxiety = 2.3 to 3.4; Physical Function = 1.9 to 2.2; and Pain Interference = 2.35 to 2.4. PROMIS Physical Function has high responsiveness, and Depression, Anxiety, and Pain Interference have moderate responsiveness among adults with knee OA. We established the first MIDs for PROMIS in this population, and provided an important standard of reference to better apply or interpret PROMIS in future trials or clinical practice. This study examined whether PROMIS Short Form instruments (Physical Function, Pain Interference, Depression, and Anxiety) were able to detect change over time among adults with knee OA, and provided minimally important change estimates
Feasibility and diagnostic accuracy of the Patient-Reported Outcomes Measurement Information System (PROMIS) item banks for routine surveillance of sleep and fatigue problems in ambulatory cancer care.
Leung, Yvonne W; Brown, Catherine; Cosio, Andrea Perez; Dobriyal, Aditi; Malik, Noor; Pat, Vivien; Irwin, Margaret; Tomasini, Pascale; Liu, Geoffrey; Howell, Doris
Routine screening for problematic symptoms is emerging as a best practice in cancer systems globally. The objective of this observational study was to assess the feasibility and diagnostic accuracy of Patient-Reported Outcomes Measurement Information System (PROMIS) computerized adaptive testing (CAT) for fatigue and sleep-disturbance items compared with legacy measures in routine ambulatory cancer care. Patients who attended outpatient clinics at the Princess Margaret Cancer Center completed PROMIS CAT item banks and legacy measures (the Functional Assessment of Chronic Illness Therapy [FACIT]-Fatigue scale and the Insomnia Severity Index [ISI]) using tablet computers during clinic visits. The completion rates, patient acceptability, and diagnostic accuracy of PROMIS CAT were evaluated against legacy measures using receiver operating characteristic (ROC) curve analysis. Participants consisted of 336 patients (mean age ± standard deviation, 57.4 ± 15.7 years; 55% females; 75% Caucasian). Over 98% of patients did not find symptom screening was burdensome, although only 65% were willing to complete the survey at every visit. PROMIS CAT scores were significantly correlated with both FACIT-Fatigue scores (r = -0.83) and ISI scores (r = -0.57; p < 0.0001 for all). Areas under the curve (AUC) by ROC analysis for fatigue were 0.946 using the FACIT-Fatigue cutoff ≤30, 0.910 for sleep disturbance, and 0.922 for sleep impairment using the ISI cutoff ≥15. The recommended T-score cut-off for PROMIS CAT Fatigue was 57, Sleep Disturbance was 57, and Sleep Impairment was 57. The current results support the feasibility and accuracy of PROMIS CAT and its potential for use in routine ambulatory cancer care. Future research will assess feedback of these data to clinicians and evaluate effects on earlier identification of and intervention for these problems. Cancer 2016. © 2016 American Cancer Society. Cancer 2016;122:2906-2917. © 2016 American Cancer
Winters, Z E; Afzal, M; Balta, V; Freeman, J; Llewellyn-Bennett, R; Rayter, Z; Cook, J; Greenwood, R; King, M T
The aim of this study was to estimate the impact 2 and 3 years after surgery of implant-assisted latissimus dorsi (LDI) and autologous latissimus dorsi (ALD) flap breast reconstructions on patient-reported outcomes (PROs), and, secondarily, to determine whether baseline characteristics can predict PROs. This was a multicentre prospective cohort study. The European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Core Questionnaire (QLQ-C30) and breast cancer module (QLQ-BR23), Functional Assessment of Cancer Therapy - Breast (FACT-B), and Hospital Anxiety and Depression Scale (HADS) PROs were completed before surgery and at 2 and 3 years after breast reconstruction. The effects of LDI and ALD, adjusted for baseline clinicodemographic characteristics, were estimated with multiple linear regressions. Effect sizes above 0·5 were considered clinically important. Some 206 patients (LDI 93, ALD 113) were recruited in 2007-2013; 66·5 per cent were node-negative and 34·6 per cent received radiotherapy. Women with adverse clinicopathological factors were more likely to have received radiotherapy and to undergo ALD. Patients in both surgical groups showed clinically important effects at 2 and 3 years, including improvements in emotional scales, but worse physical functioning, social well-being, body image and anxiety. Radiotherapy adversely affected social functioning at 2 years (P = 0·002). Women undergoing ALD reconstruction had significantly improved sexual functioning at 3 years (P = 0·003) relative to those who had LDI procedures, even after adjusting for case mix (P = 0·007). At 3 years, younger women experienced worse physical well-being than older women (P = 0·006), and chemotherapy was associated with worse arm symptoms (P = 0·005). Clinically important changes occurred in physical functioning, breast symptoms, body image and psychological distress. These results will guide selections of key PRO
Full Text Available Elizabeth Gibbons, Paul Hewitson, David Morley, Crispin Jenkinson, Ray Fitzpatrick Health Service Research Unit, Nuffield Department of Population Health, University of Oxford, Oxford, UK Background: This report presents evidence regarding the development and validation of a new questionnaire, the Outcomes and Experiences Questionnaire (OEQ. The rationale for the questionnaire is to bring together into one short instrument questions about two distinct domains – patients' reports of the outcomes of their care and how they experience care.Methods: The OEQ was developed from literature reviews, iterative drafting and discussion within the research group and cognitive testing with a sample of patients who had a hospital experience. Two validation studies were carried out with an eleven item OEQ. The goals of the studies were to examine response rates and to test specific hypotheses of how OEQ should relate to other variables normally collected in the two studies. In the first study, the OEQ was added to the follow-up questionnaires for patients (n=490 receiving surgery for hip or knee replacement or varicose vein procedures participating in the national Patient Reported Outcome Measures (PROMs program permitting the analysis of the OEQ against change scores for the measures obtained before and after surgery. In the second study the OEQ was included in a sample of patients (n=586 who had been selected to receive the National Health Service (NHS inpatient survey from three contrasting hospital trusts.Results: Results from study one provided consistent and substantial evidence of construct validity of OEQ particularly for those receiving hip or knee replacement. The OEQ sub-scales behaved differently and as predicted against other PROMs variables. Again hypotheses of how the two sub-scales regarding outcomes and experiences would relate to the existing domains of patient experience in the inpatient survey were broadly confirmed in study two
Lamberts, M.P.; Oudsten, B.L. den; Keus, F.; Vries, J. de; Laarhoven, C.J.H.M. van; Westert, G.P.; Drenth, J.P.H.; Roukema, J.A.
BACKGROUND: Up to 41% of patients report pain after cholecystectomy and in most studies follow-up for these symptoms did not exceed 5 years. The episodic nature of abdominal pain associated with symptomatic cholelithiasis warrants long-term follow-up studies. We assessed which patient and surgical f
Lamberts, M.P.; den Oudsten, B.L.; Keus, F.; de Vries, J.; van Laarhoven, C.J.H.M.; Westert, G.P.; Drenth, J.P.H.; Roukema, J.A.
Background Up to 41 % of patients report pain after cholecystectomy and in most studies follow-up for these symptoms did not exceed 5 years. The episodic nature of abdominal pain associated with symptomatic cholelithiasis warrants long-term follow-up studies. We assessed which patient and surgical f
Tijssen, M.P.W.; Cingel, R.E. van; Visser, E de; Nijhuis-Van der Sanden, M.W.G.
OBJECTIVES: To describe data of short- and midterm results of hip arthroscopy patients based on patient-reported hip function, hip functional performance and return to sports activities. DESIGN: Observational cohort study. SETTING: Sports medical center. PARTICIPANTS: 37 recreational athletes (21 me
Tijssen, M.P.W.; Cingel, R.E. van; Visser, E de; Nijhuis-Van der Sanden, M.W.G.
OBJECTIVES: To describe data of short- and midterm results of hip arthroscopy patients based on patient-reported hip function, hip functional performance and return to sports activities. DESIGN: Observational cohort study. SETTING: Sports medical center. PARTICIPANTS: 37 recreational athletes (21
Jeanne A. Teresi
Full Text Available Short form measures from the Patient Reported Outcomes Measurement Information System® (PROMIS® are used widely. The present study was among the first to examine differential item functioning (DIF in the PROMIS Depression short form scales in a sample of over 5000 racially/ethnically diverse patients with cancer. DIF analyses were conducted across different racial/ethnic, educational, age, gender and language groups. Methods: DIF hypotheses, generated by content experts, informed the evaluation of the DIF analyses. The graded item response theory (IRT model was used to evaluate the five-level ordinal items. The primary tests of DIF were Wald tests; sensitivity analyses were conducted using the IRT ordinal logistic regression procedure. Magnitude was evaluated using expected item score functions, and the non-compensatory differential item functioning (NCDIF and T1 indexes, both based on group differences in the item curves. Aggregate impact was evaluated with expected scale score (test response functions; individual impact was assessed through examination of differences in DIF adjusted and unadjusted depression estimates. Results: Many items evidenced DIF; however, only a few had slightly elevated magnitude. No items evidenced salient DIF with respect to NCDIF and the scale-level impact was minimal for all group comparisons. The following short form items might be targeted for further study because they were also hypothesized to evidence DIF. One item showed slightly higher magnitude of DIF for age: nothing to look forward to; conditional on depression, this item was more likely to be endorsed in the depressed direction by individuals in older groups as contrasted with the cohort aged 21 to 49. This item was also hypothesized to show age DIF. Only one item (failure showed DIF of slightly higher magnitude (just above threshold for Whites vs. Asians/Pacific Islanders in the direction of higher likelihood of endorsement for Asians
Full Text Available Aims: The goals of these analyses were to examine the psychometric properties and measurement equivalence of a self-reported cognition measure, the Patient Reported Outcome Measurement Information System® (PROMIS® Applied Cognition – General Concerns short form. These items are also found in the PROMIS Cognitive Function (version 2 item bank. This scale consists of eight items related to subjective cognitive concerns. Differential item functioning (DIF analyses of gender, education, race, age, and (Spanish language were performed using an ethnically diverse sample (n = 5,477 of individuals with cancer. This is the first analysis examining DIF in this item set across ethnic and racial groups. Methods: DIF hypotheses were derived by asking content experts to indicate whether they posited DIF for each item and to specify the direction. The principal DIF analytic model was item response theory (IRT using the graded response model for polytomous data, with accompanying Wald tests and measures of magnitude. Sensitivity analyses were conducted using ordinal logistic regression (OLR with a latent conditioning variable. IRT-based reliability, precision and information indices were estimated. Results: DIF was identified consistently only for the item, brain not working as well as usual. After correction for multiple comparisons, this item showed significant DIF for both the primary and sensitivity analyses. Black respondents and Hispanics in comparison to White non-Hispanic respondents evidenced a lower conditional probability of endorsing the item, brain not working as well as usual. The same pattern was observed for the education grouping variable: as compared to those with a graduate degree, conditioning on overall level of subjective cognitive concerns, those with less than high school education also had a lower probability of endorsing this item. DIF was observed for age for two items after correction for multiple comparisons for both the IRT and
Measurement equivalence of the Patient Reported Outcomes Measurement Information System® (PROMIS® Pain Interference short form items: Application to ethnically diverse cancer and palliative care populations
Jeanne A. Teresi
Full Text Available Reducing the response burden of standardized pain measures is desirable, particularly for individuals who are frail or live with chronic illness, e.g., those suffering from cancer and those in palliative care. The Patient Reported Outcome Measurement Information System® (PROMIS® project addressed this issue with the provision of computerized adaptive tests (CAT and short form measures that can be used clinically and in research. Although there has been substantial evaluation of PROMIS item banks, little is known about the performance of PROMIS short forms, particularly in ethnically diverse groups. Reviewed in this article are findings related to the differential item functioning (DIF and reliability of the PROMIS pain interference short forms across diverse socio-demographic groups. Methods: DIF hypotheses were generated for the PROMIS short form pain interference items. Initial analyses tested item response theory (IRT model assumptions of unidimensionality and local independence. Dimensionality was evaluated using factor analytic methods; local dependence (LD was tested using IRT-based LD indices. Wald tests were used to examine group differences in IRT parameters, and to test DIF hypotheses. A second DIF-detection method used in sensitivity analyses was based on ordinal logistic regression with a latent IRT-derived conditioning variable. Magnitude and impact of DIF were investigated, and reliability and item and scale information statistics were estimated. Results: The reliability of the short form item set was excellent. However, there were a few items with high local dependency, which affected the estimation of the final discrimination parameters. As a result, the item, “How much did pain interfere with enjoyment of social activities?” was excluded in the DIF analyses for all subgroup comparisons. No items were hypothesized to show DIF for race and ethnicity; however, five items showed DIF after adjustment for multiple comparisons in
Full Text Available Małgorzata Farnik, Władysław PierzchałaDepartment of Pneumonology, Silesian University of Medicine, Katowice, PolandAbstract: Patient-related outcomes measures could provide important information for the current state of the art in medical care and even have an impact on macrodecisions in the health care system. Patient-related outcomes were initially defined as subjective health indicators that allow disability and illness to be assessed, based on patient, caregiver, or physician self-reports. As illness involves psychological and behavioral complex processes of care, a multidisciplinary approach in measuring patient-reported outcomes should be recommended, such as quality of life questionnaires. Patient-related outcomes measures should correspond to specific clinical situations and bring opportunities to improve quality of care. Objective measurements enable quantitative data to be collected and analyzed. Depending on the aim of the research, investigators can use existing methods or develop new tools. This publication presents a methodology for developing patient-related outcomes measures, based on a multistage procedure. The proper definition of specific study objectives and the methodology of instrument development are crucial for successfully transferring the study concept. The model of instrument development is the process of starting from the preliminary phase and includes questionnaire design and scaling, pilot testing (cognitive debriefing, revision of the preliminary version, evaluation of the new tool, and implementation. Validation of the new instrument includes reliability, reproducibility, internal consistency, and responsiveness. The process of designing the new tool should involve a panel of experts, including clinicians, psychologists (preliminary phase, and statisticians (scale development and scoring, and patients (cognitive debriefing. Implementation of a new tool should be followed by evaluation study – assessment of
Standardizing and personalizing the treat to target (T2T) approach for rheumatoid arthritis using the Patient-Reported Outcomes Measurement Information System (PROMIS): baseline findings on patient-centered treatment priorities.
Bacalao, Emily J; Greene, George J; Beaumont, Jennifer L; Eisenstein, Amy; Muftic, Azra; Mandelin, Arthur M; Cella, David; Ruderman, Eric M
A treat to target (T2T) approach to management has become the standard of care for patients with rheumatoid arthritis (RA). While consensus T2T recommendations call for patient involvement in the treatment process, the targets commonly used to drive therapeutic decisions involve limited patient input. A pilot study was developed to explore whether the Patient-Reported Outcomes Measurement Information System (PROMIS) could add value to the T2T approach by providing a way to bring patient goals into the process. We report here the baseline data from this study. RA patients from an academic rheumatology practice were recruited to participate in this 1-year study. Patients were asked to complete PROMIS computer-assisted testing at quarterly visits during the year. At baseline, they were asked to identify the PROMIS domain (Pain Interference, Fatigue, Depression, Physical Function, and Social Function) that felt most important to their quality of life. They were then asked to select five representative items from this domain, to be followed through the year. Complete baseline data was available for 119 patients. Most selected Physical Function (39%) or Pain Interference (37%) as their highest priority PROMIS domain. Sixty percent ranked Depression as their lowest priority domain. Younger patients more frequently prioritized Social Function, while older patients more frequently prioritized Fatigue. The incorporation of PROMIS questionnaires into routine clinic visits is a feasible mechanism for incorporating patient preferences into a T2T approach to managing RA.
Revisiting the Surveillance Epidemiology and End Results Cancer Registry and Medicare Health Outcomes Survey (SEER-MHOS) Linked Data Resource for Patient-Reported Outcomes Research in Older Adults with Cancer.
Kent, Erin E; Malinoff, Rochelle; Rozjabek, Heather M; Ambs, Anita; Clauser, Steven B; Topor, Marie A; Yuan, Gigi; Burroughs, James; Rodgers, Anne B; DeMichele, Kimberly
Researchers and clinicians are increasingly recognizing the value of patient-reported outcome (PRO) data to better characterize people's health and experiences with illness and care. Considering the rising prevalence of cancer in adults aged 65 and older, PRO data are particularly relevant for older adults with cancer, who often require complex cancer care and have additional comorbid conditions. A data linkage between the Surveillance Epidemiology and End Results (SEER) cancer registry and the Medicare Health Outcomes Survey (MHOS) was created through a partnership between the National Cancer Institute and the Centers for Medicare and Medicaid Services that created the opportunity to examine PROs in Medicare Advantage enrollees with and without cancer. The December 2013 linkage of SEER-MHOS data included the linked data for 12 cohorts, bringing the number of individuals in the linked data set to 95,723 with cancer and 1,510,127 without. This article reviews the features of the resource and provides information on some descriptive characteristics of the individuals in the data set (health-related quality of life, body mass index, fall risk management, number of unhealthy days in the past month). Individuals without (n=258,108) and with (n=3,440) cancer (1,311 men with prostate cancer, 982 women with breast cancer, 689 with colorectal cancer, 458 with lung cancer) were included in the current descriptive analysis. Given increasing longevity, advances in effective therapies and earlier detection, and population growth, the number of individuals aged 65 and older with cancer is expected to reach more than 12 million by 2020. SEER-MHOS provides population-level, self-reported, cancer registry-linked data for person-centered surveillance research on this growing population.
Ryan, Colleen M; Lee, Austin F; Kazis, Lewis E; Shapiro, Gabriel D; Schneider, Jeffrey C; Goverman, Jeremy; Fagan, Shawn P; Wang, Chao; Kim, Julia; Sheridan, Robert L; Tompkins, Ronald G
Long-term follow-up care of survivors after burn injuries can potentially be improved by the application of patient-reported outcome measures (PROMs). PROMs can inform clinical decision-making and foster communication between the patient and provider. There are no previous reports using real-time, burn-specific PROMs in clinical practice to track and benchmark burn recovery over time. This study examines the feasibility of a computerized, burn-specific PROM, the Young Adult Burn Outcome Questionnaire (YABOQ), with real-time benchmarking feedback in a burn outpatient practice. The YABOQ was redesigned for formatting and presentation purposes using images and transcribed to a computerized format. The redesigned questionnaire was administered to young adult burn survivors (ages 19-30 years, 1-24 months from injury) via an ipad platform in the office before outpatient visits. A report including recovery curves benchmarked to a nonburned relatively healthy age-matched population and to patients with similar injuries was produced for the domains of physical function and social function limited by appearance. A copy of the domain reports as well as a complete copy of the patient's responses to all domain questions was provided for use during the clinical visit. Patients and clinicians completed satisfaction surveys at the conclusion of the visit. Free-text responses, included in the satisfaction surveys, were treated as qualitative data adding contextual information about the assessment of feasibility. Eleven patients and their providers completed the study for 12 clinical visits. All patients found the ipad survey and report "easy" or "very easy" to use. In nine instances, patients "agreed" or "strongly agreed" that it helped them communicate their situation to their doctor/nurse practitioner. Patients "agreed" or "strongly agreed" that the report helped them understand their course of recovery in 10 visits. In 11 visits, the patients "agreed" or "strongly agreed" that
The Electronic Patient Reported Outcome Tool: Testing Usability and Feasibility of a Mobile App and Portal to Support Care for Patients With Complex Chronic Disease and Disability in Primary Care Settings
Gill, Ashlinder; Khan, Anum Irfan; Hans, Parminder Kaur; Kuluski, Kerry; Cott, Cheryl
Background People experiencing complex chronic disease and disability (CCDD) face some of the greatest challenges of any patient population. Primary care providers find it difficult to manage multiple discordant conditions and symptoms and often complex social challenges experienced by these patients. The electronic Patient Reported Outcome (ePRO) tool is designed to overcome some of these challenges by supporting goal-oriented primary care delivery. Using the tool, patients and providers collaboratively develop health care goals on a portal linked to a mobile device to help patients and providers track progress between visits. Objectives This study tested the usability and feasibility of adopting the ePRO tool into a single interdisciplinary primary health care practice in Toronto, Canada. The Fit between Individuals, Fask, and Technology (FITT) framework was used to guide our assessment and explore whether the ePRO tool is: (1) feasible for adoption in interdisciplinary primary health care practices and (2) usable from both the patient and provider perspectives. This usability pilot is part of a broader user-centered design development strategy. Methods A 4-week pilot study was conducted in which patients and providers used the ePRO tool to develop health-related goals, which patients then monitored using a mobile device. Patients and providers collaboratively set goals using the system during an initial visit and had at least 1 follow-up visit at the end of the pilot to discuss progress. Focus groups and interviews were conducted with patients and providers to capture usability and feasibility measures. Data from the ePRO system were extracted to provide information regarding tool usage. Results Six providers and 11 patients participated in the study; 3 patients dropped out mainly owing to health issues. The remaining 8 patients completed 210 monitoring protocols, equal to over 1300 questions, with patients often answering questions daily. Providers and patients
Vainshtein, Jeffrey M. [Department of Radiation Oncology, University of Michigan, Ann Arbor, Michigan (United States); Griffith, Kent A. [Center for Cancer Biostatistics, University of Michigan School of Public Health, Ann Arbor, Michigan (United States); Feng, Felix Y.; Vineberg, Karen A. [Department of Radiation Oncology, University of Michigan, Ann Arbor, Michigan (United States); Chepeha, Douglas B. [Department of Otolaryngology–Head and Neck Surgery, University of Michigan, Ann Arbor, Michigan (United States); Eisbruch, Avraham, E-mail: firstname.lastname@example.org [Department of Radiation Oncology, University of Michigan, Ann Arbor, Michigan (United States)
Purpose: To describe voice and speech quality changes and their predictors in patients with locally advanced oropharyngeal cancer treated on prospective clinical studies of organ-preserving chemotherapy–intensity modulated radiation therapy (chemo-IMRT). Methods and Materials: Ninety-one patients with stage III/IV oropharyngeal cancer were treated on 2 consecutive prospective studies of definitive chemoradiation using whole-field IMRT from 2003 to 2011. Patient-reported voice and speech quality were longitudinally assessed from before treatment through 24 months using the Communication Domain of the Head and Neck Quality of Life (HNQOL-C) instrument and the Speech question of the University of Washington Quality of Life (UWQOL-S) instrument, respectively. Factors associated with patient-reported voice quality worsening from baseline and speech impairment were assessed. Results: Voice quality decreased maximally at 1 month, with 68% and 41% of patients reporting worse HNQOL-C and UWQOL-S scores compared with before treatment, and improved thereafter, recovering to baseline by 12-18 months on average. In contrast, observer-rated larynx toxicity was rare (7% at 3 months; 5% at 6 months). Among patients with mean glottic larynx (GL) dose ≤20 Gy, >20-30 Gy, >30-40 Gy, >40-50 Gy, and >50 Gy, 10%, 32%, 25%, 30%, and 63%, respectively, reported worse voice quality at 12 months compared with before treatment (P=.011). Results for speech impairment were similar. Glottic larynx dose, N stage, neck dissection, oral cavity dose, and time since chemo-IMRT were univariately associated with either voice worsening or speech impairment. On multivariate analysis, mean GL dose remained independently predictive for both voice quality worsening (8.1%/Gy) and speech impairment (4.3%/Gy). Conclusions: Voice quality worsening and speech impairment after chemo-IMRT for locally advanced oropharyngeal cancer were frequently reported by patients, underrecognized by clinicians, and
Vainshtein, Jeffrey M; Griffith, Kent A; Feng, Felix Y; Vineberg, Karen A; Chepeha, Douglas B; Eisbruch, Avraham
To describe voice and speech quality changes and their predictors in patients with locally advanced oropharyngeal cancer treated on prospective clinical studies of organ-preserving chemotherapy-intensity modulated radiation therapy (chemo-IMRT). Ninety-one patients with stage III/IV oropharyngeal cancer were treated on 2 consecutive prospective studies of definitive chemoradiation using whole-field IMRT from 2003 to 2011. Patient-reported voice and speech quality were longitudinally assessed from before treatment through 24 months using the Communication Domain of the Head and Neck Quality of Life (HNQOL-C) instrument and the Speech question of the University of Washington Quality of Life (UWQOL-S) instrument, respectively. Factors associated with patient-reported voice quality worsening from baseline and speech impairment were assessed. Voice quality decreased maximally at 1 month, with 68% and 41% of patients reporting worse HNQOL-C and UWQOL-S scores compared with before treatment, and improved thereafter, recovering to baseline by 12-18 months on average. In contrast, observer-rated larynx toxicity was rare (7% at 3 months; 5% at 6 months). Among patients with mean glottic larynx (GL) dose ≤20 Gy, >20-30 Gy, >30-40 Gy, >40-50 Gy, and >50 Gy, 10%, 32%, 25%, 30%, and 63%, respectively, reported worse voice quality at 12 months compared with before treatment (P=.011). Results for speech impairment were similar. Glottic larynx dose, N stage, neck dissection, oral cavity dose, and time since chemo-IMRT were univariately associated with either voice worsening or speech impairment. On multivariate analysis, mean GL dose remained independently predictive for both voice quality worsening (8.1%/Gy) and speech impairment (4.3%/Gy). Voice quality worsening and speech impairment after chemo-IMRT for locally advanced oropharyngeal cancer were frequently reported by patients, underrecognized by clinicians, and independently associated with GL dose. These findings support
Yasser El Miedany
Full Text Available Objectives. To assess the validity, reliability, and responsiveness to change of a patient self-reported questionnaire combining the Widespread Pain Index and the Symptom Severity Score as well as construct outcome measures and comorbidities assessment in fibromyalgia patients. Methods. The PROMs-FM was conceptualized based on frameworks used by the WHO Quality of Life tool and the PROMIS. Initially, cognitive interviews were conducted to identify item pool of questions. Item selection and reduction were achieved based on patients as well as an interdisciplinary group of specialists. Rasch and internal consistency reliability analyses were implemented. The questionnaire included the modified ACR criteria main items (Symptom Severity Score and Widespread Pain Index, in addition to assessment of functional disability, quality of life (QoL, review of the systems, and comorbidities. Every patient completed HAQ and EQ-5D questionnaires. Results. A total of 146 fibromyalgia patients completed the questionnaire. The PROMs-FM questionnaire was reliable as demonstrated by a high standardized alpha (0.886–0.982. Content construct assessment of the functional disability and QoL revealed significant correlation (p<0.01 with both HAQ and EQ-5D. Changes in functional disability and QoL showed significant (p<0.01 variation with diseases activity status in response to therapy. There was higher prevalence of autonomic symptoms, CVS risk, sexual dysfunction, and falling. Conclusions. The developed PROMs-FM questionnaire is a reliable and valid instrument for assessment of fibromyalgia patients. A phased treatment regimen depending on the severity of FMS as well as preferences and comorbidities of the patient is the best approach to tailored patient management.
Impact of efalizumab on patient-reported outcomes in high-need psoriasis patients: results of the international, randomized, placebo-controlled Phase III Clinical Experience Acquired with Raptiva (CLEAR trial [NCT00256139
Full Text Available Abstract Background Chronic psoriasis can negatively affect patients' lives. Assessing the impact of treatment on different aspects of a patient's health-related quality of life (HRQOL is therefore important and relevant in trials of anti-psoriasis agents. The recombinant humanized IgG1 monoclonal antibody efalizumab targets multiple T-cell-dependent steps in the immunopathogenesis of psoriasis. Efalizumab has demonstrated safety and efficacy in several clinical trials, and improves patients' quality of life. Objective: To evaluate the impact of efalizumab on HRQOL and other patient-reported outcomes in patients with moderate to severe plaque psoriasis, including a large cohort of High-Need patients for whom at least 2 other systemic therapies were unsuitable because of lack of efficacy, intolerance, or contraindication. Methods A total of 793 patients were randomized in a 2:1 ratio to receive efalizumab 1 mg/kg/wk (n = 529 or placebo (n = 264 for 12 weeks. The study population included 526 High-Need patients (342 efalizumab, 184 placebo. The treatment was evaluated by patients using the HRQOL assessment tools Short Form-36 (SF-36 and Dermatology Life Quality Index (DLQI. Other patient-reported assessments included the Psoriasis Symptom Assessment (PSA, a visual analog scale (VAS for itching, and the Patient's Global Psoriasis Assessment (PGPA. Results Efalizumab was associated with improvements at Week 12 from baseline in patient-reported outcomes, both in the total study population and in the High-Need cohort. Among all efalizumab-treated patients, the DLQI improved by 5.7 points from baseline to Week 12, relative to an improvement of 2.3 points for placebo patients (P P Conclusion A 12-week course of efalizumab improved HRQOL and other patient-reported outcomes in patients with moderate to severe plaque psoriasis. The benefits of efalizumab therapy in High-Need patients were similar to those observed in the total study population, indicating
Hahn, Elizabeth A; Burns, James L; Jacobs, Elizabeth A; Ganschow, Pamela S; Garcia, Sofia F; Rutsohn, Joshua P; Baker, David W
This study examined associations between patient characteristics, health behaviors, and health outcomes and explored the role of health literacy as a potential mediator of outcomes. English- and Spanish-speaking adults with Type 2 diabetes used a bilingual multimedia touchscreen to complete questionnaires. The behavioral model for vulnerable populations guided multivariable regression and mediation testing. Dependent variables were diabetes self-care, health status, and satisfaction with communication. Independent variables included sociodemographic and clinical characteristics, health literacy, health beliefs, and self-efficacy. Spanish speakers had lower health literacy and poorer physical, mental, and overall health compared to English speakers. Higher health literacy was associated with less social support for diet, fewer diet and medication barriers, younger age, higher diabetes knowledge, and talking with health care professionals to get diabetes information. In contrast to expectations, health literacy was not associated with diabetes self-care, health status, or satisfaction with communication, and it did not mediate the effects of other factors on these outcomes. Diabetes self-efficacy was significantly associated with health behaviors and outcomes. The association between Spanish language preference and poorer health was not mediated by this group's lower health literacy. Increasing health-related self-efficacy might be an important clinical strategy for improving outcomes in underserved patients with Type 2 diabetes.
This thesis addresses to what extent end stage renal disease (ESRD) and its treatment affects quality of life and social functioning in paediatric patients. Special emphasis is placed on the role of demographic and medical factors. Secondly, we studied the very long-term psychosocial outcomes of
Sharma, Shallu; Bünger, Cody Eric; Andersen, Thomas; Sun, Haolin; Wu, Chunsen; Hansen, Ebbe Stender
To examine correlation between postoperative radiographic and cosmetic improvements in Lenke 1C adolescent idiopathic scoliosis (AIS) with patients' self-rated outcomes of health and disability at follow-up as determined by the Scoliosis Research Society questionnaire (SRS-30), Oswestry Disability Index score (ODI) and measure of overall health quality Euroqol-5d (EQ-5D). 24 Lenke 1C scoliosis patients, mean age 16.5 (12.8-38.1) years, treated with posterior pedicle screw-only construct, were included. The coronal profile indices (radiographic and cosmetic) regarding magnitude of spinal deformity and truncal balance were measured preoperatively, postoperatively and at final follow-up. A comprehensive index of overall back symmetry was also measured by means of the Posterior Trunk Symmetry Index (POTSI). Pearson's correlation analysis determined the association between the radiographic-cosmetic indices and patient-rated outcomes. Mean follow-up for the cohort was 4.4 (±1.86) years. The thoracic apical vertebra-first thoracic vertebra horizontal distance (AV-TI) correction had significant correlation with function, self-image, and mental health SRS-30 scores (0.55, 0.54, 0.66). Similarly, thoracic apical vertebra horizontal translation from central sacral vertical line (AV-CSVL) correction at follow-up had significant correlation with self-image and management domains (0.57, 0.50). Follow-up POTSI correlated well with SRS-30 and EQ-5D scores (r = -0.64, -0.54). Postoperative leftward trunk shift/spinal imbalance did not influence overall cosmesis and outcomes; significant spinal realignment was evident in follow-up resulting in physiological balance and acceptable cosmesis and outcomes. Significant, but less than "perfect" correlations were observed between the radiographic, cosmetic measures and patient-rated outcomes. Thoracic AV-CSVL, AV-T1 correction and POTSI associated significantly with SRS-30 scores. Whereas, thoracic Cobb angle, Cobb correction, and
Jagsi, Reshma; Li, Yun; Morrow, Monica; Janz, Nancy; Alderman, Amy; Graff, John; Hamilton, Ann; Katz, Steven; Hawley, Sarah
Although breast conservation is therapeutically equivalent to mastectomy for most patients with early-stage breast cancer, an increasing number of patients are pursuing mastectomy, which may be followed by breast reconstruction. We sought to evaluate long-term quality of life and cosmetic outcomes after different locoregional management approaches, as perceived by patients themselves. We surveyed women with a diagnosis of nonmetastatic breast cancer from 2005 to 2007, as reported to the Los Angeles and Detroit population-based Surveillance, Epidemiology, and End Results registries. We received responses from 2290 women approximately 9 months after diagnosis (73% response rate) and from 1536 of these 4 years later. We evaluated quality of life and patterns and correlates of satisfaction with cosmetic outcomes overall and, more specifically, within the subgroup undergoing mastectomy with reconstruction, using multivariable linear regression. Of the 1450 patients who responded to both surveys and experienced no recurrence, 963 underwent breast-conserving surgery, 263 mastectomy without reconstruction, and 222 mastectomy with reconstruction. Cosmetic satisfaction was similar between those receiving breast conservation therapy and those receiving mastectomy with reconstruction. Among patients receiving mastectomy with reconstruction, reconstruction type and radiation receipt were associated with satisfaction (P mastectomy with reconstruction. In patients undergoing postmastectomy radiation, the use of autologous reconstruction may mitigate the deleterious impact of radiation on cosmetic outcomes.
Macefield, Rhiannon C; Reeves, Barnaby C; Milne, Thomas K; Nicholson, Alexandra; Blencowe, Natalie S; Calvert, Melanie; Avery, Kerry NL; Messenger, David E; Bamford, Richard; Pinkney, Thomas D; Blazeby, Jane M
Background: Surgical site infections (SSIs) are the third most common hospital-associated infection and can lead to significant patient morbidity and healthcare costs. Identification of SSIs is key to surveillance and research but reliable assessment is challenging, particularly after hospital discharge when most SSIs present. Existing SSI measurement tools have limitations and their suitability for post-discharge surveillance is uncertain. Aims: This study aimed to develop a single measure to identify SSI after hospital discharge, suitable for patient or observer completion. Methods: A three-phase mixed methods study was undertaken: Phase 1, an analysis of existing tools and semi-structured interviews with patients and professionals to establish the content of the measure; Phase 2, development of questionnaire items suitable for patients and professionals; Phase 3, pre-testing the single measure to assess acceptability and understanding to both stakeholder groups. Interviews and pre-testing took place over 12 months in 2014–2015 with patients and professionals from five specialties recruited from two UK hospital Trusts. Findings: Analyses of existing tools and interviews identified 19 important domains for assessing SSIs. Domains were developed into provisional questionnaire items. Pre-testing and iterative revision resulted in a final version with 16 items that were understood and easily completed by patients and observers (healthcare professionals). Conclusion: A single patient and observer measure for post-discharge SSI assessment has been developed. Further testing of the validity, reliability and accuracy of the measure is underway.
Lai, Hung-Wen; Lin, Hui-Yu; Chen, Shu-Ling; Chen, Shou-Tung; Chen, Dar-Ren; Kuo, Shou-Jen
Endoscopy-assisted breast surgery (EABS), a technique that optimizes cosmetic outcome because it is performed through small wounds hidden in inconspicuous areas, could be an alternative surgical technique for benign breast tumors. In this study, we report the preliminary results of 323 EABS procedures performed at our institution for the management of benign breast tumors. The medical records of patients who underwent EABS for benign breast lesions during the periods August 2010 to December 2015 were collected from the Changhua Christian Hospital EABS database. Data on clinicopathologic characteristics, type of surgery, hospital stay, and complications were analyzed to determine the effectiveness of the procedure for benign breast tumors. The operating time with the number of procedure performed was analyzed for learning curve evaluation. Patient satisfaction with cosmetic outcome was evaluated with a self-report questionnaire. A total of 323 EABS procedures were performed in 286 patients with benign breast lesions, including 249 (90.5%) patients with unilateral lesions. The mean age was 36 years, the mean tumor size was 2.2 cm, and the mean distance from the nipple to the tumor was 5.2 cm. Most (93.8%, 303/323) of these tumors were excised through a transareolar wound, 2.4% (8/323) through an axillary wound, and 0.3% (1/323) through the infra-mammary fold. Histopathologic analysis revealed that 63.5% (202/318) of the tumors were fibroadenoma-related lesions. The mean operative time was 81.4 min (59~89 min), which was decreased with experience increased. The overall rate of complications was 6.5%, and all were minor and wound-related. Among the 110 patients who participated in the self-report cosmetic outcome evaluation, 85.4% reported being satisfied with the cosmetic result, and almost all were satisfied with breast symmetry. Of the patients interviewed, 92.7% reported that they would choose the same procedure if they had to undergo the operation again. Our
Supporting Goal-Oriented Primary Health Care for Seniors with Complex Care Needs Using Mobile Technology: Evaluation and Implementation of the Health System Performance Research Network, Bridgepoint Electronic Patient Reported Outcome Tool
Wodchis, Walter P; Upshur, Ross; Cott, Cheryl; McKinstry, Brian; Mercer, Stewart; Palen, Ted E; Ramsay, Tim; Thavorn, Kednapa
Background Older adults experiencing multiple chronic illnesses are at high risk of hospitalization and health decline if they are unable to manage the significant challenges posed by their health conditions. Goal-oriented care approaches can provide better care for these complex patients, but clinicians find the process of ascertaining goals “too complex and too-time consuming,” and goals are often not agreed upon between complex patients and their providers. The electronic patient reported outcomes (ePRO) mobile app and portal offers an innovative approach to creating and monitoring goal-oriented patient-care plans to improve patient self-management and shared decision-making between patients and health care providers. The ePRO tool also supports proactive patient monitoring by the patient, caregiver(s), and health care provider. It was developed with and for older adults with complex care needs as a means to improve their quality of life. Objective Our proposed project will evaluate the use, effectiveness, and value for money of the ePRO tool in a 12-month multicenter, randomized controlled trial in Ontario; targeting individuals 65 or over with two or more chronic conditions that require frequent health care visits to manage their health conditions. Methods Intervention groups using the ePRO tool will be compared with control groups on measures of quality of life, patient experience, and cost-effectiveness. We will also evaluate the implementation of the tool. Results The proposed project presented in this paper will be funded through the Canadian Institute for Health Research (CIHR) eHealth Innovation Partnerships Program (eHIPP) program (CIHR–143559). The expected completion date of the study is November, 2019. Conclusions We anticipate our program of work will support improved quality of life and patient self-management, improved patient-centered primary care delivery, and will encourage the adoption of goal-oriented care approaches across primary
Linked Patient-Reported Outcomes Data From Patients With Multiple Sclerosis Recruited on an Open Internet Platform to Health Care Claims Databases Identifies a Representative Population for Real-Life Data Analysis in Multiple Sclerosis.
Risson, Valery; Ghodge, Bhaskar; Bonzani, Ian C; Korn, Jonathan R; Medin, Jennie; Saraykar, Tanmay; Sengupta, Souvik; Saini, Deepanshu; Olson, Melvin
An enormous amount of information relevant to public health is being generated directly by online communities. To explore the feasibility of creating a dataset that links patient-reported outcomes data, from a Web-based survey of US patients with multiple sclerosis (MS) recruited on open Internet platforms, to health care utilization information from health care claims databases. The dataset was generated by linkage analysis to a broader MS population in the United States using both pharmacy and medical claims data sources. US Facebook users with an interest in MS were alerted to a patient-reported survey by targeted advertisements. Eligibility criteria were diagnosis of MS by a specialist (primary progressive, relapsing-remitting, or secondary progressive), ≥12-month history of disease, age 18-65 years, and commercial health insurance. Participants completed a questionnaire including data on demographic and disease characteristics, current and earlier therapies, relapses, disability, health-related quality of life, and employment status and productivity. A unique anonymous profile was generated for each survey respondent. Each anonymous profile was linked to a number of medical and pharmacy claims datasets in the United States. Linkage rates were assessed and survey respondents' representativeness was evaluated based on differences in the distribution of characteristics between the linked survey population and the general MS population in the claims databases. The advertisement was placed on 1,063,973 Facebook users' pages generating 68,674 clicks, 3719 survey attempts, and 651 successfully completed surveys, of which 440 could be linked to any of the claims databases for 2014 or 2015 (67.6% linkage rate). Overall, no significant differences were found between patients who were linked and not linked for educational status, ethnicity, current or prior disease-modifying therapy (DMT) treatment, or presence of a relapse in the last 12 months. The frequencies of the
Linked Patient-Reported Outcomes Data From Patients With Multiple Sclerosis Recruited on an Open Internet Platform to Health Care Claims Databases Identifies a Representative Population for Real-Life Data Analysis in Multiple Sclerosis
Ghodge, Bhaskar; Bonzani, Ian C; Korn, Jonathan R; Medin, Jennie; Saraykar, Tanmay; Sengupta, Souvik; Saini, Deepanshu; Olson, Melvin
Background An enormous amount of information relevant to public health is being generated directly by online communities. Objective To explore the feasibility of creating a dataset that links patient-reported outcomes data, from a Web-based survey of US patients with multiple sclerosis (MS) recruited on open Internet platforms, to health care utilization information from health care claims databases. The dataset was generated by linkage analysis to a broader MS population in the United States using both pharmacy and medical claims data sources. Methods US Facebook users with an interest in MS were alerted to a patient-reported survey by targeted advertisements. Eligibility criteria were diagnosis of MS by a specialist (primary progressive, relapsing-remitting, or secondary progressive), ≥12-month history of disease, age 18-65 years, and commercial health insurance. Participants completed a questionnaire including data on demographic and disease characteristics, current and earlier therapies, relapses, disability, health-related quality of life, and employment status and productivity. A unique anonymous profile was generated for each survey respondent. Each anonymous profile was linked to a number of medical and pharmacy claims datasets in the United States. Linkage rates were assessed and survey respondents’ representativeness was evaluated based on differences in the distribution of characteristics between the linked survey population and the general MS population in the claims databases. Results The advertisement was placed on 1,063,973 Facebook users’ pages generating 68,674 clicks, 3719 survey attempts, and 651 successfully completed surveys, of which 440 could be linked to any of the claims databases for 2014 or 2015 (67.6% linkage rate). Overall, no significant differences were found between patients who were linked and not linked for educational status, ethnicity, current or prior disease-modifying therapy (DMT) treatment, or presence of a relapse in
Habitual functional electrical stimulation therapy improves gait kinematics and walking performance, but not patient-reported functional outcomes, of people with multiple sclerosis who present with foot-drop.
Marietta L van der Linden
Full Text Available BACKGROUND: People with Multiple Sclerosis (pwMS often experience a disturbed gait function such as foot-drop. The objective of this pilot study was to investigate the medium term effects of using Functional Electrical Stimulation (FES to treat foot-drop over a period 12 weeks on gait and patient reported outcomes of pwMS. METHODS AND FINDINGS: Nine pwMS aged 35 to 64 (2 males, 7 females were assessed on four occasions; four weeks before baseline, at baseline and after six weeks and twelve weeks of FES use. Joint kinematics and performance on the 10 meter and 2 minute walk tests (10WT, 2 minWT were assessed with and without FES. Participants also completed the MS walking Scale (MSWS, MS impact scale (MSIS29, Fatigue Severity Score (FSS and wore an activity monitor for seven days after each assessment. Compared to unassisted walking, FES resulted in statistically significant improvements in peak dorsiflexion in swing (p = 0.006, 10MWT (p = 0.006 and 2 minWT (p = 0.002. Effect sizes for the training effect, defined as the change from unassisted walking at baseline to that at 12 weeks, indicated improved ankle angle at initial contact (2.6°, 95% CI -1° to 4°, d = 0.78, and a decrease in perceived exertion over the 2 min walking tests (-1.2 points, 95% CI -5.7 to 3.4, d = -0.86. Five participants exceeded the Minimally Detectable Change (MDC for a training effect on the 10mWT, but only two did so for the 2 minWT. No effects of the use of FES for 12 weeks were found for MSWS, MSIS29, FSS or step count. CONCLUSION: Although FES to treat foot-drop appears to offer the potential for a medium term training effect on ankle kinematics and walking speed, this was not reflected in the patient reported outcomes. This observed lack of relationship between objective walking performance and patient reported outcomes warrants further investigation. TRIAL REGISTRATION: ClinicalTrials.gov NCT01977287.
Bluett, James; Barton, Anne; Hyrich, Kimme L; Cordingley, Lis; Verstappen, Suzanne M M
Treatment response to methotrexate (MTX) for rheumatoid arthritis (RA) is not universal and non-adherence may partially explain this. The aims of this systematic review were to: (1) summarise existing rates of adherence to MTX, (2) identify predictors of adherence to MTX, and (3) assess the association between non-adherence and patient outcomes. The authors conducted a systematic search of papers published from January 1980 to February 2015 in PubMed, PsycINFO, EMBASE and CINAHL databases. Studies were eligible for inclusion if: (1) MTX was used as monotherapy or in combination with other therapies, (2) MTX was used in an RA or inflammatory polyarthritis population, (3) adherence was defined and measured as the extent to which patients followed their MTX regimen during the period of prescription, and (4) it was an original piece of research. In total, 10 studies met the inclusion criteria and 8 were evaluated as high quality. Rates of adherence ranged from 59% to 107%, and exposed differences in definitions of adherence, study methodologies and sample heterogeneity. A number of potential predictors of MTX adherence were identified; the strongest being related to beliefs in the necessity and efficacy of MTX, absence of low mood, mild disease and MTX monotherapy. Furthermore, 3 studies tested the association of adherence with disease activity as an outcome measure; all 3 found non-adherence associated with poor treatment response. This systematic review shows the importance of adherence to MTX treatment and summarises the associated modifiable factors. PMID:26848403
Full Text Available Hip resurfacing has been considered a good treatment option for younger, active osteoarthritis patients. However, there are several identified issues concerning risk for neck fractures and issues related to current metal-on-metal implant designs. Neck-preserving short-stem implants have been discussed as a potential alternative, but it is yet unclear which method is better suited for younger adults. We compared hip disability and osteoarthritis outcome scores (HOOS from a young group of patients (n=52, age 48.9 ± 6.1 years who had received hip resurfacing (HR with a cohort of patients (n=73, age 48.2 ± 6.6 years who had received neck-preserving, short-stem implant total hip arthroplasty (THA. Additionally, durations for both types of surgery were compared. HOOS improved significantly preoperatively to last followup (>1 year in both groups (p<0.0001, η2=0.69; there were no group effects or interactions. Surgery duration was significantly longer for resurfacing (104.4 min ± 17.8 than MiniHip surgery (62.5 min ± 14.8, U=85.0, p<0.0001, η2=0.56. The neck-preserving short-stem approach may be preferable to resurfacing due to the less challenging surgery, similar outcome, and controversy regarding resurfacing implant designs.
Shaikh, Talha; Li, Tianyu; Handorf, Elizabeth A; Johnson, Matthew E; Wang, Lora S; Hallman, Mark A; Greenberg, Richard E; Price, Robert A; Uzzo, Robert G; Ma, Charlie; Chen, David; Geynisman, Daniel M; Pollack, Alan; Horwitz, Eric M
To assess the long-term quality of life (QoL) outcomes from a phase 3 trial comparing 2 modes of intensity modulated radiation therapy (IMRT): conventional IMRT (CIMRT) versus hypofractionated IMRT (HIMRT) in patients with localized prostate cancer. Between 2002 and 2006, 303 men with low-risk to high-risk prostate cancer were randomized to 76 Gy in 38 fractions (CIMRT) versus 70.2 Gy in 26 fractions (HIMRT). QoL was compared by use of the Expanded Prostate Cancer Index Composite (EPIC), the International Prostate Symptom Score (IPSS), and EuroQoL (EQ5D) questionnaires. The primary outcome of the QoL analysis was a minimum clinically important difference defined as a 0.5 standard deviation change from baseline for each respective QoL parameter. Treatment effects were evaluated with the use of logistic mixed effects regression models. A total of 286, 299, and 218 patients had baseline EPIC, IPSS, or EQ5D data available and were included in the analysis. Overall, there was no statistically significant difference between the 2 treatment arms in terms of EPIC, IPSS, or EQ5D scores over time, although there was a trend toward lower EPIC urinary incontinence scores in the HIMRT arm. More patients in the HIMRT arm had a lower EPIC urinary incontinence score relative to baseline versus patients in the CIMRT arm with long-term follow-up. On multivariable analysis, there was no association between radiation fractionation scheme and any QoL parameter. When other clinical factors were examined, lymph node radiation was associated with worse EPIC hormonal scores versus patients receiving no lymph node radiation. In general, QoL outcomes were generally stable over time, with the exception of EPIC hormonal and EQ5D scores. In this randomized prospective study, there were stable QoL changes in patients receiving HIMRT or CIMRT. Our results add to the growing body of literature suggesting that HIMRT may be an acceptable treatment modality in clinically localized prostate cancer
Ibsen, Charlotte; Schiøttz-Christensen, Berit; Melchiorsen, Hanne
OBJECTIVE: To link the items in the Patient-Reported Outcome Measures (PROMs): Roland Morris Disability Questionnaire, Short Form 36 (SF-36) and pain scores, to the Brief International Classification of Functioning, Disability and Health (ICF) Core Set for low back pain, and to examine the extent...... to which a clinician's assessment of patients' problems according to the Brief ICF Core Set correlates with the scores of matching items from the PROMs. METHODS: The PROMs were linked to the Brief ICF Core Set for low back pain. Secondly, a cross-sectional study was conducted including 70 patients with low...... back pain. The patients completed the PROMs, and the Brief ICF Core Set for low back pain was assessed by a clinician using qualifiers. RESULTS: The items in the PROMs were successfully linked to the ICF. Twelve of the 38 unique ICF categories derived from the PROMs were covered by the Brief ICF Core...
Radiation Therapy Versus No Radiation Therapy to the Neo-breast Following Skin-Sparing Mastectomy and Immediate Autologous Free Flap Reconstruction for Breast Cancer: Patient-Reported and Surgical Outcomes at 1 Year-A Mastectomy Reconstruction Outcomes Consortium (MROC) Substudy.
Cooke, Andrew L; Diaz-Abele, Julian; Hayakawa, Tom; Buchel, Ed; Dalke, Kimberly; Lambert, Pascal
To determine whether adjuvant radiation therapy (RT) is associated with adverse patient-reported outcomes and surgical complications 1 year after skin-sparing mastectomy and immediate autologous free flap reconstruction for breast cancer. We compared 24 domains of patient-reported outcome measures 1 year after autologous reconstruction between patients who received adjuvant RT and those who did not. A total of 125 patients who underwent surgery between 2012 and 2015 at our institution were included from the Mastectomy Reconstruction Outcomes Consortium study database. Adjusted multivariate models were created incorporating RT technical data, age, cancer stage, estrogen receptor, chemotherapy, breast size, body mass index, and income to determine whether RT was associated with outcomes. At 1 year after surgery, European Organisation for Research and Treatment of Cancer (EORTC) Breast Cancer-Specific Quality of Life Questionnaire breast symptoms were significantly greater in 64 patients who received RT (8-point difference on 100-point ordinal scale, PBREAST-Q (Post-operative Reconstruction Module), Patient-Report Outcomes Measurement Information System Profile 29, McGill Pain Questionnaire-Short Form (MPQ-SF) score, Generalized Anxiety Disorder Scale, and Patient Health Questionnaire-were not statistically different between groups. Surgical complications were uncommon and did not differ by treatment. RT to the neo-breast compared with no RT following immediate autologous free flap reconstruction for breast cancer is well tolerated at 1 year following surgery despite patients undergoing RT also having a higher cancer stage and more intensive surgical and systemic treatment. Neo-breast symptoms are more common in patients receiving RT by the EORTC Breast Cancer-Specific Quality of Life Questionnaire but not by the BREAST-Q. Patient-reported results at 1 year after surgery suggest RT following immediate autologous free flap breast reconstruction is well tolerated
Corcos, Jacques; Angulo, Javier C; Garely, Alan D; Carlsson, Martin; Gong, Jason; Guan, Zhonghong
To assess the onset of efficacy of fesoterodine 4 mg versus placebo in subjects with overactive bladder (OAB) symptoms. Subjects who reported OAB symptoms for ≥ 3 months and recorded ≥ 8 micturitions and ≥ 1 urgency urinary incontinence (UUI) episode per 24 hours in 3-day baseline diaries were randomized to fesoterodine 4 mg, tolterodine extended release (ER) 4 mg, or placebo. This is an analysis of first week data from a 12-week, double-blind trial. ClinicalTrials.gov unique ID: NCT00444925. Baseline to week 1 changes in 3-day bladder diary variables, Patient Perception of Bladder Condition (PPBC), and Urgency Perception Scale (UPS) scores reported by subjects receiving fesoterodine 4 mg or placebo. By week 1, fesoterodine 4 mg (n = 679) was associated with significantly greater improvements compared with placebo (n = 334) in micturitions, urgency, severe urgency and UUI episodes, frequency-urgency sum, and MVV per 24 hours and 3-day diary-dry rate (all p fesoterodine 4 mg versus placebo (p = 0.0143); changes in UPS scores were not significantly different (p = 0.077). The results provide evidence that patients receiving fesoterodine 4 mg for their OAB symptoms may expect to experience a response as early as 1 week after initiating treatment. One limitation is that, although 65% of subjects had received treatment with antimuscarinics before the study, whether subjects were dissatisfied with previous treatment and reasons for dissatisfaction were not collected. This might affect the magnitude of outcome improvements. Also, it is not known whether the UPS is sensitive enough to detect treatment differences as early as week 1.
Full Text Available Study Objective. To measure the association of symptoms attributed to residual effects of sleep medication (e.g., drowsiness, difficulty concentrating, and impaired memory on self-reported functioning and satisfaction with these medications. Methods. Individuals using prescription medications for insomnia were invited to complete an Internet-based survey. Respondents were compared according to the presence of self-reported residual effects; relationships between severity of these effects and outcomes were modeled using regression. Measures included the Brief Insomnia Questionnaire, Work Productivity and Activity Impairment Questionnaire, and SATMED-Q. Subgroup analyses were conducted with patients aged ≥65 years. Approximately 80% reported experiencing ≥1 residual effect. The severity of residual effects was associated with increased residual effect-related work impairment, including absenteeism (RR = 1.46, p<0.001, presenteeism (RR = 1.12, p<0.001, overall work impairment (RR = 1.13, p<0.001, and nonwork activity impairment (RR = 1.11, p<0.001. More severe residual symptoms were also associated with increased difficulty in home management (Beta = .31, p<0.001, ability to work (Beta = .31, p<0.001, social relationships, (Beta = .32, p<0.001, close personal relationships (Beta = .30, p<0.001, and lower medication satisfaction (Beta = -.37, p<0.001. Conclusions. Individuals using medications for insomnia commonly experience symptoms considered as residual effects, and these symptoms are associated with greater interference of sleep-related problems at work, at home, and with social relationships.
Gautschi, Oliver P; Joswig, Holger; Corniola, Marco V; Smoll, Nicolas R; Schaller, Karl; Hildebrandt, Gerhard; Stienen, Martin N
The Timed Up and Go (TUG) test is a reliable tool for evaluating objective functional impairment (OFI) in patients with degenerative disc disease before a surgical intervention. The aim of this study is to assess the validity of the TUG test to measure change in function postoperatively. In a prospective two-center study, OFI was assessed by the TUG test in patients scheduled for lumbar spine surgery, as well as 3 days (D3) and 6 weeks (W6) postoperatively. At each time point, the TUG test results were correlated with established subjective measures of pain intensity (visual analogue scale (VAS) for back and leg pain), functional impairment (Oswestry Disability Index (ODI)) and health-related quality of life (HRQoL; Short Form-12 (SF12)). The patient cohort comprised 136 patients with a mean age of 57.7 years; 76 were males, 54 had a microdiscectomy for lumbar disc herniation, 58 a decompression for a lumbar spinal stenosis, 24 had a surgical fusion procedure. The mean OFI t-score was 125.1 before surgery, and as patients improved on the subjective measures in the postoperative interval, the OFI t-score likewise decreased to 118.8 (D3) and 103.4 (W6). The Pearson correlation coefficient (PCC) between the OFI t-score and VAS leg pain was 0.187 preoperatively (p = 0.029) and 0.252 at W6 (p = 0.003). The PCC between OFI t-score and the ODI was 0.324 preoperatively (p < 0.001) and 0.413 at W6 (p < 0.001). The PCC between OFI t-score and physical HRQoL (SF12) was -0.091 preoperatively (p = 0.293) and -0.330 at W6 (p < 0.001). The TUG test is sensitive to change, and reflects the postoperative functional outcome even more exact than preoperatively, as indicated by better correlation coefficients of the OFI t-score with subjective measures of pain intensity, functional impairment and HRQoL.
Rinkel, Rico N; Verdonck-de Leeuw, Irma M; Doornaert, Patricia; Buter, Jan; de Bree, Remco; Langendijk, Johannes A; Aaronson, Neil K; Leemans, C René
The objective of this study is to assess swallowing and speech outcome after chemoradiation therapy for head and neck cancer, based on the patient-reported outcome measures Swallowing Quality of Life Questionnaire (SWAL-QOL) and Speech Handicap Index (SHI), both provided with cut-off scores. This is a cross-sectional study. Department of Otolaryngology/Head and Neck Surgery of a University Medical Center. Sixty patients, 6 months to 5 years after chemoradiation for head and neck squamous cell carcinoma. Swallowing Quality of Life Questionnaire (SWAL-QOL) and SHI, both validated in Dutch and provided with cut-off scores. Associations were tested between the outcome measures and independent variables (age, gender, tumor stage and site, and radiotherapy technique, time since treatment, comorbidity and food intake). Fifty-two patients returned the SWAL-QOL and 47 the SHI (response rate 87 and 78 %, respectively). Swallowing and speech problems were present in 79 and 55 %, respectively. Normal food intake was noticed in 45, 35 % had a soft diet and 20 % tube feeding. Patients with soft diet and tube feeding reported more swallowing problems compared to patients with normal oral intake. Tumor subsite was significantly associated with swallowing outcome (less problems in larynx/hypopharynx compared to oral/oropharynx). Radiation technique was significantly associated with psychosocial speech problems (less problems in patients treated with IMRT). Swallowing and (to a lesser extent) speech problems in daily life are frequently present after chemoradiation therapy for head and neck cancer. Future prospective studies will give more insight into the course of speech and swallowing problems after chemoradiation and into efficacy of new radiation techniques and swallowing and speech rehabilitation programs.
Arnold, Benjamin; Mitchell, Sandra A; Lent, Lauren; Mendoza, Tito R; Rogak, Lauren J; Barragán, Natalie M; Willis, Gordon; Medina, Mauricio; Lechner, Suzanne; Penedo, Frank J; Harness, Jay K; Basch, Ethan M
The U.S. NCI's PRO-CTCAE is a library of self-report items for assessing symptomatic adverse events in cancer clinical trials from the patient perspective. The aim of this study was to translate and linguistically validate a Spanish version. PRO-CTCAE's 124 items were translated from English into Spanish using multiple forward and back translations. Native Spanish speakers undergoing cancer treatment were enrolled at six cancer treatment sites. Participants each completed approximately 50 items and were then interviewed using cognitive probes. The interviews were analyzed at the item level by linguistic themes, and responses were examined for evidence of equivalence to English. Items for which ≥20 % of participants experienced difficulties were reviewed, and phrasing was revised and then retested in subsequent interviews. Items where difficulties were also reviewed and were considered for rephrasing and retesting. One hundred nine participants from diverse Spanish-speaking countries were enrolled (77 in Round 1 and 32 in Round 2). A majority of items we