Patient-reported Outcome Measures Of Quality Research Articles

Patient-reported measures of tinnitus for individuals with neurofibromatosis type 2-related schwannomatosis: Recommendations for clinical trials.

Neurofibromatosis type 2-related schwannomatosis is a genetic disease characterized by the development of bilateral vestibular schwannomas, ependymomas, meningiomas, and cataracts. Mild to profound hearing loss and tinnitus are common symptoms reported by individuals with neurofibromatosis type 2. While tinnitus is known to have a significant and negative impact on the quality of life of individuals from the general population, the impact on individuals with neurofibromatosis type 2 is unknown. Consensus regarding the selection of suitable patient-reported outcome measures for assessment could advance further research into tinnitus in neurofibromatosis type 2 patients. The purpose of this work is to achieve a consensus recommendation by the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration for patient-reported outcome measures used to evaluate quality of life in the domain of tinnitus for neurofibromatosis type 2 clinical trials. The Response Evaluation in Neurofibromatosis and Schwannomatosis Patient-Reported Outcomes Communication Subgroup systematically evaluated patient-reported outcome measures of quality of life in the domain of tinnitus for individuals with neurofibromatosis type 2 using previously published Response Evaluation in Neurofibromatosis and Schwannomatosis rating procedures. Of the 19 identified patient-reported outcome measures, 3 measures were excluded because they were not validated as an outcome measure or could not have been used as a single outcome measure for a clinical trial. Sixteen published patient-reported outcome measures for the domain of tinnitus were scored and compared on their participant characteristics, item content, psychometric properties, and feasibility for use in clinical trials. The Tinnitus Functional Index was identified as the most highly rated measure for the assessment of tinnitus in populations with neurofibromatosis type 2, due to strengths in the areas of item content, psychometric properties, feasibility, and available scores. Response Evaluation in Neurofibromatosis and Schwannomatosis currently recommends the Tinnitus Functional Index for the assessment of tinnitus in neurofibromatosis type 2 clinical trials.

The Validity of Patient-Reported Outcome Measures of Quality of Life in Palliative Care: A Systematic Review.

Importance: A recent systematic review and meta-analysis found that palliative care was not associated with improvement in quality of life (QOL) in terminal noncancer illness. Among potential reasons for a null effect, it is unclear if patient-reported outcome measures (PROMs) measuring QOL were derived or validated among populations with advanced life-limiting illness (ALLI). Objective: To systematically review the derivation and validation of QOL PROMs from a recent meta-analysis of randomized controlled trials (RCT) of palliative care interventions in people with terminal noncancer illness. Evidence Review: EMBASE, MEDLINE, and PsycINFO were searched from inception to January 8, 2023 for primary validation studies of QOL PROMs in populations with ALLI, defined as adults with a progressive terminal condition and an estimated median survival of less than or equal to one year. The primary outcome was the proportion of PROMs that were derived or validated in ≥1 ALLI population. Findings: Twenty-one unique studies of derivation (n = 13) and validation (n = 11, 3 studies evaluated both) provided data on 9657 participants (mean age 63 years, 50% female) across 15 unique QOL PROMs and subscales. Among studies of validation, 9 were in people with cancer (n = 2289, n = 5 PROMs), 1 in neurodegenerative disease (n = 23, n = 1 PROM), and 1 with mixed diseases (n = 248, n = 1 PROM). Across 15 QOL PROMs and subscales, 47% (n = 7) were derived or validated in an ALLI population. The majority of these seven PROMs were exclusively derived or validated among people with cancer (57%, n = 4). QOL PROMs such as Quality of Life at End of Life, EuroQoL-5 Dimension 5-level, and 36-item Short Form Survey demonstrated validity in more than one terminal noncancer illness. Conclusions: Most QOL PROMs that measured the effect of palliative care on QOL in RCTs were neither derived nor validated in an ALLI population. These findings raise questions about the inferences that palliative care does not improve QOL among people with terminal noncancer illness.

Abstract 13728: Generation of a Patient-Reported Outcome Measure of Quality of Life in Heart Valve Disease

Introduction: Heart valve disease (HVD) is common globally, yet there is no questionnaire measuring quality of life (QOL) across the lifespan of HVD. This limits assessment of changes in QOL in response to intervention in HVD. Research Question: What are all features of QOL in lived HVD, and how may these be best selected between to create a well-performing questionnaire? Goal: We aimed to generate a patient-reported outcome measure of QOL in common and significant types of HVD: aortic stenosis, mitral regurgitation, mitral valve disease in rheumatic heart disease, and valve repair/replacement. Methods: Participants were recruited from echocardiographic databases in two tertiary surgical centres in three phases. Phase One described QOL in interviews; Phase Two pilot-tested the questionnaire; and Phase Three selected between items using item distributions, exploratory factor analysis (EFA), and item response theory (IRT) to create the final questionnaire, called the Valve Quality of life (VALVQ). Results: In Phase One, 34 people with HVD, three family members, and five clinical experts were interviewed. Key features of QOL were physical capacity relative to ‘normal’, symptom interpretation causing reduced activity, and fatigue. In Phase Two, we showed in 62 participants that the resulting questionnaire - and its semantic differential item format - was functional. In Phase Three, 290 participants returned questionnaires. Items were removed for poor distributions, then EFA identified ‘physical capacity’ and ‘disease perception’ as key factors. A two-parameter Graded Response Model was used to generate the final 12-item VALVQ, which is shown in the attached table. Conclusion: Through application of sequential analyses across three phases, we generated the final 12-item VALVQ. It has two factors - physical capacity and disease perception - and can be deployed for future research on QOL in HVD, whether before or after intervention and across multiple forms of HVD.

Patient-Reported Outcome Measures of Quality of Life in People Affected by Diabetic Foot: A Psychometric Systematic Review

This psychometric systematic review aimed to identify the most suitable patient-reported outcome measures (PROMs) of quality of life (QoL) in people affected by diabetic foot. We performed a literature search in MEDLINE (PubMed), CINAHL (EBSCOhost), and PsycINFO (EBSCOhost) databases from inception to February 1, 2022. We also searched gray literature databases. Eligible studies were full-text reports developing a QoL condition-specific PROM or assessing one or more of its measurement properties in people affected by diabetic foot. We assessed the methodological quality of included studies independently using the "Consensus-Based Standards for the Selection of Health Measurement Instruments Risk of Bias" checklist. The measurement properties were evaluated using specific criteria. We graded the quality of the evidence using a "Grading of Recommendations Assessment, Development and Evaluation" approach modified by Consensus-Based Standards for the Selection of Health Measurement Instruments. Forty-three reports (46 studies) providing information on the measurement properties of 10 different PROMs were included. We did not identify any instruments that could be recommended for use. We identified 2 PROMs that were not recommended for use and 8 that were potentially recommended but would require further investigation. Of these 8 PROMs, 4 had better evidence for content validity. Available PROMs to measure QoL in people affected by diabetic foot have limited evidence for their measurement properties. There is no fully suitable PROM. Pending further evidence, 4 PROMs could potentially be recommended for use.

Patient-Reported Outcome Measures in Dermatology: A Systematic Review.

By relying on data from existing patient-reported outcome measures of quality of life, the true impact of skin conditions on patients’ lives may be underestimated. This study systematically reviewed all dermatology-specific (used across skin conditions) patient-reported outcome measures and makes evidence-based recommendations for their use. The study protocol is registered on PROSPERO (CRD42018108829). PubMed, PsycInfo and CINAHL were searched from inception to 25 June 2018. The Consensus-based Standards for the Selection of Health Measurement Instruments (COSMIN) criteria were used to assess the measurement properties and methodological quality of studies. A total of 12,925 abstracts were identified. Zero patient-reported outcome measures were assigned to category A (ready for use without further validation), 31 to category B (recommended for use, but only with further validation) and 5 to category C (not recommended for use). There is no gold-standard dermatology-specific patient-reported outcome measure that can be recommended or used without caution. A new measure that can comprehensively capture the impact of dermatological conditions on the patient’s life is needed.

A Comparison of Patient-Reported Outcome Measures of Quality of Life By Dialysis Modality in the Treatment of Kidney Failure: A Systematic Review.

Background:There is an increasing demand to incorporate patient-reported outcome measures (PROMs) such as quality of life (QOL) in decision-making when selecting a chronic dialysis modality.Objective:To compare the change in QOL over time among similar patients on different dialysis modalities to provide unique and novel insights on the impact of dialysis modality on PROMs.Design:Systematic reviews, randomized controlled trials, and nonrandomized controlled trials were examined via a comprehensive search strategy incorporating multiple bibliographic databases.Setting:Data were extracted from relevant studies from January 1, 2000 to December 31, 2019 without limitations on country of study conduction.Patients:Eligible studies included adults (≥18 years) with end-stage kidney disease of any cause who were prescribed dialysis treatment (either as lifetime treatment or bridge to transplant).Measurements:The 5 comparisons were peritoneal dialysis (PD) vs in-center hemodialysis (ICHD), home hemodialysis (HHD) vs ICHD, HHD modalities compared with one another, HHD vs PD, and self-care ICHD vs traditional nurse-based ICHD.Methods:Included studies compared adults on different dialysis modalities with repeat measures within individuals to determine changes in QOL between dialysis modalities (in-center or home dialysis). Methodological quality was assessed by the Scottish Intercollegiate Guidelines Network (SIGN 50) checklist. A narrative synthesis was conducted, synthesizing the direction and size of any observed effects across studies.Results:Two randomized controlled trials and 9 prospective cohort studies involving a combined total of 3711 participants were included. Comparing PD and ICHD, 5 out of 9 studies found significant differences (P < .05) favoring PD in the change of multiple QOL domains, including “physical component score,” “role of social component score,” “cognitive status,” “role limitation due to emotional function,” “role limitation due to physical function,” “bodily pain,” “burden of kidney disease,” “effects of kidney disease on daily life,” “symptoms/problems,” “sexual function,” “finance,” and “patient satisfaction.” Conversely, 3 of these studies demonstrated statistically significant differences (P < .05) favoring ICHD in the domains of “role limitation due to physical function,” “general health,” “support from staff,” “sleep quality,” “social support,” “health status,” “social interaction,” “body image,” and “overall health.” Comparing HHD and ICHD, significant differences (P < .05) favoring HHD for the QOL domains of “general health,” “burden of kidney disease,” and the visual analogue scale were reported.Limitations:Our study is constrained by the small sample sizes of included studies, as well as heterogeneity among both study populations and validated QOL scales, limiting inter-study comparison.Conclusions:We identified differences in specific QOL domains between dialysis modalities that may aid in patient decision-making based on individual priorities.Trial registration:PROSPERO Registration Number: CRD42016046980.Primary funding source:The original research for this study was derived from the Canadian Agency for Drugs and Technologies in Health (CADTH) 2017 optimal use report, titled “Dialysis Modalities for the Treatment of End-Stage Kidney Disease: A Health Technology Assessment.” The CADTH receives funding from Canada’s federal, provincial, and territorial governments, with the exception of Quebec.

Poor quality patient reported outcome measures bias effect estimates in orthopaedic randomized studies

ObjectivesThe objective was to assess the potential for biased treatment effects associated with patient-reported outcome measures (PROMs) of varying psychometric quality in randomized clinical trials (RCTs) for rotator cuff disease (RCD). Study Design and SettingWe searched for RCTs published in the past 5 years (January 2011 to December 2016) in the top five 2015 impact factor orthopedic journals. We accepted RCTs including human participants with RCD, published in English, and using PROMs specific to RCD. We extracted data on study design, sample size, risk of bias for RCTs, quality of PROM used, estimates of effect, and associated measures of variance. PROMs were given numerical ratings of psychometric quality from a prior publication. Continuous measures of effect were transformed by dividing the effect estimate by the standard deviation. Multilevel linear regression analyses were performed to determine whether PROM quality was associated with the magnitude of effect. ResultsOverall, we included 72 RCTs reporting 174 separate outcomes. Mean sample size was 66.8 (95% CI 62.30 to 71.27), mean risk of bias score across all studies was 7.00/10 (95% CI 6.72 to 7.29), psychometric quality summary scores ranged from −2 to 10, and the standardized mean effect estimate was 0.47 (95% CI −0.17 to 1.11). Regression revealed that higher-quality PROMs had smaller estimates of effect (β = −0.32; 95% CI −0.51 to −0.13; P = 0.001). We also found that a longer follow-up period predicted slightly increased effect estimates (β = 0.08; 95% CI 0.02 to 0.13; P = 0.007). ConclusionsPROMs with poor or unknown psychometric properties overestimate treatment effects in clinical research of RCD by 68.4% (β −0.32/standardized mean effect 0.47). To our knowledge, this is the first empirical evidence that variations in the quality of PROMs bias treatment effect estimates. Researchers and clinicians using data from PROMs must be cautious to explore the quality of that measure so as to not mislead decision-making resulting from biased outcomes.

Psychometric validation of a multi-dimensional capability instrument for outcome measurement in mental health research (OxCAP-MH)

BackgroundPatient reported outcome measures (PROMs) are widely used in mental healthcare research for quality of life assessment but most fail to capture the breadth of health and non-health domains that can be impacted. We report the psychometric validation of a novel, multi-dimensional instrument based on Amartya Sen’s capability approach intended for use as an outcome measure in mental health research.MethodsThe Oxford Capabilities Questionnaire for Mental Health (OxCAP-MH) is a 16-item self-complete capability measure that covers multiple domains of functioning and welfare. Data for validation of the instrument were collected through a national randomised controlled trial of community treatment orders for patients with psychosis. Complete OxCAP-MH data were available for 172 participants. Internal consistency was established with Cronbach’s alpha; an interclass correlation coefficient was used to assess test-retest reliability in a sub-sample (N = 50) tested one week apart. Construct validity was established by comparing OxCAP-MH total scores with established instruments of illness severity and functioning: EuroQol (EQ-5D), Brief Psychiatric Rating Scale (BPRS), Global Assessment of Functioning (GAF) and Objective Social Outcomes Index (SIX). Sensitivity was established by calculating standard error of measurement using distributional methods.ResultsThe OxCAP-MH showed good internal consistency (Cronbach’s alpha 0.79) and test-retest reliability (ICC = 0.86). Convergent validity was evidenced by strong correlations with the EQ-5D (VAS 0.52, p < .001) (Utility 0.45, p < .001), and divergent validity through more modest associations with the BPRS (−0.41, p < .001), GAF (0.24, p < .001) and SIX (0.12, p = ns). A change of 9.2 points on a 0–100 scale was found to be meaningful on statistical grounds.ConclusionsThe OxCAP-MH has demonstrable reliability and construct validity and represents a promising multi-dimensional alternative to existing patient-reported outcome measures for quality of life used in mental health research.

Patient-centered quality of life measures after alloplastic temporomandibular joint replacement surgery

The purpose of this study was to evaluate patient-reported outcome measures of quality of life (QoL) for patients with end-stage temporomandibular joint (TMJ) disease who have undergone TMJ prosthetic replacement. The records of 36 patients who had undergone alloplastic total joint replacement procedures were analyzed. Patients were treated using either TMJ Concepts or Biomet/Lorenz prosthetics. Patients were asked to complete a 12-item TMJ-S-QoL survey, which encompassed questions pertaining to pain, speech, chewing function, and various aspects of social life and mental health. The questions were answered on a 5-point scale. Data were analyzed using the Wilcoxon signed-rank test. Among the 36 patients (six male and 30 female), 18 responded to the survey. Markers of QoL after surgery were compared to the preoperative period. Significant improvements were reported for pain (94.4% of patients), chewing (83.3% of patients), speech (55.6% of patients), anxiety (72.2% of patients), activity (66.7% of patients), recreation (61.1% of patients), and mood (66.7% of patients) (all P<0.05). TMJ prosthetic replacement significantly enhanced QoL among patients suffering from chronic pain, limited range of motion, anxiety, impaired speech, and chewing due to end-stage TMJ disease in this sample of surgical patients.

Leveraging Outcomes Research to Optimize Prostate Cancer Care

IntroductionTreatment of localized prostate cancer is subject to patient preference as there are multiple acceptable options with variable side effects. Patient reported outcome measures and decision aids have the potential to improve the decision process by helping patients make high quality decisions. We discuss the importance of and barriers to incorporating these tools into clinical practice. MethodsWe reviewed the literature and summarized key articles and studies of patient reported outcomes and decision aids related to the management of localized prostate cancer. ResultsMultiple high quality patient reported outcome measures that are commonly used in research are beginning to make inroads into clinical practice. We recommend using EPIC (Expanded Prostate Index Composite)-Short Form to measure and track outcomes in patients with localized prostate cancer. Decision aids improve patient knowledge and satisfaction, and decrease patient uncertainty and indecisiveness. Barriers to implementing these tools in clinical practice can be overcome by engaging providers and staff, and establishing office protocols. ConclusionsIncorporating patient reported outcome measures and decision aids into clinical practice is likely to improve the quality of decision making in patients with prostate cancer but this requires thoughtful implementation.

Outcome Measures Other than Morbidity and Mortality for Patients with Incurable Cancer and Gastrointestinal Obstruction

To prospectively evaluate outcome measures of patients undergoing palliative surgical evaluation for gastrointestinal obstruction. Patients with an incurable malignancy undergoing consultation for gastrointestinal obstruction were prospectively enrolled from November 2009 to July 2012. We evaluated two patient-reported outcome measures of quality of life (Functional Assessment of Cancer Therapy-General [FACT-G]) and treatment satisfaction (Functional Assessment of Chronic Illness Therapy-Treatment Satisfaction-General Version 1 [FACIT-TS-G]) and five observational outcome measures (symptom improvement, 30 "good days," ability to tolerate diet at discharge, discharge home, and death within 90 days). Of 53 patients enrolled, 13 had gastric outlet obstruction, 22 had small bowel obstruction, and 18 had large bowel obstruction. Patient-reported measures could not be analyzed because only 19 patients (36%) completed the FACT-G and FACIT-TS-G survey at 1-month follow-up. However, we were able to obtain results for the 5 clinical observational outcomes in all patients. Symptom improvement was obtained in 41 (77%) patients, 30 "good days" in 40 (75%), ability to tolerate diet at discharge in 45 (85%), discharge to home in 46 (87%), and 18 (34%) of patients died within 90 days of evaluation. Large bowel obstruction was associated with symptom improvement, and noncolorectal cancer histology and carcinomatosis were negatively associated with having 30 "good days." The ability to tolerate oral intake at discharge was associated with Eastern Cooperative Oncology Group performance status and no recent chemotherapy administration. Death within 90 days was independently associated with noncolorectal cancer histology, ascites, and nonsurgical treatment. Observational outcome measures can provide follow-up data and the identification of variables associated with outcome for patients who are unable to respond to outpatient surveys.

Evaluation of Ankylosing Spondylitis Quality of Life (EASi-QoL): Reliability and Validity of a New Patient-reported Outcome Measure

There is currently no universally accepted measure of quality of life in ankylosing spondylitis (AS). Our objective was to develop and evaluate a patient-reported outcome measure of quality of life in AS, EASi-QoL. We used patient interviews, a literature review, and completion of an individualized measure of AS quality of life during clinic-based and pilot surveys to derive questionnaire content. Classical and modern psychometrics were then used to evaluate the questionnaire using data from a large UK-based postal survey of 1000 patients with AS. Data analysis from the interviews and clinic-based and postal surveys produced a 57-item self-completed questionnaire. Fifteen items were removed as a result of patient interviews and the pilot survey. In total, 612 (64.0%) patients responded to the main postal survey. After assessment of data quality, confirmatory factor analysis, and Rasch analysis, 20 items were found to contribute to 4 domains of AS-related quality of life: physical function, disease activity, emotional well-being, and social participation. Item-total correlations ranged from 0.66 to 0.84. Cronbach's alpha and test-retest reliability estimates were 0.88-0.92 and 0.88-0.93, respectively. Confirmed hypothesized correlations with the AS Quality of Life questionnaire, the Bath AS Disease Activity Index, Bath AS Functional Index, SF-36, EQ-5D, and the Hospital Anxiety and Depression Scale were evidence for the construct validity of the EASi-QoL. The EASi-QoL has good evidence of data quality, internal reliability, test-retest reliability, and content and construct validity, and should be considered for use with patients in routine practice settings and in evaluative studies including clinical trials. Measurement responsiveness and minimal important change are currently being assessed.

Quality of life assessment in renal transplant: review and future directions

We performed a systematic search of the literature using the MEDLINE (through 2006) database to identify the patient-reported outcome measures of quality of life (QOL) used most often in the renal transplant literature. After applying limits and reviewing references, we identified 338 renal transplant articles with a formal QOL assessment. Among the most frequently cited instruments were generic QOL tools, such as the Short Form-36, the Sickness Impact Profile, and the World Health Organization Quality of Life questionnaire. Other frequently cited instruments were more targeted in nature, such as the End-stage Renal Disease-Symptom Checklist and the Kidney Disease Quality of life questionnaire. In the present article, we review the most commonly used instruments, their use in renal transplant, and suggest future directions to improve QOL assessment in this population.