scholarly journals Reporting of Patient-Reported Outcomes in Randomized Trials: The CONSORT PRO Extension

2013 ◽  
Vol 16 (4) ◽  
pp. 455-456 ◽  
Author(s):  
Donald Patrick
2020 ◽  
Author(s):  
Karine E Manera ◽  
David W Johnson ◽  
Yeoungjee Cho ◽  
Benedicte Sautenet ◽  
Jenny Shen ◽  
...  

Abstract Background Randomized trials can provide evidence to inform decision-making but this may be limited if the outcomes of importance to patients and clinicians are omitted or reported inconsistently. We aimed to assess the scope and heterogeneity of outcomes reported in trials in peritoneal dialysis (PD). Methods We searched the Cochrane Kidney and Transplant Specialized Register for randomized trials in PD. We extracted all reported outcome domains and measurements and analyzed their frequency and characteristics. Results From 128 reports of 120 included trials, 80 different outcome domains were reported. Overall, 39 (49%) domains were surrogate, 23 (29%) patient-reported and 18 (22%) clinical. The five most commonly reported domains were PD-related infection [59 (49%) trials], dialysis solute clearance [51 (42%)], kidney function [45 (38%)], protein metabolism [44 (37%)] and inflammatory markers/oxidative stress [42 (35%)]. Quality of life was reported infrequently (4% of trials). Only 14 (12%) trials included a patient-reported outcome as a primary outcome. The median number of outcome measures (defined as a different measurement, aggregation and metric) was 22 (interquartile range 13–37) per trial. PD-related infection was the most frequently reported clinical outcome as well as the most frequently stated primary outcome. A total of 383 different measures for infection were used, with 66 used more than once. Conclusions Trials in PD include important clinical outcomes such as infection, but these are measured and reported inconsistently. Patient-reported outcomes are infrequently reported and nearly half of the domains were surrogate. Standardized outcomes for PD trials are required to improve efficiency and relevance.


JAMA ◽  
2013 ◽  
Vol 309 (8) ◽  
pp. 814 ◽  
Author(s):  
Melanie Calvert ◽  
Jane Blazeby ◽  
Douglas G. Altman ◽  
Dennis A. Revicki ◽  
David Moher ◽  
...  

2019 ◽  
Vol 111 (11) ◽  
pp. 1170-1178 ◽  
Author(s):  
Derek Kyte ◽  
Ameeta Retzer ◽  
Khaled Ahmed ◽  
Thomas Keeley ◽  
Jo Armes ◽  
...  

Abstract Background Patient-reported outcomes (PROs) are captured within cancer trials to help future patients and their clinicians make more informed treatment decisions. However, variability in standards of PRO trial design and reporting threaten the validity of these endpoints for application in clinical practice. Methods We systematically investigated a cohort of randomized controlled cancer trials that included a primary or secondary PRO. For each trial, an evaluation of protocol and reporting quality was undertaken using standard checklists. General patterns of reporting where also explored. Results Protocols (101 sourced, 44.3%) included a mean (SD) of 10 (4) of 33 (range = 2–19) PRO protocol checklist items. Recommended items frequently omitted included the rationale and objectives underpinning PRO collection and approaches to minimize/address missing PRO data. Of 160 trials with published results, 61 (38.1%, 95% confidence interval = 30.6% to 45.7%) failed to include their PRO findings in any publication (mean 6.43-year follow-up); these trials included 49 568 participants. Although two-thirds of included trials published PRO findings, reporting standards were often inadequate according to international guidelines (mean [SD] inclusion of 3 [3] of 14 [range = 0–11]) CONSORT PRO Extension checklist items). More than one-half of trials publishing PRO results in a secondary publication (12 of 22, 54.5%) took 4 or more years to do so following trial closure, with eight (36.4%) taking 5–8 years and one trial publishing after 14 years. Conclusions PRO protocol content is frequently inadequate, and nonreporting of PRO findings is widespread, meaning patient-important information may not be available to benefit patients, clinicians, and regulators. Even where PRO data are published, there is often considerable delay and reporting quality is suboptimal. This study presents key recommendations to enhance the likelihood of successful delivery of PROs in the future.


2018 ◽  
Vol 28 (5) ◽  
pp. 1439-1456 ◽  
Author(s):  
Daniel O Scharfstein ◽  
Aidan McDermott

Randomized trials with patient-reported outcomes are commonly plagued by missing data. The analysis of such trials relies on untestable assumptions about the missing data mechanism. To address this issue, it has been recommended that the sensitivity of the trial results to assumptions should be a mandatory reporting requirement. In this paper, we discuss a recently developed methodology (Scharfstein et al., Biometrics, 2018) for conducting sensitivity analysis of randomized trials in which outcomes are scheduled to be measured at fixed points in time after randomization and some subjects prematurely withdraw from study participation. The methodology is explicated in the context of a placebo-controlled randomized trial designed to evaluate a treatment for bipolar disorder. We present a comprehensive data analysis and a simulation study to evaluate the performance of the method. A software package entitled SAMON (R and SAS versions) that implements our methods is available at www.missingdatamatters.org .


2021 ◽  
pp. 135910532110593
Author(s):  
Michiel Tack

MetaBLIND is the largest meta-epidemiological study on the impact of blinding in randomized trials to date. We examined MetaBLIND data on the impact of blinding patients on patient-reported outcomes. 68 out of 132 included trials tested knowledge recall and had questionable relevance to clinical trials. In 17 out of 18 comparisons, the number of trials in the blinded or nonblinded group was 2 or lower. In several key trials, the blinding status was uncertain. Effect sizes compared in MetaBLIND appear to reflect random differences in study design and setting rather than the impact of blinding trial participants.


2015 ◽  
Vol 33 (8) ◽  
pp. 910-915 ◽  
Author(s):  
Massimo Di Maio ◽  
Ciro Gallo ◽  
Natasha B. Leighl ◽  
Maria Carmela Piccirillo ◽  
Gennaro Daniele ◽  
...  

Purpose Information about symptomatic toxicities of anticancer treatments is not based on direct report by patients, but rather on reports by clinicians in trials. Given the potential for under-reporting, our aim was to compare reporting by patients and physicians of six toxicities (anorexia, nausea, vomiting, constipation, diarrhea, and hair loss) within three randomized trials. Patients and Methods In one trial, elderly patients with breast cancer received adjuvant chemotherapy; in two trials, patients with advanced non–small-cell lung cancer received first-line treatment. Toxicity was prospectively collected by investigators (graded by National Cancer Institute Common Toxicity Criteria [version 2.0] or Common Terminology Criteria for Adverse Events [version 3]). At the end of each cycle, patients completed the European Organisation for Research and Treatment of Cancer quality-of-life questionnaires, including toxicity-related symptom items. Possible answers were “not at all,” “a little,” “quite a bit,” and “very much.” Analysis was limited to the first three cycles. For each toxicity, agreement between patients and physicians and under-reporting by physicians (ie, toxicity reported by patients but not reported by physicians) were calculated. Results Overall, 1,090 patients (2,482 cycles) were included. Agreement between patients and physicians was low for all toxicities. Toxicity rates reported by physicians were always lower than those reported by patients. For patients who reported toxicity (any severity), under-reporting by physicians ranged from 40.7% to 74.4%. Examining only patients who reported “very much” toxicity, under-reporting by physicians ranged from 13.0% to 50.0%. Conclusion Subjective toxicities are at high risk of under-reporting by physicians, even when prospectively collected within randomized trials. This strongly supports the incorporation of patient-reported outcomes into toxicity reporting in clinical trials.


2021 ◽  
Author(s):  
David T. Liss ◽  
Toshiko Uchida ◽  
Cheryl L. Wilkes ◽  
Ankitha Radakrishnan ◽  
Jeffrey A. Linder

ABSTRACTImportanceGeneral health checks—also known as general medical exams, periodic health evaluations, checkups, or wellness visits—to identify and prevent disease are extremely common in adult primary care. Although general health checks are often expected and advocated by patients, clinicians, payers, and health systems, others question their value. The current evidence was updated and recommendations provided for conducting general health checks in adults.ObservationsRandomized trials and observational studies with control groups from prior systematic reviews and an updated literature review through December 2020 were included. Out of 19 included randomized trials (906 to 59,616 participants; follow-up, 1 to 30 years), 5 evaluated a single general health check and 7 evaluated annual health checks. All of 12 included observational studies (240 to 471,415 participants; follow-up, cross-sectional to 5 years) evaluated a single general health check. General health checks were generally not associated with decreased mortality, cardiovascular events, or cardiovascular disease incidence. For example, in the South-East London Screening Study (n=7,229), adults age 40 to 64 who were invited to two health checks over two years experienced no 8-year mortality benefit (6% overall). However, general health checks were associated with increased detection of chronic diseases, such as depression and hypertension; moderate improvements in controlling risk factors such as blood pressure and cholesterol; increased clinical preventive service uptake, such as colorectal and cervical cancer screening; and improvements in patient-reported outcomes, such as quality of life and self-rated health. General health checks were sometimes associated with modest improvements in health behaviors such as physical activity and diet. For example, in the OXCHECK trial (n=4121), fewer intervention participants exercised less than once per month (68% versus 71%). Potential adverse effects in individual studies included an increased risk of stroke and increased mortality attributed to increased completion of advanced directives.Conclusions and RelevanceGeneral health checks were not associated with reduced mortality or cardiovascular events, but were associated with increased chronic disease recognition and treatment; risk factor control, preventive service uptake, and patient-reported outcomes. Primary care teams may reasonably offer general health checks, especially for groups at high risk of overdue preventive services, uncontrolled risk factors, low self-rated health, or poor connection to primary care.


Author(s):  
Christine de la Loge ◽  
Béatrice Taugaut ◽  
Fatoumata Fofana ◽  
Jérémy Lambert ◽  
Michael Hennig ◽  
...  

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