Patient-reported Outcomes Extension Research Articles

PCR218 Evaluating the Adherence of Reporting Patient Reported Outcomes to the Consort PRO Extension, CONSORT-Outcomes Extension and ISOQOL Standards Among Aggressive Large B Cell Lymphoma Patients: Systematic Literature Review of RCTs

To evaluate the adherence of reporting Patient Reported Outcomes (PROs) to the 2013 CONSORT PRO extension, CONSORT-Outcomes 2022 extension and ISOQOL standards in Randomized Control Trials (RCTs) among Aggressive Large B Cell Lymphoma (LBCL) patients.

Evaluating The Reporting of Patient-Reported Outcomes in Surgical Management of Stress Urinary Incontinence in Women: An Analysis of Randomized Controlled Trials.

Stress urinary incontinence (SUI) significantly reduces women's quality of life (QoL). Use of patient-reported outcomes (PROs) is increasing in randomized controlled trials (RCTs), thus standardization is important to ensure reporting completeness. We aim to evaluate completeness of reporting of RCTs for surgical management of SUI in women based on an adaptation of the Consolidated Standards of Reporting Trials statement with PRO extension (CONSORT-PRO). A literature search was conducted and all RCTs meeting inclusion criteria were evaluated using the CONSORT-PRO adapted checklist and the Cochrane Collaboration risk of bias assessment tool (RoB). We calculated a completion percentage score for each trial's adherence to the CONSORT-PRO adapted checklist and used bivariate regression analysis to examine associations between trial characteristics and completion percentage scores. Forty-three RCTs underwent data extraction and analysis. Mean completion percentage of the CONSORT-PRO was 50.53% (SD=15.63). A total of 38 (of 43; 88.37%) RCTs received an RoB 2.0 rating of "some concern." RCTs with follow-up longer than 3months had statistically significantly higher CONSORT-PRO completion: 3-6months (p=.049), 6-12months (p=.009), more than 12months (p=.021). Compared with studies without a conflict of interest statement, studies reporting a conflict of interest (p<.001) or reporting no conflict of interest (p=.048) had higher reporting completeness. Our results suggest many RCTs addressing surgical management of SUI in women have poor adherence to CONSORT-PRO reporting guidelines. Improving reporting completeness through adherence to the CONSORT-PRO checklist can better inform clinical decision making and improve QoL.

Use and Reporting of Patient-Reported Outcomes in Trials of Palliative Radiotherapy

Approximately 50% of all patients with cancer have an indication for radiotherapy, and approximately 50% of radiotherapy is delivered with palliative intent, with the aim of alleviating symptoms. Symptoms are best assessed by patient-reported outcomes (PROs), yet their reliable interpretation requires adequate reporting in publications. To investigate the use and reporting of PROs in clinical trials of palliative radiotherapy. This preregistered systematic review searched PubMed/Medline, EMBASE, and the Cochrane Center Register of Controlled Trials for clinical trials of palliative radiotherapy published from 1990 to 2020. Key eligibility criteria were palliative setting, palliative radiotherapy as treatment modality, and clinical trial design (per National Institutes of Health definition). Two authors independently assessed eligibility. Trial characteristics were extracted and standard of PRO reporting was assessed in adherence to the Consolidated Standards of Reporting Trials (CONSORT) PRO extension. The association of the year of publication with the use of PROs was assessed by logistic regression. Factors associated with higher CONSORT-PRO adherence were analyzed by multiple regression. This study is reported following the PRISMA guidelines. Among 7377 records screened, 225 published clinical trials representing 24 281 patients were eligible. Of these, 45 trials (20%) used a PRO as a primary end point and 71 trials (31%) used a PRO as a secondary end point. The most prevalent PRO measures were the Numeric Rating Scale/Visual Analogue Scale (38 trials), European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire C30 (32 trials), and trial-specific unvalidated measures (25 trials). A more recent year of publication was significantly associated with a higher chance of PROs as a secondary end point (odds ratio [OR], 1.04 [95% CI, 1.00-1.07]; P = .03) but not as primary end point. Adherence to CONSORT-PRO was poor or moderate for most items. Mean (SD) adherence to the extension adherence score was 46.2% (19.6%) for trials with PROs as primary end point and 31.8% (19.8%) for trials with PROs as a secondary end point. PROs as a primary end point (regression coefficient, 9.755 [95% CI, 2.270-17.240]; P = .01), brachytherapy as radiotherapy modality (regression coefficient, 16.795 [95% CI, 5.840-27.751]; P = .003), and larger sample size (regression coefficient, 0.028 [95% CI, 0.006-0.049]; P = .01) were significantly associated with better PRO reporting per extension adherence score. In this systematic review of palliative radiotherapy trials, the use and reporting of PROs had room for improvement for future trials, preferably with PROs as a primary end point.

Patient-reported outcomes (PROs) in randomised controlled trials in diabetes and pregnancy: protocol for a systematic review

IntroductionDiabetes mellitus is the most common metabolic complication of pregnancy and its prevalence worldwide is rising. The number of randomised controlled trials (RCTs) being conducted in people with diabetes is...

Temporal trends in the inclusion of patient-reported outcomes in heart failure randomized trials published in high-impact medical journals: a systematic bibliometric review

Abstract Background Patient reported outcomes (PROs) are important measures of health, particularly in symptomatic conditions such as heart failure (HF), and regulatory agencies have encouraged their collection in randomized controlled trials (RCTs). Purpose To assess temporal trends in the inclusion of patient-reported outcomes (PROs) in heart failure (HF) randomized controlled trials (RCTs) published in high-impact medical journals, explore trial characteristics associated with inclusion of PROs, and describe the quality of PRO reporting in HF RCTs. Methods We searched MEDLINE, EMBASE and CINAHL for studies published between January 1, 2000 and July 17, 2020. We included RCTs published in journals with impact factor ≥10. We assessed temporal trends using the Jonckheere-Terpstra test and conducted multivariable logistic regression analysis to explore trial characteristics independently associated with inclusion of PROs. We assessed the quality of PRO reporting using the Consolidated Standards for Trial Reporting PRO extension statement. Results We identified 12,342 articles, of which 417 RCTs met inclusion criteria. PROs were included in 224 RCTs (53.7%, 95% confidence interval [CI]: 48.8%-58.6%), of which 44 (19.6%) reported PRO as primary or co-primary endpoint. The proportion of RCTs with PROs increased significantly between 2000–2003 (37.4%) and 2016–2020 (65.1%) (p&amp;lt;0.001). PROs had higher odds of inclusion in RCTs that were multicenter (odds ratio [OR]: 1.95; 95% CI: 1.05–3.64; p=0.036); medium-sized (n=51–250) (OR: 2.29; 95% CI: 1.24–4.23; p=0.008); coordinated in in Central and South America (OR: 6.79; 95% CI: 1.34–34.36; p=0.021); and assessed health services (OR: 4.21; 1.97–8.98; p&amp;lt;0.001), device / surgical (OR: 6.24; 95% CI 3.05–12.80; p&amp;lt;0.001), or exercise and rehabilitation interventions (OR: 3.98; 95% CI 1.59–9.97; p=0.003). A majority (54.9%) of the 224 RCTs reported four or less of the eleven CONSORT-PRO items. The median number of CONSORT-PRO items reported was 4 (interquartile range [IQR] 3–6 items per trial), with improved reporting in trials with PRO as primary or co-primary endpoint. Conclusions PROs are included in in just over half of HF RCTs, with an increased reporting of PROs between 2000 and 2020. Large, pharmacotherapy trials conducted in North America and Europe were less likely to include PROs. The quality of PRO reporting in HF RCTs was modest, with trials most often reporting four of eleven CONSORT-PRO items. Funding Acknowledgement Type of funding sources: Public grant(s) – National budget only. Main funding source(s): Canadian Institutes of Health Research

Quality of patient‐reported outcomes in oncology clinical trials using immune checkpoint inhibitors: A systematic review

BackgroundThere are limited data regarding the quality of patient‐reported outcome (PRO) data in immune checkpoint inhibitor (ICI) clinical trial publications.MethodsA systematic search of citations from various databases was conducted to identify prospective clinical trials involving ICI in advanced tumors from 2003 to 2020. A 30‐point score was adapted from the CONSORT PRO extension statement to assess adherence to CONSORT PRO reporting. Linear regression was used to identify factors associated with quality reporting.ResultsAfter the review of 8058 articles, 33 trials were included with ICIs as either monotherapy (91%) or part of a combination regimen (9%). The median score was 23.5 points (range 15–29). In the majority of cases (82%), PROs were reported in a separate publication from the original study. Most of the trials were conducted in the metastatic setting and predominantly in melanoma, lung, and renal cell carcinoma (RCC) (73%). Univariate analysis revealed that trials with greater than 250 patients were associated with a higher score. The score was more likely to be lower in disease sites other than melanoma, lung, and RCC and was higher in the KEYNOTE than in the CHECKMATE trial series. There was no significant correlation between the score and whether a trial met its primary end‐point or if the trial improved or worsened the quality of life. In the multivariate analysis, the number of patients enrolled to the trial, disease site, and trial series remained significant.ConclusionsThe quality of reporting of PROs in ICI phase II and III clinical trials is heterogeneous across various cancer sites. As PRO data are increasingly used to counsel patients and complement clinical decision making, innovative and collaborative efforts are required to improve the reporting of these essential data.

Quality of patient-reported outcome reporting according to the CONSORT statement in randomized controlled trials with glioblastoma patients.

Randomized controlled trials (RCTs) represent the best evidence in oncology research. Glioblastoma is the most frequent and deadly primary brain tumor, affecting health-related quality of life. An important end point is patient-reported outcomes (PROs). There are no data regarding how well publications of glioblastoma RCTs report PROs. A specific PRO extension of the Consolidated Standards of Reporting Trials (CONSORT) statement was created to improve the quality of reporting. The aim of this study was to evaluate adherence to the CONSORT-PRO statement in reporting RCTs addressing the treatment of patients with glioblastoma. PRO analysis methodology was explored and criteria associated with higher quality of reporting were investigated. From PubMed/MEDLINE and the Cochrane Library databases, all phase 2 and 3 RCTs related to glioblastoma published between 1995 and 2018 were reviewed according to the CONSORT-PRO statements. An overall quality score on a 0 to 100 scale was defined based on these criteria and factors associated with this score were identified. Forty-four RCTs were identified as relevant according to predefined criteria. The median overall quality score was 26. No difference was observed regarding reporting quality over the years. CONSORT-PRO items concerning data collection and analysis were poorly reported. Thirty-four trials (77%) used longitudinal data. The most frequent statistical method for PROs analysis was the mean change from baseline (63%). Factors associated with improved overall quality score were the presence of a secondary publication dedicated to PROs results, the statement of any targeted dimensions, and when trials reported results using multiple methods. Despite the importance of measuring PROs in patients with glioblastoma, employment of the CONSORT-PRO statement is poor in RCTs.

How are patient-reported outcomes and symptoms being measured in adults with relapsed/refractory multiple myeloma? A systematic review.

Patients with relapsed and/or refractory multiple myeloma (RRMM) are living longer due in part to changing treatment patterns. It is important to understand how changing treatment patterns affect patients' lives beyond extending survival. Research suggests that direct patient report is the best way to capture information on how patients feel and function in response to their disease and its treatment. Therefore, the purpose of this review is to summarize evidence of patients' experience collected through patient-reported outcomes (PRO) in RRMM patients, and to explore PRO reporting quality. We conducted a systematic search to identify manuscripts reporting PROs in RRMM and summarized available evidence. We assessed PRO reporting quality using the Consolidated Standards of Reporting Trials (CONSORT) PRO Extension checklist. Our search resulted in 30 manuscripts. Thirteen unique PRO measures were used to assess 18 distinct PRO domains. Pain, fatigue, and emotional function were commonly assessed domains though reporting formats limited our ability to understand prevalence and severity of PRO challenges in RRMM. Evaluation of PRO reporting quality revealed significant reporting deficiencies. Several reporting criteria were included in less than 25% of manuscripts. Existing evidence provides a limited window for understanding the patient experience of RRMM and is further limited by suboptimal reporting quality. Observational studies are needed to describe prevalence, severity and patterns of PROs in RRMM overtime. Future studies that incorporate PROs would benefit from following existing guidelines to ensure that study evidence and conclusions can be fully assessed by readers, clinicians and policy makers.

Does the quality of patient-reported outcomes (PROs) assessment in randomized controlled trials (RCTs) differ across cancer types and over time? A pooled analysis of 610 RCTs published between 2004 and 2018.

e18218 Background: PRO endpoints are increasingly being used in cancer RCTs. However, the PRO assessment in such trials often suffers from serious methodological shortcomings, and the results seldom impact on clinical policy or practice. Methods: We performed a systematic review to identify RCTs with a PRO endpoint in breast, colorectal, lung, prostate, gynaecological and bladder cancer. A checklist score for quality of PRO reporting (ranging between 0-100), based on that of the International Society for Quality of Life Research (ISOQOL) and the CONSORT PRO extension, was computed for each RCT. Analyses were also conducted by type of PRO endpoint (primary versus secondary) and year of publication (i.e. before and after the publication of the CONSORT PRO extension). Results: We identified 610 RCTs with a total of 323,482 patients. PROs were most frequently used in RCTs of breast (N = 176), followed by lung (N = 123), prostate (N = 108), colorectal (N = 103), gynaecological (N = 83) and bladder (N = 17) cancer. Quality of PRO reporting (mean score: 56.4) was highest in RCTs conducted in prostate cancer (PCa) (Table). Regardless of cancer type, quality of reporting was typically higher in RCTs where PROs were primary endpoints. Quality of reporting was higher for RCTs published after the CONSORT PRO Extension (2013), with the exception of RCTs conducted in PCa, where quality was stable over time. Conclusions: PRO reporting of RCTs conducted in PCa has better quality than in the other cancer sites that were reviewed. Regardless of cancer site, quality of PRO reporting has improved after the publication of the CONSORT PRO Extension. [Table: see text]

Patient-centred outcomes in lateral elbow tendinopathy: A systematic review of available evidence in UK populations

For Patient-Reported Outcome Measures to be useful, they must have evidenced reliability, validity and responsiveness in both the condition and population of interest. The aim of this study is to systematically review the evidence for Patient-Reported Outcome Measures in UK patients with lateral elbow tendinopathy. A systematic search was performed in Ovid MEDLINE, Embase and CINAHL. Studies were included if reporting administration of Patient-Reported Outcome Measures in UK populations with lateral elbow tendinopathy. Patient-Reported Outcome Measures characteristics and target populations were assessed using a structured classification system. Patient-Reported Outcome Measures reporting in randomised controlled trials was assessed against Consolidated Standards of Reporting Trials (CONSORT) standards (Patient-Reported Outcome extension). A total of 16 articles were included. Out of seven different Patient-Reported Outcome Measures, there was evidence of partial validation for five. The assessment of validity, reliability and responsiveness of all Patient-Reported Outcome Measures in lateral elbow tendinopathy UK populations extended to just 20 individual patients. No articles conformed to the CONSORT Patient-Reported Outcome extension standards. There exists a paucity of data on the psychometrics of Patient-Reported Outcome Measures in UK lateral elbow tendinopathy populations. Without these data, trial design and interpretation are significantly hindered. The high prevalence of this condition and significant volume of studies being conducted into novel treatments highlight the need for this knowledge gap to be resolved.

A systematic evaluation of compliance and reporting of patient-reported outcome endpoints in ovarian cancer randomised controlled trials: implications for generalisability and clinical practice

BackgroundThis study aimed to evaluate the patient-reported outcome (PRO) content of ovarian cancer randomised-controlled trial (RCT) publications, describe PRO compliance, and explore potential relationships among these and completeness of PRO protocol content.MethodsPublications of Phase III ovarian cancer RCTs with PRO endpoints were identified by Medline and Cochrane systematic search: January 2000 to February 2016. Two reviewers determined the number of Consolidated Standards of Reporting Trials (CONSORT)-PRO Extension items addressed in publications. Compliance rates (defined as the proportion of participants included in the principal PRO analysis, of those from whom PRO assessments were expected) were extracted. The relationship between CONSORT-PRO score and compliance rates was explored using scatter plots. Additionally CONSORT-PRO score and PRO compliance rates respectively were compared with corresponding PRO protocol scores obtained from a previous study.ResultsThirty-six eligible RCTs (n = 33 with secondary PRO endpoint) were identified and analysed. The average number of CONSORT-PRO items addressed in publications was 6.7 (48%; Range 0–13.5/14). Three RCTs did not report PRO results; in 1 case due to poor compliance. Some compliance information was reported in 26 RCTs, but was considered complete for only 10 (28%) RCTs. Compliance rates were poor overall, ranging from 59 to 83%; therefore missing PRO data from 17 to 41% of participants in these trials could have been avoided.Of the 26 (73%) RCTs for which PRO protocol completeness scores were available, 6 RCTs reported complete compliance information and the 3 of these RCTs with highest PRO compliance had highest protocol checklist scores.ConclusionsFew RCTs reported PRO compliance information in a manner enabling assessment of the generalisability of PRO results. This information is particularly important in RCTs of advanced ovarian cancer because it is important to be able to determine if missing data was due to worsening illness compared to methodological issues. Poor compliance appeared related to poor PRO protocol content, and in one case prevented PRO results from being reported, highlighting the need to address compliance strategies in the protocol. Adhering to protocol and CONSORT-PRO reporting guidance should improve PRO implementation and reporting respectively in ovarian cancer RCTs and allow results to meaningfully inform clinical practice.

Systematic review reveals lack of quality in reporting health-related quality of life in patients with gastroenteropancreatic neuroendocrine tumours.

BackgroundGastroenteropancreatic neuroendocrine tumours (GEP-NET) are often slow-growing and patients may live for years with metastasised disease. Hence, along with increasing overall and progression-free survival, treatments aim at preserving patients’ well-being and health-related quality of life (HRQoL). However, studies on systematic HRQoL assessment in patients with GEP-NET are scarce. Therefore, the purpose of the current review is to systematically evaluate the methodological quality of the identified studies.MethodsA targeted database search was performed in PubMed, EMBASE, and CENTRAL. Data extraction was conducted by two independent researchers according to predefined criteria. For study evaluation, the Minimum Standard Checklist for Evaluating HRQoL Outcomes in Cancer Clinical Trials and the CONSORT Patient-Reported Outcome extension were adapted.ResultsThe database search yielded 48 eligible studies. We found the awareness for the need of HRQoL measurement to be growing and application of cancer-specific instruments gaining acceptance. Overall, studies were too heterogeneous in terms of patient characteristics and treatment interventions to draw clear conclusions for clinical practice. More importantly, a range of methodological shortcomings has been identified which were mainly related to the assessment and statistical analysis, as well as the reporting and interpretation of HRQoL data.ConclusionDespite an increasing interest in HRQoL in GEP-NET patients, there is still a lack of knowledge on this issue. A transfer of HRQoL results into clinical practice is hindered not only by the scarceness of studies, but also by the often limited quality of HRQoL processing and reporting.Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-016-0527-2) contains supplementary material, which is available to authorized users.

Quality of patient-reported outcome reporting across cancer randomized controlled trials according to the CONSORT patient-reported outcome extension: A pooled analysis of 557 trials.

The main objectives of this study were to identify the number of randomized controlled trials (RCTs) including a patient-reported outcome (PRO) endpoint across a wide range of cancer specialties and to evaluate the completeness of PRO reporting according to the Consolidated Standards of Reporting Trials (CONSORT) PRO extension. RCTs with a PRO endpoint that had been performed across several cancer specialties and published between 2004 and 2013 were considered. Studies were evaluated on the basis of previously defined criteria, including the CONSORT PRO extension and the Cochrane Collaboration's tool for assessing the risk of bias of RCTs. Analyses were also conducted by the type of PRO endpoint (primary vs secondary) and by the cancer disease site. A total of 56,696 potentially eligible records were scrutinized, and 557 RCTs with a PRO evaluation, enrolling 254,677 patients overall, were identified. PROs were most frequently used in RCTs of breast (n = 123), lung (n = 85), and colorectal cancer (n = 66). Overall, PROs were secondary endpoints in 421 RCTs (76%). Four of 6 evaluated CONSORT PRO items were documented in less than 50% of the RCTs. The level of reporting was higher in RCTs with a PRO as a primary endpoint. The presence of a supplementary report was the only statistically significant factor associated with greater completeness of reporting for both RCTs with PROs as primary endpoints (β = .19, P = .001) and RCTs with PROs as secondary endpoints (β = .30, P < .001). Implementation of the CONSORT PRO extension is equally important across all cancer specialties. Its use can also contribute to revealing the robust PRO design of some studies, which might be obscured by poor outcome reporting.

Patient-reported outcomes in ovarian cancer clinical trials

There is general acceptance of the importance of incorporating patient-reported outcome (PRO) measures including health-related quality of life (HRQOL) into clinical trials, and there are now a number of guidance documents available on how to use PRO's for regulatory authorities and in comparative effectiveness research. The methods used to collect, analyse and report PRO data in clinical trials have received considerable scrutiny, revealing many shortcomings in the standard of reporting of HRQOL in clinical trials as well as in how PRO's have been selected and analysed in clinical trials. This has led to the recent Consolidated Standards of Reporting Clinical Trials—PRO extension statement which lays down a framework for selection and reporting analysis of PROs, either as primary or secondary trial end points, thus ensuring scientific rigour. Adherence to these guidelines can only improve the conduct of clinical trials and interpretation of their results, which may help avoid missing out on opportunities as in the past. We review pertinent literature on PRO measures and discuss how various recent PRO guidance documents should be applied to ovarian cancer clinical trials.

The CONSORT Patient-Reported Outcome (PRO) extension: implications for clinical trials and practice.

To inform clinical guidelines and patient care we need high quality evidence on the relative benefits and harms of intervention. Patient reported outcome (PRO) data from clinical trials can “empower patients to make decisions based on their values” and “level the playing field between physician and patient”. While clinicians have a good understanding of the concept of health-related quality of life and other PROs, evidence suggests that many do not feel comfortable in using the data from trials to inform discussions with patients and clinical practice. This may in part reflect concerns over the integrity of the data and difficulties in interpreting the results arising from poor reporting.The new CONSORT PRO extension aims to improve the reporting of PROs in trials to facilitate the use of results to inform clinical practice and health policy. While the CONSORT PRO extension is an important first step in the process, we need broader engagement with the guidance to facilitate optimal reporting and maximize use of PRO data in a clinical setting. Endorsement by journal editors, authors and peer reviewers are crucial steps. Improved design, implementation and transparent reporting of PROs in clinical trials are necessary to provide high quality evidence to inform evidence synthesis and clinical practice guidelines.

Reporting of Patient-Reported Outcomes in Randomized Trials

The CONSORT (Consolidated Standards of Reporting Trials) Statement aims to improve the reporting of randomized controlled trials (RCTs); however, it lacks guidance on the reporting of patient-reported outcomes (PROs), which are often inadequately reported in trials, thus limiting the value of these data. In this article, we describe the development of the CONSORT PRO extension based on the methodological framework for guideline development proposed by the Enhancing the Quality and Transparency of Health Research (EQUATOR) Network. Five CONSORT PRO checklist items are recommended for RCTs in which PROs are primary or important secondary end points. These recommendations urge that the PROs be identified as a primary or secondary outcome in the abstract, that a description of the hypothesis of the PROs and relevant domains be provided (ie, if a multidimensional PRO tool has been used), that evidence of the PRO instrument's validity and reliability be provided or cited, that the statistical approaches for dealing with missing data be explicitly stated, and that PRO-specific limitations of study findings and generalizability of results to other populations and clinical practice be discussed. Examples and an updated CONSORT flow diagram with PRO items are provided. It is recommended that the CONSORT PRO guidance supplement the standard CONSORT guidelines for reporting RCTs with PROs as primary or secondary outcomes. Improved reporting of PRO data should facilitate robust interpretation of the results from RCTs and inform patient care.