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This review of reviews aimed to appraise the use of the CONSORT-PRO Extension as an evaluation tool for assessing the reporting of patient-reported outcome (PROs) in publications, and to describe the reporting of PRO research across reviews. We also outlined how variation in such evaluations impacts knowledge translation and may lead to potential misuse of the CONSORT-PRO Extension. We systematically searched Medline, Pubmed and CINAHL from 2013 to 2025 March 2021 for reviews of the completeness of reporting of PRO endpoints according to CONSORT-PRO criteria. Two reviewers extracted details of each review, the percentage of included studies that addressed each CONSORT-PRO item, and key recommendations from each review. Fourteen reviews met inclusion criteria, and only six of these used the full CONSORT-PRO checklist with minimal justified modifications. The remaining eight studies made significant or unjustified adjustments to the CONSORT-PRO Extension. Review studies also varied in how they scored multi-component CONSORT-PRO items. CONSORT-PRO items were often unreported in trial reports, and certain CONSORT-PRO items were reported less often than others. The reporting of statistical approaches to dealing with missing PRO data were poor in RCTs included in all 14 review articles. Studies reviewing PRO publications often omitted recommended CONSORT-PRO items from their evaluations, which may cause confusion among readers regarding how best to report their PRO research according to the CONSORT-PRO extension. Many trials published since CONSORT-PRO's release did not report recommended CONSORT-PRO items, which may lead to misinterpretation and consequently to research waste., (© 2022. The Author(s).)

Purpose: Missing patient-reported outcome (PRO) data can seriously threaten the validity of randomized clinical trials (RCTs). Identifying which factors predict missing instruments may help researchers develop strategies to prevent it from happening. This study examined the association of factors with time to the first missing instrument after randomization in three cooperative group RCTs., Methods: We performed descriptive analyses and Cox proportional hazards regressions for three RCTs selected from the Canadian Cancer Trials Group: MA17 (breast cancer), PR7 (prostate cancer), and LY12 (non-Hodgkin's lymphoma). The outcome was the time from randomization to the first missing instrument. Variables for 15 factors were used as covariates based on availability and previously-reported putative associations with missing PRO data., Results: Nine percent of 1352 subjects on MA17, 37% of 923 subjects on PR7, and 59% of 477 subjects on LY12 had a missing instrument. Twenty-five percent of subjects on MA17 had first missing instrument within 4.6 years. The median time to first missing instrument was: not observed for MA17, 7.3 years for PR7, 0.12 years for LY12. Cox regression revealed statistically significant independent associations with outcome for only five factors: baseline age (PR7) and level of well-being (LY12), and centre level of activity (LY12), presence of post-graduate residency training program (MA17, PR7), and centre geographic location (PR7, LY12)., Conclusion: Many factors reported to have association with missing instruments do not seem to predict time to the first missing instrument after randomization in RCTs. Context is important in understanding the few that may., (© 2021. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)

Purpose: Patient-reported outcomes (PROs) are increasingly used in clinical trials to provide patients' perspectives regarding symptoms, health-related quality of life, and satisfaction with treatments. A range of guidance documents exist for the selection of patient-reported outcome measures (PROMs) in clinical trials, and it is unclear to what extent these documents present consistent recommendations., Methods: We conducted a targeted review of publications and regulatory guidance documents that advise on the selection of PROMs for use in clinical trials. A total of seven guidance documents from the US Food and Drug Administration, European Medicines Agency, and scientific consortia from professional societies were included in the final review. Guidance documents were analyzed using a content analysis approach comparing them with minimum standards recommended by the International Society for Quality of Life Research., Results: Overall there was substantial agreement between guidance regarding the appropriate considerations for PROM selection within a clinical trial. Variations among the guidance primarily related to differences in their format and differences in the perspectives and mandates of their respective organizations. Whereas scientific consortia tended to produce checklist or rating-type guidance, regulatory groups tended to use more narrative-based approaches sometimes supplemented with lists of criteria., Conclusion: The consistency in recommendations suggests an emerging consensus in the field and supports use of any of the major guidance documents available to guide PROM selection for clinical trials without concern of conflicting recommendations. This work represents an important first step in the international PROTEUS Consortium's ongoing efforts to optimize the use of PROs in clinical trials.

Purpose: Patient-reported outcome (PRO) data from clinical trials can promote valuable patient-clinician communication and aid the decision-making process regarding treatment options. Despite these benefits, both patients and doctors face challenges in interpreting PRO scores. The purpose of this study was to identify best practices for presenting PRO results expressed as proportions of patients with changes from baseline (improved/stable/worsened) for use in patient educational materials and decision aids., Methods: We electronically surveyed adult cancer patients/survivors, oncology clinicians, and PRO researchers, and conducted one-on-one cognitive interviews with patients/survivors and clinicians. Participants saw clinical trial data comparing two treatments as proportions changed using three different formats: pie charts, bar graphs, icon arrays. Interpretation accuracy, clarity, and format preference were analyzed quantitatively and online survey comments and interviews, qualitatively., Results: The internet sample included 629 patients, 139 clinicians, and 249 researchers; 10 patients and 5 clinicians completed interviews. Bar graphs were less accurately interpreted than pie charts (OR 0.39; p < .0001) and icon arrays (OR 0.47; p < .0001). Bar graphs and icon arrays were less likely to be rated clear than pie charts (OR 0.37 and OR 0.18; both p < .0001). Qualitative data informed interpretation of these findings., Conclusions: For communicating PROs as proportions changed in patient educational materials and decision aids, these results support the use of pie charts.

Purpose: To introduce the EPIC-CP symptom screening tool in routine ambulatory cancer care, and to evaluate its acceptability and perceived usefulness from the perspective of patients and clinicians., Methods: Eligible prostate cancer patients from four cancer centres were recruited (November 2014-June 2015) from radiation or surgical oncology clinics. A physician and/or health care professional reviewed the EPIC-CP results as part of the clinical encounter. Patient experience with the tool was evaluated using a nine-item Patient Exit Survey (PES). Clinician experience was evaluated through semi-structured qualitative interviews. Patient and clinician results were compared to identify common themes., Results: A total of 333 patients were enrolled, of whom, 287 completed the PES. Most patients had one clinical encounter, although the number of EPIC-CP assessments ranged from 1 to 11 per patient, for a total of 937 EPIC-CP questionnaires completed. Item completion rates were high (91-100%), with items addressing sexual health among the lowest (91-92%). On the PES, most patients (70%) agreed with the item: "Completing this questionnaire helped me tell the clinicians about how I have been feeling". Thematic analysis from clinician interviews revealed that the EPIC-CP captures essential prostate-specific effects that facilitated person-centred communication and customization of interventions. Targeted clinical education and patient resources were seen as necessary for uptake., Conclusions: EPIC-CP was generally endorsed by clinicians and patients. The implementation of a disease-specific measure in place of a generic symptom screening tool has the potential to improve the quality of the clinical encounter and provide outcome measures for further health services research. Provincial implementation of this tool as a standard of care is recommended.

Purpose: Patient-reported outcomes (PROs) can promote patient-centered care in multiple ways: (1) using an individual patient's PRO data to inform his/her management, (2) providing PRO results from comparative research studies in patient educational materials/decision aids, and (3) reporting PRO results from comparative research studies in peer-reviewed publications. Patients and clinicians endorse the value of PRO data; however, variations in how PRO measures are scored and scaled, and in how the data are reported, make interpretation challenging and limit their use in clinical practice. We conducted a modified Delphi process to develop stakeholder-engaged, evidence-based recommendations for PRO data display for the three above applications to promote understanding and use., Methods: The Consensus Panel included cancer survivors/caregivers, oncologists, PRO researchers, and application-specific end-users (e.g., electronic health record vendors, decision aid developers, journal editors). We reviewed the data display issues and their evidence base during pre-meeting webinars. We then surveyed participants' initial perspectives, which informed discussions during an in-person meeting to develop consensus statements. These statements were ratified via a post-meeting survey., Results: Issues addressed by consensus statements relevant to both individual and research data applications were directionality (whether higher scores are better/worse) and conveying score meaning (e.g., none/mild/moderate/severe). Issues specific to individual patient data presentation included representation (bar charts vs. line graphs) and highlighting possibly concerning scores (absolute and change). Issues specific to research study results presentation included handling normed data, conveying statistically significant differences, illustrating clinically important differences, and displaying proportions improved/stable/worsened., Conclusions: The recommendations aim to optimize accurate and meaningful interpretation of PRO data.

Purpose: The EORTC QLQ-C30 and the Brief Pain Inventory (BPI) are validated tools for measuring quality of life (QOL) and the impact of pain in patients with advanced cancer. Interpretation of these instrument scores can be challenging and it is difficult to know what numerical changes translate to clinically significant impact in patients' lives. To address this issue, our study sought to establish the minimal clinically important differences (MCID) for these two instruments in a prospective cohort of patients with advanced cancer and painful bone metastases., Methods: Both anchor-based and distribution-based methods were used to estimate the MCID scores from patients enrolled in a randomized phase III trial evaluating two different re-irradiation treatment schedules. For the anchor-based method, the global QOL item from the QLQ-C30 was chosen as the anchor. Spearman correlation coefficients were calculated for all items and only those items with moderate or better correlation (|r| ≥ 0.30) with the anchor were used for subsequent analysis. A 10-point difference in the global QOL score was used to classify improvement and deterioration, and the MCID scores were calculated for each of these categories. These results were compared with scores obtained by the distribution-method, which estimates the MCID purely from the statistical characteristics of the sample population., Results: A total of 375 patients were included in this study with documented pain responses and completed QOL questionnaires at 2 months. 9/14 items in the QLQ-C30 and 6/10 items in the BPI were found to have moderate or better correlation with the anchor. For deterioration, statistically significant MCID scores were found in all items of the QLQ-C30 and BPI. For improvement, statistically significant MCID scores were found in 7/9 items of the QLQ-C30 and 2/6 items of the BPI. The MCID scores for deterioration were uniformly higher than the MCIDs for improvement. Using the distribution-based method, there was good agreement between the 0.5 standard deviation (SD) values and anchor-based scores for deterioration. For improvement, there was less agreement and the anchor-based scores were lower than the 0.5 SD values obtained from the distribution-based method., Conclusion: We present MCID scores for the QLQ-C30 and BPI instruments obtained from a large cohort of patients with advanced cancer undergoing re-irradiation for painful bone metastases. The results from this study were compared to other similar studies which showed larger MCID scores for improvement compared to deterioration. We hypothesize that disease trajectory and patient expectations are important factors in understanding the contrasting results. The results of this study can guide clinicians and researchers in the interpretation of these instruments.

Purpose: Patient-reported outcome (PRO) results from clinical trials can inform clinical care, but PRO interpretation is challenging. We evaluated the interpretation accuracy and perceived clarity of various strategies for displaying clinical trial PRO findings., Methods: We conducted an e-survey of oncology clinicians and PRO researchers (supplemented by one-on-one clinician interviews) that randomized respondents to view one of the three line-graph formats (average scores over time for two treatments on four domains): (1) higher scores consistently indicating "better" patient status; (2) higher scores indicating "more" of what was being measured (better for function, worse for symptoms); or (3) normed scores. Two formats displayed proportions changed (pie/bar charts). Multivariate modeling was used to analyze interpretation accuracy and clarity ratings., Results: Two hundred and thirty-three clinicians and 248 researchers responded; ten clinicians were interviewed. Line graphs with "better" directionality were more likely to be interpreted accurately than "normed" line graphs (OR 1.55; 95% CI 1.01-2.38; p = 0.04). No significant differences were found between "better" and "more" formats. "Better" formatted graphs were also more likely to be rated "very clear" versus "normed" formatted graphs (OR 1.91; 95% CI 1.44-2.54; p < 0.001). For proportions changed, respondents were less likely to make an interpretation error with pie versus bar charts (OR 0.35; 95% CI 0.2-0.6; p < 0.001); clarity ratings did not differ between formats. Qualitative findings informed the interpretation of the survey findings., Conclusions: Graphic formats for presenting PRO data differ in how accurately they are interpreted and how clear they are perceived to be. These findings will inform the development of best practices for optimally reporting PRO findings.

Purpose: This study assessed the uptake of the CONsolidated Standards of Reporting Trials (CONSORT)-Patient-Reported Outcomes (PRO) statement; determined if use of CONSORT-PRO was associated with more complete reporting of PRO endpoints in randomised controlled trials (RCTs) and identified the extent to which high-impact journals publishing RCTs with PRO endpoints endorse CONSORT-PRO., Methods: CONSORT-PRO citations were identified by systematically searching Medline, EMBASE and Google from 2013 (year CONSORT-PRO released) to 17 December 2015. RCTs that cited CONSORT-PRO (cases) were compared to a comparable control sample of RCTs in terms of adherence to CONSORT-PRO using t tests. General linear models assessed the relationship between CONSORT-PRO score and key, pre-specified variables. The 100 highest-impact journals that published RCTs with PRO endpoints (2014-2015) were identified via a systematic Medline search. Instructions for authors were reviewed to determine whether journals endorsed CONSORT-PRO., Results: Total CONSORT-PRO scores ranged from 47 to 100% for cases and 25-96% for controls. Cases had significantly higher total CONSORT-PRO scores compared to controls: t = 2.64, p = 0.01. 'Citing CONSORT-PRO', 'journal endorsing CONSORT-PRO' and 'dedicated PRO paper' were significant predictors of higher CONSORT-PRO adherence score: R 2  = 0.48, p < 0.001. 11/100 top-ranked journals endorsed CONSORT-PRO in their instructions to authors, seven of these journals published RCTs included as cases in this study., Conclusion: This study demonstrated improved PRO reporting associated with journal endorsement and author use of the CONSORT-PRO extension. Despite growing awareness, more work is needed to promote appropriate use of CONSORT-PRO to improve completeness of reporting; in particular, stronger journal endorsement of CONSORT-PRO.

Purpose: Validated tools for evaluating quality of life (QOL) in patients with bone metastases include the EORTC QLQ-BM22 and QLQ-C15-PAL modules. A statistically significant difference in metric scores may not be clinically significant. To aid in their interpretation, we performed analyses to determine the minimal clinically important differences (MCID) for these QOL instruments., Methods: Both anchor-based and distribution-based methods were used to determine the MCID among patients with bone metastases enrolled in a randomized phase III trial. For the anchor-based approach, overall QOL as measured by the QLQ-C15-PAL module was used as the anchor and only the subscales with moderate or better correlation were used for subsequent MCID analysis. In the anchor-based approach, patients were classified as improved, stable or deteriorated by the change in the overall QOL score from baseline to follow-up after 42 days. The MCID and confidence interval was then calculated for all subscales. In the distribution-based approach, the MCID was expressed as a proportion of the standard deviation and standard error measurement from the subscale score distribution., Results: A total of 204 patients completed the questionnaires at baseline and follow-up. Only the dyspnea and insomnia subscales did not have at least moderate correlation with the overall QOL anchor. Using the anchor-based approach, 10/11 subscales had an MCID score significantly different than 0 for improvement and 3/11 subscales had a significant MCID score for deterioration. The magnitude of MCID scores was higher for improvement in comparison with deterioration. For improvement, the anchor-based approach showed good agreement with the distribution-based approach when using 0.5 SD as the MCID. However, there was greater lack of agreement between these approaches for deterioration., Conclusion: We present the MCID scores for the EORTC QLQ-BM22 and QLQ-C15-PAL QOL instruments. The results of this study can guide clinicians in the interpretation of these instruments., Clinical Trials Registry: NCT01248585.

Purpose: An essential aspect of patient-centered outcomes research (PCOR) and comparative effectiveness research (CER) is the integration of patient perspectives and experiences with clinical data to evaluate interventions. Thus, PCOR and CER require capturing patient-reported outcome (PRO) data appropriately to inform research, healthcare delivery, and policy. This initiative's goal was to identify minimum standards for the design and selection of a PRO measure for use in PCOR and CER., Methods: We performed a literature review to find existing guidelines for the selection of PRO measures. We also conducted an online survey of the International Society for Quality of Life Research (ISOQOL) membership to solicit input on PRO standards. A standard was designated as "recommended" when >50 % respondents endorsed it as "required as a minimum standard.", Results: The literature review identified 387 articles. Survey response rate was 120 of 506 ISOQOL members. The respondents had an average of 15 years experience in PRO research, and 89 % felt competent or very competent providing feedback. Final recommendations for PRO measure standards included: documentation of the conceptual and measurement model; evidence for reliability, validity (content validity, construct validity, responsiveness); interpretability of scores; quality translation, and acceptable patient and investigator burden., Conclusion: The development of these minimum measurement standards is intended to promote the appropriate use of PRO measures to inform PCOR and CER, which in turn can improve the effectiveness and efficiency of healthcare delivery. A next step is to expand these minimum standards to identify best practices for selecting decision-relevant PRO measures.

Purpose: To develop expert consensus on a suite of reporting standards for HRQL outcomes of RCTs., Methods: A Task Force of The International Society of Quality of Life Research (ISOQOL) undertook a systematic review of the literature to identify candidate reporting standards for HRQL in RCTs. Subsequently, a web-based survey was circulated to the ISOQOL membership. Respondents were asked to rate candidate standards on a 4-point Likert scale based on their perceived value in reporting studies in which HRQL was a study outcome (primary or secondary). Results were synthesized into draft reporting guidelines, which were further reviewed by the membership to inform the final guidance., Results: Forty-six existing candidate standards for reporting HRQL results in RCTs were synthesized to produce a 40 item survey that was completed electronically by 161 respondents. The majority of respondents rated all 40 items to be either 'essential' or 'desirable' when HRQL was a primary RCT outcome. Ratings changed when HRQL was a secondary study outcome. Feedback on the survey findings resulted in the Task Force generalizing the guidance to include patient-reported outcomes (PROs). The final guidance, which recommends standards for use in reporting PROs generally, and more specifically, for PROs identified as primary study outcomes, was approved by the ISOQOL Board of Directors., Conclusions: ISOQOL has developed a suite of recommended standards for reporting PRO results of RCTs. Improved reporting of PROs will enable accurate interpretation of evidence to inform patient choice, aid clinical decision making, and inform health policy.

Purpose: Measurement and reporting of health-related quality of life (HRQL) data have evolved considerably over the past 10 years. Our goal was to identify the current barriers to, and enablers of, the effective translation of HRQL outcome data from randomized clinical trials by investigating physician attitudes, knowledge, and education needs., Methods: We undertook a mixed qualitative and quantitative study of 33 oncologists' attitudes and educational needs around the value, interpretation, and application of HRQL data from cancer clinical trials. The approach was designed to identify barriers and enablers relating to the characteristics of the knowledge itself, to the potential users of the knowledge, and to the environment in which the knowledge is used., Results: The majority of barriers and enablers identified were "second order", i.e., related to the understandability and generalizability of the data, its presentation, its accessibility within the medical literature, and its relevance to specific patient populations., Conclusions: Our results suggest knowledge translation (KT) of HRQL results would improve if the clinical trial HRQL data were easily accessible to clinicians, and presented in a comprehensible and clinically applicable format, which includes discussion of the relevance of the measurement domains and implications of the findings. We recommend that standards of clinical trial HRQL reporting be implemented in clinical journals.

Purpose: To assess the patterns of, and trends over time in, health-related quality of life (HRQL) reporting in randomized controlled trials (RCTs)., Methods: The English-language literature of RCTs published in 2002-2008 was identified using Medline, Embase, and Healthstar databases, in addition to the Cochrane Clinical Trials Registry. Eligible trials were phase III studies that included an HRQL outcome. Data were abstracted on eight outcomes derived from previously recommended quality standards for reporting HRQL, and on four outcomes describing how HRQL data are presented in RCT reports. Two readers examined each article; discrepancies were resolved through discussion and third review if required., Results: A sample of 794 RCTs was identified. HRQL was a primary outcome in 25.4% (200/794). One hundred and ten RCTs (14%) used "supplementary" reports (separate from the first publication) to report HRQL findings. The proportion of RCTs that met the eight quality indicators ranged from 15% (HRQL used in the calculation of sample size) to 81% (reporting instrument validity). RCTs with HRQL as a primary outcome or with a supplementary report had higher concordance on the quality measures. Reporting improved on many indicators over time. Substantive variation in how HRQL data are presented in RCTs was evident., Conclusions: Current practice of reporting HRQL outcomes in RCTs remains highly variable, both with regard to quality of reporting and the patterns of data analysis and presentation. This variation presents challenges for clinicians to apply these data in clinical practice. Consistent reporting practices, which are interpretable by clinicians, are required, as are processes to achieve this consistency in future reports.

Health-related quality of life (HRQL) is a prevalent outcome measure in clinical trials, but it is not known how best to communicate HRQL results to new patients with cancer. The purpose was to explore cancer patients' attitudes toward, and preferences for, 10 visual and written formats for communicating HRQL information. Using standardized qualitative methods, six focus groups were held (two groups in each of three cancer centres) with patients who had completed treatment for cancer at least 6 months earlier. Groups were stratified according to patients' education. To ensure consistency across centres, group moderators used the same detailed guide, training video, and props. Three investigators independently coded the resulting transcripts. Participants also rated each of the 10 presentation formats as to their perceived usefulness. Fourteen men and 19 women with a variety of cancer diagnoses participated; 13 (39%) participated in the three lower-education groups and 20 (61%) in the three higher-education groups. We found that simple formats (simple graphs or written text) were generally preferred to more complex graphical information, regardless of educational level. The same format was rated favourably by the highest proportion of participants in both the high (85%) and low (85%) education level groups. Individual patients, however, varied as to which visual format they most preferred. Patients did not wish to receive HRQL information out of context or without explanation. We conclude that cancer patients desire HRQL information, but vary in their preferences for its communication. Simple formats are generally preferred.