Reporting of patient reported outcome (PRO) in clinical trials: A systematic review of clinical trials.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 6590-6590 ◽  
Author(s):  
Liat Vidal-Fisher ◽  
Laura Vidal Boixader ◽  
Vasily Andrianov ◽  
Kelly Kevelin Curtis ◽  
Daniel Shepshelovich ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Randomized Trials ◽  
Patient Reported Outcome ◽  
Phase 3 ◽  
The Past ◽  
Patient Reported ◽  
Oncology Clinical Trials ◽  
Eortc Qlq C30 ◽  
Eortc Qlq

6590 Background: Clinically relevant outcomes as improvements in overall survival (OS) or quality of life (QOL) should guide decision-making. The FDA encourages the implementation of patient-centric measures in clinical trials. Over the past decade a growing number of protocols have included PROs in their outcome measures. We aimed to evaluate the frequency at which PRO measures, incorporated into clinical trials, are made publicly available, when trial results are published. Methods: We searched Citeline Trialtrove database, a registry of clinical trials, for randomized phase 3 trials of patients with non-small cell lung cancer (NSCLC), recruiting during 2006-2016, with PRO listed. We excluded trials evaluating supportive treatment, and those unpublished. We identified publications associated with the trial, and extracted various parameters, including whether or not PRO outcome was published. Results: Of 158 NSCLC trials identified, 99 listed at least 1 PRO. 24 trials were excluded (supportive care, unpublished). The commonly used scales were EORTC-QLQ C30, QLQ-LC13, LCSS, FACT-L, and EQ-5D. Study sponsor was industry in 45 trials, and industry-academic in 12 trials. Of 75 trials analyzed, 41 (55%) published the results of the PRO endpoint. Only 37% of the 75 included a comprehensive report of PRO, and many publications referenced the PRO briefly. Of 41 trials that reported PRO, only 21 (51%) provided information about missing PRO data. 59 trials were published as full-text, of which 40 (68%) reported the PRO, and 53% reported the PRO more than 6 months after the initial full publication. Of the trials that showed PFS benefit, with no OS benefit, 22/34 (65%) published the results of the PRO endpoints. Conclusions: Despite a growing emphasis on QOL and the inclusion of PROs in oncology clinical trials, and despite patient and healthcare provider efforts to record PRO data, a significant number of NSCLC randomized trials do not report the PRO. The collection of PRO data should result in routine, timely and appropriate reporting as part of the trial outcome publication, to allow for a thorough assessment of investigational clinically relevant treatment effect.

scholarly journals Review and Assessment of Patient-Reported Outcomes (PRO) in Patients with Chemotherapy-Induced Anemia (CIA)

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 5025-5025
Author(s):  
Antonia Morga ◽  
Chris Atzinger ◽  
Alexandra Barsdorf ◽  
Mary New ◽  
Ana Filipa Alexandre
Keyword(s):  
Clinical Trials ◽  
Conceptual Model ◽  
Cancer Patients ◽  
Patient Reported Outcomes ◽  
Signs And Symptoms ◽  
Phase 2 ◽  
Patient Reported ◽  
Eortc Qlq C30 ◽  
Eortc Qlq

Abstract OBJECTIVES: Chemotherapy-induced anemia (CIA) is a common adverse event of cancer treatment and is associated with several symptoms that negatively impact health-related quality of life (HRQoL). Due to the nature of CIA signs and symptoms, the use of fit-for-purpose PRO measures reflecting patients' experience of the disease and its treatment is an important step in drug development and evaluation. In this project, we aimed to review and assess the most commonly used PRO measures to best capture the experiences of patients with non-myeloid malignancies. METHODS: A targeted literature review (TLR), including searches of the OVID database, US FDA/European Medicines Agency (EMA), clinical trial databases, regulatory labelling data and health technology assessment (HTA) reports/payer landscape recommendations, was conducted to identify PRO measures used to capture symptoms and HRQoL outcomes in non-myeloid cancer patients with CIA (2008-2021). PRO measures were identified from published studies, phase 2-4 clinical trials, and categorized according to the concept(s) measured and cancer type. Study outcomes were used to determine the optimal PRO strategy to capture the CIA symptoms of patients with non-myeloid cancer. The concepts captured from PROs identified in the TLR were used to develop a preliminary 'conceptual model': a graphical representation of signs and symptoms, and the perceived impact on HRQoL. Selected PRO measures, most relevant to the study objectives, were evaluated for their reliability, validity, responsiveness, and extent to which they cover HRQoL domains of interest and, in relation to the current regulatory environment, industry standards and guidance (e.g., FDA Guidance For Industry: Patient-Reported Outcome Measures, 2009). Reviews of the HTA guidelines and reports were conducted to provide insights into the applicability of PRO data from an HTA perspective. RESULTS: The conceptual model captured fatigue as the most important disease-related symptom experienced by non-myeloid cancer patients with CIA (Figure). The OVID search identified 17 PRO measures, which were then categorized by the key CIA-related concepts they assessed, including: sleep quality; HRQoL; anemia-related symptoms; depression/anxiety; and work productivity/activity impairment. The most frequently used PRO was the Functional Assessment of Cancer Therapy-Fatigue (FACT-F, N=11), followed by the FACT-Anemia (FACT-An, N=6). Ten PRO measures were identified from Phase 2-4 ongoing/completed US clinical trials and 6 PROs from EU clinical trials, with FACT-F (N=13) being the most common instrument used . FDA and EMA HRQoL-related product labelling information included the FACT-An, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) and Cancer Linear Analogue Scale (CLAS). From the TLR results, 9 PRO measures were selected for further review: FACT-An (An, Anemia); FACT-F; FACT-G (G, General); Functional Assessment of Chronic Illness Therapy-F; Patient-reported Outcomes Measurement Information System-F; Brief Fatigue Inventory; Visual Analogue Scale-F; Fatigue Symptom Inventory; and EORTC QLQ-C30. Of these, FACT-An was the only PRO measure specifically developed to measure fatigue and HRQoL in anemic cancer patients. FACT-An includes four HRQoL domains (assessing physical, social/family, emotional and functional wellbeing) from the FACT-G, plus an anemia subscale that comprises a fatigue subscale (13 items) and 7 anemia-specific questions. Results from the HTA/payer landscape review also indicated that the majority of recent HTA submissions reported findings from EORTC QLQ-C30 alongside the EORTC disease-specific adaptations for oncology-related indications and the EQ-5D. Many of the recent HTAs reported data from 2 or more different PRO measures. CONCLUSIONS: CIA impacts various aspects of patients' HRQoL; however, current PRO measures may only partially capture concepts of interest to patients. Multiple measures may be needed to adequately reflect all domains affected by CIA. Future studies may consider conducting concept elicitation interviews with patients and clinicians, to inform future updates to the conceptual model, and validation work to estimate meaningful change thresholds for these measures if not available. Figure 1 Figure 1. Disclosures Morga: Astellas Pharma Europe Ltd.: Current Employment. Atzinger: Astellas Pharma Inc.: Current Employment. Barsdorf: Clinical Outcomes Solutions: Current Employment. New: IQVIA: Current Employment. Alexandre: Astellas Pharma Europe B.V.: Current Employment.

scholarly journals Scope and heterogeneity of outcomes reported in randomized trials in patients receiving peritoneal dialysis

2020 ◽  
Author(s):  
Karine E Manera ◽  
David W Johnson ◽  
Yeoungjee Cho ◽  
Benedicte Sautenet ◽  
Jenny Shen ◽  
...  
Keyword(s):  
Peritoneal Dialysis ◽  
Patient Reported Outcomes ◽  
Randomized Trials ◽  
Primary Outcome ◽  
Median Number ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Inform Decision Making ◽  
Solute Clearance

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.

scholarly journals Measurement properties of patient-reported outcome measures (PROMs) in hyperhidrosis: a systematic review

2021 ◽  
Author(s):  
Michaela Gabes ◽  
Helge Knüttel ◽  
Gesina Kann ◽  
Christina Tischer ◽  
Christian J. Apfelbacher
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Measurement Properties ◽  
High Quality ◽  
High Quality Evidence ◽  
Patient Reported ◽  
Quality Evidence

Abstract Purpose To critically appraise, compare and summarize the quality of all existing PROMs that have been validated in hyperhidrosis to at least some extend by applying the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology. Thereby, we aim to give a recommendation for the use of PROMs in future clinical trials in hyperhidrosis. Methods We considered studies evaluating, describing or comparing measurement properties of PROMs as eligible. A systematic literature search in three big databases (MEDLINE, EMBASE and Web of Science) was performed. We assessed the methodological quality of each included study using the COSMIN Risk of Bias checklist. Furthermore, we applied predefined quality criteria for good measurement properties and finally, graded the quality of the evidence. Results Twenty-four articles reporting on 13 patient-reported outcome measures were included. Three instruments can be further recommended for use. They showed evidence for sufficient content validity and moderate- to high-quality evidence for sufficient internal consistency. The methodological assessment showed existing evidence gaps for eight other PROMs, which therefore require further validation studies to make an adequate decision on their recommendation. The Hyperhidrosis Disease Severity Measure-Axillary (HDSM-Ax) and the short-form health survey with 36 items (SF-36) were the only questionnaires not recommended for use in patients with hyperhidrosis due to moderate- to high-quality evidence for insufficient measurement properties. Conclusion Three PROMs, the Hyperhidrosis Quality of Life Index (HidroQoL), the Hyperhidrosis Questionnaire (HQ) and the Sweating Cognitions Inventory (SCI), can be recommended for use in future clinical trials in hyperhidrosis. Results obtained with these three instruments can be seen as trustworthy. Nevertheless, further validation of all three PROMs is desirable. Systematic review registration PROSPERO CRD42020170247

scholarly journals Patient-Reported Outcome Measures for Post-mastectomy Breast Reconstruction: A Systematic Review of Development and Measurement Properties

2020 ◽  
Vol 28 (1) ◽  
pp. 386-404 ◽  
Author(s):  
C. F. Davies ◽  
R. Macefield ◽  
K. Avery ◽  
J. M. Blazeby ◽  
S. Potter
Keyword(s):  
Systematic Review ◽  
Breast Reconstruction ◽  
Content Validity ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Measurement Properties ◽  
Future Studies ◽  
Patient Reported ◽  
Eortc Qlq

Abstract Background Breast reconstruction (BR) is performed to improve outcomes for patients undergoing mastectomy. A recently developed core outcome set for BR includes six patient-reported outcomes that should be measured and reported in all future studies. It is vital that any instrument used to measure these outcomes as part of a core measurement set be robustly developed and validated so data are reliable and accurate. The aim of this systematic review is to evaluate the development and measurement properties of existing BR patient-reported outcome measures (PROMs) to inform instrument selection for future studies. Methods A PRISMA-compliant systematic review of development and validation studies of BR PROMs was conducted to assess their measurement properties. PROMs with adequate content validity were assessed using three steps: (1) the methodological quality of each identified study was assessed using the COSMIN Risk of Bias checklist; (2) criteria were applied for assessing good measurement properties; and (3) evidence was summarized and the quality of evidence assessed using a modified GRADE approach. Results Fourteen articles reported the development and measurement properties of six PROMs. Of these, only three (BREAST-Q, BRECON-31, and EORTC QLQ-BRECON-23) were considered to have adequate content validity and proceeded to full evaluation. This showed that all three PROMs had been robustly developed and validated and demonstrated adequate quality. Conclusions BREAST-Q, BRECON-31, and EORTC QLQ-BRECON-23 have been well-developed and demonstrate adequate measurement properties. Work with key stakeholders is now needed to generate consensus regarding which PROM should be recommended for inclusion in a core measurement set.

Comparison of patient-reported late treatment toxicity (LENT–SOMA) with quality of life (EORTC QLQ-C30 and QLQ-H&N35) assessment after head and neck radiotherapy

2010 ◽  
Vol 97 (2) ◽  
pp. 270-275 ◽  
Author(s):  
Kean Fatt Ho ◽  
Damien J.J. Farnell ◽  
Jacqueline A. Routledge ◽  
Meriel P. Burns ◽  
Andrew J. Sykes ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Head And Neck ◽  
Treatment Toxicity ◽  
Patient Reported ◽  
Eortc Qlq C30 ◽  
Late Treatment ◽  
Eortc Qlq ◽  
Head And Neck Radiotherapy

scholarly journals Capturing Patient-Reported Outcome (PRO) Data Electronically: The Past, Present, and Promise of ePRO Measurement in Clinical Trials

2014 ◽  
Vol 8 (4) ◽  
pp. 301-309 ◽  
Author(s):  
Stephen Joel Coons ◽  
Sonya Eremenco ◽  
J. Jason Lundy ◽  
Paul O’Donohoe ◽  
Hannah O’Gorman ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Patient Reported Outcome ◽  
The Past ◽  
Patient Reported

Difficulties with Measuring the Patient's Perspective in Non Hodgkin's Lymphoma - a Review of Patient Reported Outcome Measures

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4703-4703
Author(s):  
Jyotsna Mehta ◽  
Florence Joulain ◽  
Peter C Trask ◽  
Bonnie Teschendorf
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Clinical Benefit ◽  
Cell Lymphoma ◽  
B Cell Lymphoma ◽  
Medical Attention ◽  
Eortc Qlq C30 ◽  
Eortc Qlq ◽  
Pro Instruments

Abstract Abstract 4703 Aims: Patients with NHL present with a classic array of symptoms reflecting the origin of the cancer itself. Symptoms include painless swelling of lymph nodes, increased sensitivity to alcohol, weight loss which can be substantial, persistent fever, soaking night sweats, itchy skin, coughing, difficulty breathing, chest or upper back pain and persistent weakness and tiredness. The severity of symptoms alters the patient's perception of quality of life, their capacity to perform usual activities, and results in seeking medical attention. Due to profound symptoms, it is critical to use excellent measurement tools to record change and demonstrate clinical benefit in trials. The objective of the study was to describe and assess the PRO instruments used in lymphoma, summarize PROs used in ongoing clinical trials, identify gaps in existing PRO measures and evaluate the potential for labeling when using these measures for patient self-report in advanced NHL, particularly as it relates to selecting and implementing PROs. Methods: An in depth literature review was conducted. Elsevier and Medline databases were consulted using Embase platform. Studies were included if they were: published from 01/01–12/11, in English, and included terms related to lymphoma disease and PROs. A thorough review of abstracts was performed. Studies where PRO instruments were used and/or psychometric validation was performed were included. References for selected articles were used to identify other relevant sources. Clinical trials.gov was also used to search for lymphoma trials from 2001–2011. Results: Of 1278 hits, 17 articles met the search criteria. 7 PRO instruments were reported or used in NHL: 2 lymphoma specific questionnaires FACT-Lym (Functional Assessment of Cancer Therapy-Lymphoma module), FACT FLymSI-18 (FACT-Lymphoma Symptom Index), 5 cancer-specific instruments (EORTC-QLQ-C30, FACT-G, CARES (Cancer Rehabilitation Evaluation System), CARES-SF, QOL-CS (Quality of Life-Cancer Survivors), IOC (Impact Of Cancer scale). The 2 most widely used PROs were FACT-Lym and the EORTC QLQ C30. PRO instruments have been included in ongoing phase 2 and 3 clinical trials (Table 1) as secondary endpoints, including 2 in Diffuse Large B-cell lymphoma (DLBCL), 4 in Follicular lymphoma (FL), and 1 each in indolent NHL or mantle cell lymphoma (MCL). EORTC QLQ C30 was the most common instrument followed by FACT-Lym. No specific labeling claims were found in labels made to date for NHL related compounds to FDA or EMA. Conclusions: While there are some lymphoma specific measures, most ongoing trials are using only cancer specific instruments such as EORTC QLQ C30. Several instruments contain only general cancer related symptoms, but not NHL specific symptoms. To more completely understand the burden of disease and treatment effects with NHL through the eyes of patients, evaluation of existing instruments as well as potential instrument development/modification may be warranted. NHL symptom-specific measures could demonstrate therapeutic effectiveness, enhance our understanding of the impact of NHL and provide more evidence of clinical benefit for developing treatments. Disclosures: Mehta: Sanofi: Employment. Joulain:Sanofi: Employment, Equity Ownership. Trask:Sanofi: Employment.

scholarly journals Multicenter Prospective Cohort Study of the Patient-Reported Outcome Measures PRO-CTCAE and CAT EORTC QLQ-C30 in Major Abdominal Cancer Surgery (PATRONUS): A Student-Initiated German Medical Audit (SIGMA) Study

2021 ◽  
Author(s):  
André L. Mihaljevic ◽  
Keyword(s):  
Quality Of Life ◽  
Postoperative Complications ◽  
Computerized Adaptive Testing ◽  
Cancer Surgery ◽  
Life Questionnaire ◽  
Cancer Symptoms ◽  
Patient Reported ◽  
Eortc Qlq C30 ◽  
Eortc Qlq

Abstract Background The patient-reported outcomes (PRO) version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) and the computerized adaptive testing (CAT) version of the EORTC quality-of-life questionnaire QLQ-C30 have been proposed as new PRO measures in oncology; however, their implementation in patients undergoing cancer surgery has not yet been evaluated. Methods Patients undergoing elective abdominal cancer surgery were enrolled in a prospective multicenter study, and postoperative complications were recorded according to the Dindo–Clavien classification. Patients reported PRO data using the CAT EORTC QLQ-C30 and the PRO-CTCAE to measure 12 core cancer symptoms. Patients were followed-up for 6 months postoperatively. The study was carried out by medical students of the CHIR-Net SIGMA study network. Results Data of 303 patients were obtained and analyzed across 15 sites. PRO-CTCAE symptoms ‘poor appetite’, ‘fatigue’, ‘exhaustion’ and ‘sleeping problems’ increased after surgery and climaxed 10–30 days postoperatively. At 3–6 months postoperatively, no PRO-CTCAE symptom differed significantly to baseline. Patients reported higher ‘social functioning’ (p = 0.021) and overall quality-of-life scores (p < 0.05) 6 months after cancer surgery compared with the baseline level. There was a lack of correlation between postoperative complications or death and any of the PRO items evaluated. Feasibility endpoints for student-led research were met. Conclusion The two novel PRO questionnaires were successfully applied in surgical oncology. Postoperative complications do not affect health-reported quality-of-life or common cancer symptoms following major cancer surgery. The feasibility of student-led multicenter clinical research was demonstrated, but might be enhanced by improved student training.

scholarly journals Development of Patient-Reported Outcome Measures (Symptoms and Impacts) in Adults with Pyruvate Kinase Deficiency

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3447-3447
Author(s):  
Sam Salek ◽  
Audra N. Boscoe ◽  
Chris Evans ◽  
Shayna Egan ◽  
Ted Wells ◽  
...  
Keyword(s):  
Pyruvate Kinase ◽  
Research Funding ◽  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Specific Patient ◽  
Patient Reported ◽  
Item Wording ◽  
Eortc Qlq C30 ◽  
Eortc Qlq ◽  
Disease Specific

Introduction: Pyruvate kinase (PK) deficiency is a rare, congenital autosomal recessive hemolytic anemia managed with supportive treatments, including transfusion, splenectomy, and iron chelation. Disease-directed treatments, including a small molecule PK activator and gene therapy, are currently in development. No disease-specific patient-reported outcome (PRO) measures have been validated for use in this patient population. The objective of this initiative was to develop PRO measures for assessing symptoms and impacts of PK deficiency and compare them to existing, non-disease-specific measures previously recommended for use in this disease area. Methods: A targeted literature review was conducted to inform the development of a preliminary hypothesized conceptual framework to identify signs, symptoms, and impacts commonly experienced by patients with PK deficiency and to inform the direction and content of interviews with such patients. Concept elicitation interviews were conducted with 21 adults with PK deficiency from the US, Netherlands, and Germany. Draft items were then tested in cognitive interviews with 20 adults with PK deficiency to further establish content validity and revised based on the results. A comparison was conducted between concepts included in the newly developed PK deficiency disease-specific measures and the domain structure and item concepts included in the EORTC QLQ-C30 and SF-36v2 to evaluate the extent of differences and conceptual overlap with instruments that had previously been recommended in this population. Specific attributes compared included face validity (i.e., conceptual coverage and inclusion of proximal symptoms and/or impacts) and measurement characteristics (i.e., item wording, recall, and response options). Results: Two measures, the PK Deficiency Diary (PKDD), a 7-item measure of the core signs and symptoms of PK deficiency, and PK Deficiency Impact Assessment (PKDIA), a 14-item measure of the impacts of PK deficiency on patients' HRQoL, were developed. A comparison of the newly drafted measures to the EORTC QLQ-C30 and SF-36v2 demonstrated minimal similarities in concepts, domains, item wording, and recall period. Of the 7 concepts in the PKDD, only 3 were common to the EORTC QLQ-C30, 4 were common to the SF36v2, and 2 were related but did not match exactly (i.e., "bone pain" and "pain"). Of the 12 distinct concepts in the PKDIA, only 5 were common to the EORTC QLQ-C30, 2 were common to the SF-36v2, and 3 were related but did not match exactly (i.e., difficulty starting things, difficulty finishing things, and difficulty performing moderate physical activity). Conclusions: This research demonstrates that the EORTC-QLQ-C30 and SF-36v2 lack the appropriate conceptual relevance and coverage of disease-specific signs, symptoms, and impacts most relevant and burdensome to patients with PK deficiency. The newly developed PKDD and PKDIA may be useful tools in clinical trials in patients with PK deficiency. Psychometric validation of these measures is currently underway. Disclosures Salek: Pfizer: Honoraria, Speakers Bureau; Merck: Consultancy; Agios Pharmaceuticals, Inc.: Consultancy, Honoraria. Boscoe:Agios Pharmaceuticals, Inc.: Employment, Equity Ownership. Evans:Agios Pharmaceuticals, Inc.: Consultancy, Research Funding. Egan:Agios Pharmaceuticals, Inc.: Consultancy, Research Funding. Wells:Agios Pharmaceuticals, Inc.: Consultancy, Research Funding. Piantedosi:Agios Pharmaceuticals, Inc.: Employment. Grace:Agios Pharmaceuticals, Inc: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Research Funding. Storm:Agios: Employment.

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