Patient-reported outcome measurement and reporting for patients with advanced renal cell carcinoma: A systematic literature review Review
| Authors: | Motzer, R. J.; Rane, P. P.; Saretsky, T. L.; Pawar, D.; Martin Nguyen, A.; Sundaram, M.; Burgents, J.; Pandey, R.; Rudell, K. |
| Review Title: | Patient-reported outcome measurement and reporting for patients with advanced renal cell carcinoma: A systematic literature review |
| Abstract: | Context: In the oncology setting, patient-reported outcome measures (PROMs) provide important data that help to ensure patient-relevant endpoints are captured and reported. Use of this information for treatment decision-making by clinicians and patients in real-world settings is facilitated by consistent and transparent reporting of trial methods. Objective: To identify and compare PROMs used in advanced renal cell carcinoma (RCC) trials in terms of the rationale for the choice of measure, endpoint hierarchy (primary, secondary, exploratory), assessment time points, statistical methods, and statistical metrics for interpretation. Evidence acquisition: A systematic literature review via searches of four online databases (2016–2021) and recent conference abstracts (2019–2021) identified 2616 articles, of which 33 were included in the review according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Evidence synthesis: Among the 33 clinical studies included, 19 different PROMs were identified: three kidney cancer–specific scales, two cancer-specific scales, two generic scales, and 12 symptom-specific scales. The endpoint hierarchy for patient reported outcome (PRO) assessment was reported in 42% of the studies; one study included PROs as a primary endpoint. Reporting of time points, minimal important differences, and statistical analyses was highly heterogeneous. Conclusions: A diverse range of PROMs have been included in clinical studies for patients with advanced/metastatic RCC. Prespecified analyses for PRO assessments were generally not stated, while analytical methods and reporting varied. An improvement in alignment across studies would better inform regulatory, market-access, reimbursement, and clinical decision-making to improve patient care. Patient summary: We reviewed how the impact of cancer therapies on health outcomes from the patient's point of view is being measured in clinical trials for kidney cancer. The techniques and reporting varied across trials. Standardisation of how these data are captured and reported may improve care and decision-making for patients with kidney cancer. © 2023 European Association of Urology |
| Keywords: | review; sorafenib; angiogenesis inhibitor; bevacizumab; sunitinib; advanced cancer; alpha interferon; research design; outcome assessment; methodology; ipilimumab; quality of life; renal cell carcinoma; kidney neoplasms; protein tyrosine kinase inhibitor; temsirolimus; kidney tumor; carcinoma, renal cell; systematic review; pazopanib; patient-reported outcomes; patient reported outcome measures; axitinib; short form 36; everolimus; hospital anxiety and depression scale; randomized controlled trial (topic); international index of erectile function; patient outcome assessment; phase 2 clinical trial (topic); phase 3 clinical trial (topic); center for epidemiological studies depression scale; functional assessment of cancer therapy; patient-reported outcome; systematic literature review; cabozantinib; evaluation and follow up; nivolumab; lenvatinib; brief fatigue inventory; humans; human; pembrolizumab; functional assessment of chronic illness therapy fatigue scale; atezolizumab; functional assessment of cancer therapy general; immunological antineoplastic agent; md anderson symptom inventory; preferred reporting items for systematic reviews and meta-analyses; european organization for research and treatment of cancer quality of life questionnaire core 30; european quality of life 5 dimensions visual analogue scale; work productivity and activity impairment questionnaire; catequentinib; androgen deficiency in the aging male; cancer therapy satisfaction questionnaire; conference abstract; hand foot and mucositis symptom index |
| Journal Title: | European Urology |
| Volume: | 84 |
| Issue: | 4 |
| ISSN: | 0302-2838 |
| Publisher: | Elsevier Science, Inc. |
| Date Published: | 2023-10-01 |
| Start Page: | 406 |
| End Page: | 417 |
| Language: | English |
| DOI: | 10.1016/j.eururo.2023.07.006 |
| PUBMED: | 37550153 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | Review -- MSK corresponding author is Robert Motzer -- Source: Scopus |
