Patient-reported outcome (PRO) assessment and reporting in first-line (1L) locally advanced or metastatic urothelial cancer (la/mUC) clinical trials: Results of a systematic literature review (SLR).

Abstract

757 Background: Although maintaining health-related quality of life (HRQOL) is a primary goal of advanced cancer care, HRQOL and PROs usually are not measured or reported as robustly as efficacy outcomes in clinical trials. One example is la/mUC, an aggressive and incurable disease with a profound effect on patient HRQOL and functioning. With its changing therapeutic landscape in which newer agents with various efficacy and toxicity profiles are incorporated into clinical care, more attention must be given to the optimal deployment of PRO measures (PROMs). Our aim was to conduct a critical evaluation of currently used PROMs in la/mUC 1L trials. Methods: A SLR was conducted using 5 online databases to identify publications up to May 29, 2024 and recent conference abstracts of phase 2 or 3 clinical trials and real-world evidence (RWE) studies reporting PROs in the treatment of la/mUC. Results: Forty-nine studies were identified in the SLR (37 clinical trials; 12 RWE studies). Of the 37 clinical trials, 18 were in the 1L population. The most common PROMs in 1L trials were the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 (n = 15) and the EuroQol-5D (n = 6). Other PROMs used were the Brief Pain Inventory–Short Form (n = 2), Functional Assessment of Cancer Therapy–Bladder (FACT-Bl; n = 1), National Comprehensive Cancer Network/FACT Bladder Symptom Index-18 (n = 1), and the Generic Quality of Life Inventory-74 (n = 1). PROMs were almost exclusively included as secondary endpoints (n = 16); only 2 trials included PROMs as exploratory endpoints. Prespecified analyses for HRQOL assessments were generally not stated in publications, and analytical methods and reporting varied. Of the 15 trials that used the EORTC QLQ-C30, 93% reported general health status/QOL, and 60% and 47% reported functional and symptom scales, respectively. Overall, HRQOL, functioning, and/or symptoms were maintained or improved, but meaningful changes in PRO scores reported were difficult to interpret due to the ways in which minimal clinically important difference thresholds were applied. Conclusions: Understanding the impact of various 1L treatment regimens on PROs relies on consistent assessment and transparent reporting. The findings of this SLR highlight the challenges in conducting cross-trial comparisons of PROs due to heterogeneity in study designs, patient characteristics, choice of PROMs, statistical analyses, and reporting of outcomes. To understand the symptom burden associated with novel therapeutics, it is recommended that the la/mUC research community develop a consensus regarding PRO development and implementation. This will facilitate interpretation of the patient experience, enhance clinical care, and guide informed treatment decision-making by clinicians and patients with la/mUC.