Health-related quality of life in biliary tract cancer clinical trials. The current state and future directions

Abstract

Background

Patients with biliary tract cancers (BTCs) often experience a decline in health-related quality of life (HRQoL). Patient-reported outcome measures (PROMs), usually collected through HRQoL questionnaires, offer a standardized approach to capturing the patient’s perspective. This study aimed to provide a comprehensive analysis of HRQoL in BTC clinical trials.

Design

After database screening, the 30 most impactful clinical trials in BTC were identified for evaluation of HRQoL reporting and analysis. The primary objective was to determine the proportion of trials that included HRQoL assessment. Secondary objectives were to examine different aspects of HRQoL analysis and to assess the robustness of reporting using the recently developed European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS) HRQoL checklist.

Results

Fifteen trials (50%) included HRQoL in their design, with published results available for 14. The most frequent HRQoL outcome was stability (i.e. neither improvement nor deterioration). The median ESMO-MCBS HRQoL checklist score was 11 (interquartile range 9.00-11.75).

Conclusions

HRQoL remains only partially addressed in BTC clinical trials. Tools such as the ESMO HRQoL checklist and electronic PROMs may strengthen the assessment and highlight the importance of HRQoL in both clinical trials and routine practice.