Traditionally, the efficacy of cancer treatment in patients with advance or metastatic disease in clinical studies has been studied using overall survival (OS) and more recently tumor-based endpoints such as progression-free survival, measurements of response to treatment. However, these seem not to be the relevant clinical endpoints in current situation if such endpoints were no validated as surrogate of OS to demonstrate the clinical efficacy. Appropriate, meaningful, primary patient-oriented and patient-reported endpoints that adequately measure the effects of new therapeutic interventions are then crucial for the advancement of clinical research in metastatic colorectal cancer to complement the results of tumor-based endpoints. Health-related quality of life (HRQoL), is effectively an evaluation of quality of life and its relationship with health over time. HRQoL includes the patient report at least of the way a disease or its treatment affects its physical, emotional and social well-being. Over the past few years, several phase III trials in a variety of solid cancers have assessed the incremental value of HRQoL in addition to the traditional endpoints of tumor response and survival results. HRQoL could provide not only complementary clinical data to the primary outcomes, but also more precise predictive and prognostic value. This endpoint is useful for both clinicians and patients in order to achieve the dogma of precision medicine.
The present article examines the use of HRQoL in phase III metastatic colorectal cancer clinical trials, outlines the importance of HRQoL assessment methods, analysis, and results presentation. Moreover, it discusses the relevance of including HRQoL as a primary/co-primary endpoint to support the progression-free survival results and to assess efficacy of treatment in the advanced disease setting.
Health-related quality of life, longitudinal, methodology, colorectal cancer, clinical trial, endpoint
Topic: cancer colorectal cancer emotion phase 3 clinical trials complement system proteins neoplasm metastasis personal satisfaction neoplasms prognosis quality of life cancer therapy colorectal cancer metastatic health-related quality of life therapeutic intervention surrogate endpoints clinical research treatment effectiveness progression-free survival precision medicine
Issue Section: Gastrointestinal tumors Review