Health-related quality-of-life as co-primary endpoint in randomized clinical trials in oncology

作者：Fiteni Frederic^*; Pam Alhousseiny; Anota Amelie; Vernerey Dewi; Paget Bailly Sophie; Westeel Virginie; Bonnetain Franck

来源：Expert Review of Anticancer Therapy, 2015, 15(8): 885-891.

DOI：10.1586/14737140.2015.1047768

• clinical trial
• composite endpoint
• co-primary endpoint
• endpoint
• health-related quality of life
• methodology

Overall survival (OS) has been considered as the most relevant primary endpoint but trials using OS often require large numbers of patients and long-term follow-up. Therefore composite endpoints, which are assessed earlier, are frequently used as primary endpoint but suffer from important limitations specially a lack of validation as surrogate of OS. Therefore, Health-related quality of life (HRQoL) could be considered as an outcome to judge efficacy of a treatment. An alternative approach would be to combine HRQoL with composite endpoints as co-primary endpoint to ensure a clinical benefit for patients of a new therapy. The decision rules of such design, the procedure to control the Type I error and the determination of sample size remain questions to debate. Here, we discusses HRQoL as co-primary endpoints in randomized clinical trials in oncology and provide some solutions to promote such design.

• 出版日期2015