Health-related quality-of-life as co-primary endpoint in randomized clinical trials in oncology.
Fiche publication
Date publication
mai 2015
Auteurs
Membres identifiés du Cancéropôle Est :
Pr WESTEEL Virginie
Tous les auteurs :
Fiteni F, Pam A, Anota A, Vernerey D, Paget-Bailly S, Westeel V, Bonnetain F
Lien Pubmed
Résumé
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.
Référence
Expert Rev Anticancer Ther. 2015 May 31:1-7.