title: Health-related quality-of-life as co-primary endpoint in randomized clinical trials in oncology.
abstract: 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.
authors.affiliation: University Hospital of Besançon, Methodology and Quality of Life in Oncology Unit, Besançon, France.
authors.forename: Frédéric
authors.initials: F
authors.lastname: Fiteni
authors.forename: Alhousseiny
authors.initials: A
authors.lastname: Pam
authors.forename: Amélie
authors.initials: A
authors.lastname: Anota
authors.forename: Dewi
authors.initials: D
authors.lastname: Vernerey
authors.forename: Sophie
authors.initials: S
authors.lastname: Paget-Bailly
authors.forename: Virginie
authors.initials: V
authors.lastname: Westeel
authors.forename: Franck
authors.initials: F
authors.lastname: Bonnetain
mesh_terms: D023381:Endpoint Determination; D006801:Humans; D009369:Neoplasms / Q000628:therapy*; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D015996:Survival Rate; D016896:Treatment Outcome
keywords: clinical trial; co-primary endpoint; composite endpoint; endpoint; health-related quality of life; methodology