Outcome measures in multimodal rectal cancer trials. Issue 5 (May 2020)

• Record Type: Journal Article
• Authors: Fokas, Emmanouil; Glynne-Jones, Robert; Appelt, Ane; Beets-Tan, Regina; Beets, Geerard; Haustermans, Karin; Marijnen, Corrie; Minsky, Bruce D; Ludmir, Ethan; Quirke, Phil; Sebag-Montefiore, David; Garcia-Aguilar, Julio; Gambacorta, Maria Antonietta; Valentini, Vincenzo; Buyse, Marc; Rödel, Claus
• Is Part Of: Lancet oncology. Volume 21:Issue 5(2020)
• Page Start: e252
• Page End: e264
• Publication Date: 2020-05
• Subjects: Oncology -- Periodicals; Neoplasms -- Periodicals; Cancérologie -- Périodiques; Oncologie; Oncology; Periodicals; Electronic journals; 616.994005
• DOI: 10.1016/S1470-2045(20)30024-3
• Languages: English
• ISSNs: 1470-2045

Abstract

Summary: There is a large variability regarding the definition and choice of primary endpoints in phase 2 and phase 3 multimodal rectal cancer trials, resulting in inconsistency and difficulty of data interpretation. Also, surrogate properties of early and intermediate endpoints have not been systematically assessed. We provide a comprehensive review of clinical and surrogate endpoints used in trials for non-metastatic rectal cancer. The applicability, advantages, and disadvantages of these endpoints are summarised, with recommendations on clinical endpoints for the different phase trials, including limited surgery or non-operative management for organ preservation. We discuss how early and intermediate endpoints, including patient-reported outcomes and involvement of patients in decision making, can be used to guide trial design and facilitate consistency in reporting trial results in rectal cancer.