• Emmanouil Fokas, Robert Glynne-Jones, Ane Appelt, Regina Beets-Tan, Geerard Beets, Karin Haustermans, Corrie Marijnen, Bruce D Minsky, Ethan Ludmir, Phil Quirke, David Sebag-Montefiore, Julio Garcia-Aguilar, Maria Antonietta Gambacorta, Vincenzo Valentini, Marc Buyse, and Claus Rödel.
• Department of Radiotherapy and Oncology, University of Frankfurt, Frankfurt, Germany; German Cancer Consortium, German Cancer Research Center, Heidelberg, Germany; Frankfurt Cancer Institute, Frankfurt Germany. Electronic address: emmanouil.fokas@kgu.de.
• Lancet Oncol. 2020 May 1; 21 (5): e252-e264.

AbstractThere 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.Copyright © 2020 Elsevier Ltd. All rights reserved.

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