Outcome measures in multimodal rectal cancer trials

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, Claus Rodel

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

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Abstract

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.

Original languageEnglish
Pages (from-to)E252-E264
Number of pages13
JournalLancet oncology
Volume21
Issue number5
DOIs
Publication statusPublished - May 2020

Keywords

  • TOTAL MESORECTAL EXCISION
  • SURROGATE END-POINTS
  • RANDOMIZED PHASE-III
  • SHORT-COURSE RADIOTHERAPY
  • DISEASE-FREE SURVIVAL
  • QUALITY-OF-LIFE
  • PREOPERATIVE RADIOTHERAPY
  • FOLLOW-UP
  • POSTOPERATIVE CHEMORADIOTHERAPY
  • LOCAL RECURRENCE

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