1. Outcome measures in multimodal rectal cancer trials
- Author
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Karin Haustermans, Geerard L. Beets, David Sebag-Montefiore, Vincenzo Valentini, Marc Buyse, Bruce D. Minsky, Regina G. H. Beets-Tan, Julio Garcia-Aguilar, Phil Quirke, Ane L Appelt, Emmanouil Fokas, Rob Glynne-Jones, Ethan B. Ludmir, Claus Rödel, Maria Antonietta Gambacorta, and Corrie A.M. Marijnen
- Subjects
Research design ,medicine.medical_specialty ,Time Factors ,Endpoint Determination ,Colorectal cancer ,MEDLINE ,LOCAL RECURRENCE ,03 medical and health sciences ,0302 clinical medicine ,QUALITY-OF-LIFE ,Clinical endpoint ,DISEASE-FREE SURVIVAL ,Humans ,Combined Modality Therapy ,Medicine ,SHORT-COURSE RADIOTHERAPY ,Patient Reported Outcome Measures ,Intensive care medicine ,PREOPERATIVE RADIOTHERAPY ,Settore MED/36 - DIAGNOSTICA PER IMMAGINI E RADIOTERAPIA ,030304 developmental biology ,Clinical Trials as Topic ,0303 health sciences ,Rectal Neoplasms ,Surrogate endpoint ,business.industry ,TOTAL MESORECTAL EXCISION ,Outcome measures ,RANDOMIZED PHASE-III ,medicine.disease ,Treatment Outcome ,Oncology ,Research Design ,030220 oncology & carcinogenesis ,FOLLOW-UP ,business ,SURROGATE END-POINTS ,POSTOPERATIVE CHEMORADIOTHERAPY - 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.
- Published
- 2020
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