TY - JOUR
T1 - Outcome measures in multimodal rectal cancer trials
AU - Fokas, Emmanouil
AU - Glynne-Jones, Robert
AU - Appelt, Ane
AU - Beets-Tan, Regina
AU - Beets, Geerard
AU - Haustermans, Karin
AU - Marijnen, Corrie
AU - Minsky, Bruce D.
AU - Ludmir, Ethan
AU - Quirke, Phil
AU - Sebag-Montefiore, David
AU - Garcia-Aguilar, Julio
AU - Gambacorta, Maria Antonietta
AU - Valentini, Vincenzo
AU - Buyse, Marc
AU - Rodel, Claus
N1 - Publisher Copyright:
© 2020 Elsevier Ltd
PY - 2020/5
Y1 - 2020/5
N2 - 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.
AB - 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.
KW - TOTAL MESORECTAL EXCISION
KW - SURROGATE END-POINTS
KW - RANDOMIZED PHASE-III
KW - SHORT-COURSE RADIOTHERAPY
KW - DISEASE-FREE SURVIVAL
KW - QUALITY-OF-LIFE
KW - PREOPERATIVE RADIOTHERAPY
KW - FOLLOW-UP
KW - POSTOPERATIVE CHEMORADIOTHERAPY
KW - LOCAL RECURRENCE
U2 - 10.1016/S1470-2045(20)30024-3
DO - 10.1016/S1470-2045(20)30024-3
M3 - (Systematic) Review article
SN - 1470-2045
VL - 21
SP - E252-E264
JO - Lancet oncology
JF - Lancet oncology
IS - 5
ER -