TY - JOUR
T1 - Neoadjuvant Chemotherapy, Excision, and Observation for Early Rectal Cancer
T2 - The Phase II NEO Trial (CCTG CO.28) Primary End Point Results
AU - Kennecke, Hagen F.
AU - O'Callaghan, Chris J.
AU - Loree, Jonathan M.
AU - Moloo, Hussein
AU - Auer, Rebecca
AU - Jonker, Derek J.
AU - Raval, Manoj
AU - Musselman, Reilly
AU - Ma, Grace
AU - Caycedo-Marulanda, Antonio
AU - Simianu, Vlad V.
AU - Patel, Sunil
AU - Pitre, Lacey D.
AU - Helewa, Ramzi
AU - Gordon, Vallerie L.
AU - Neumann, Katerina
AU - Nimeiri, Halla
AU - Sherry, Max
AU - Tu, Dongsheng
AU - Brown, Carl J.
N1 - Publisher Copyright:
© American Society of Clinical Oncology.
PY - 2023/1/10
Y1 - 2023/1/10
N2 - PURPOSEOrgan-sparing therapy for early-stage I/IIA rectal cancer is intended to avoid functional disturbances or a permanent ostomy associated with total mesorectal excision (TME). The objective of this phase II trial was to determine the outcomes and organ-sparing rate of patients with early-stage rectal cancer treated with neoadjuvant chemotherapy followed by transanal excision surgery (TES).METHODSThis phase II trial included patients with clinical T1-T3abN0 low- or mid-rectal adenocarcinoma eligible for endoscopic resection who were treated with 3 months of chemotherapy (modified folinic acid-fluorouracil-oxaliplatin 6 or capecitabine-oxaliplatin). Those with evidence of response proceeded to transanal endoscopic surgery 2-6 weeks later. The primary end point was protocol-specified organ preservation rate, defined as the proportion of patients with tumor downstaging to ypT0/T1N0/X and who avoided radical surgery.RESULTSOf 58 patients enrolled, all commenced chemotherapy and 56 proceeded to surgery. A total of 33/58 patients had tumor downstaging to ypT0/1N0/X on the surgery specimen, resulting in an intention-to-treat protocol-specified organ preservation rate of 57% (90% CI, 45 to 68). Of 23 remaining patients recommended for TME surgery on the basis of protocol requirements, 13 declined and elected to proceed directly to observation resulting in 79% (90% CI, 69 to 88) achieving organ preservation. The remaining 10/23 patients proceeded to recommended TME of whom seven had no histopathologic residual disease. The 1-year and 2-year locoregional relapse-free survival was, respectively, 98% (95% CI, 86 to 100) and 90% (95% CI, 58 to 98), and there were no distant recurrences or deaths. Minimal change in quality of life and rectal function scores was observed.CONCLUSIONThree months of induction chemotherapy may successfully downstage a significant proportion of patients with early-stage rectal cancer, allowing well-tolerated organ-preserving surgery.
AB - PURPOSEOrgan-sparing therapy for early-stage I/IIA rectal cancer is intended to avoid functional disturbances or a permanent ostomy associated with total mesorectal excision (TME). The objective of this phase II trial was to determine the outcomes and organ-sparing rate of patients with early-stage rectal cancer treated with neoadjuvant chemotherapy followed by transanal excision surgery (TES).METHODSThis phase II trial included patients with clinical T1-T3abN0 low- or mid-rectal adenocarcinoma eligible for endoscopic resection who were treated with 3 months of chemotherapy (modified folinic acid-fluorouracil-oxaliplatin 6 or capecitabine-oxaliplatin). Those with evidence of response proceeded to transanal endoscopic surgery 2-6 weeks later. The primary end point was protocol-specified organ preservation rate, defined as the proportion of patients with tumor downstaging to ypT0/T1N0/X and who avoided radical surgery.RESULTSOf 58 patients enrolled, all commenced chemotherapy and 56 proceeded to surgery. A total of 33/58 patients had tumor downstaging to ypT0/1N0/X on the surgery specimen, resulting in an intention-to-treat protocol-specified organ preservation rate of 57% (90% CI, 45 to 68). Of 23 remaining patients recommended for TME surgery on the basis of protocol requirements, 13 declined and elected to proceed directly to observation resulting in 79% (90% CI, 69 to 88) achieving organ preservation. The remaining 10/23 patients proceeded to recommended TME of whom seven had no histopathologic residual disease. The 1-year and 2-year locoregional relapse-free survival was, respectively, 98% (95% CI, 86 to 100) and 90% (95% CI, 58 to 98), and there were no distant recurrences or deaths. Minimal change in quality of life and rectal function scores was observed.CONCLUSIONThree months of induction chemotherapy may successfully downstage a significant proportion of patients with early-stage rectal cancer, allowing well-tolerated organ-preserving surgery.
UR - https://www.scopus.com/pages/publications/85141702241
U2 - 10.1200/JCO.22.00184
DO - 10.1200/JCO.22.00184
M3 - Article
C2 - 35981270
AN - SCOPUS:85141702241
SN - 0732-183X
VL - 41
SP - 233
EP - 242
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
IS - 2
ER -