TY - JOUR
T1 - Assessment of a Watch-and-Wait Strategy for Rectal Cancer in Patients with a Complete Response after Neoadjuvant Therapy
AU - Smith, J. Joshua
AU - Strombom, Paul
AU - Chow, Oliver S.
AU - Roxburgh, Campbell S.
AU - Lynn, Patricio
AU - Eaton, Anne
AU - Widmar, Maria
AU - Ganesh, Karuna
AU - Yaeger, Rona
AU - Cercek, Andrea
AU - Weiser, Martin R.
AU - Nash, Garrett M.
AU - Guillem, Jose G.
AU - Temple, Larissa K.F.
AU - Chalasani, Sree B.
AU - Fuqua, James L.
AU - Petkovska, Iva
AU - Wu, Abraham J.
AU - Reyngold, Marsha
AU - Vakiani, Efsevia
AU - Shia, Jinru
AU - Segal, Neil H.
AU - Smith, James D.
AU - Crane, Christopher
AU - Gollub, Marc J.
AU - Gonen, Mithat
AU - Saltz, Leonard B.
AU - Garcia-Aguilar, Julio
AU - Paty, Philip B.
N1 - Publisher Copyright:
© 2019 American Medical Association. All rights reserved.
PY - 2019/4
Y1 - 2019/4
N2 - Importance: The watch-and-wait (WW) strategy aims to spare patients with rectal cancer unnecessary resection. Objective: To analyze the outcomes of WW among patients with rectal cancer who had a clinical complete response to neoadjuvant therapy. Design, Setting, and Participants: This retrospective case series analysis conducted at a comprehensive cancer center in New York included patients who received a diagnosis of rectal adenocarcinoma between January 1, 2006, and January 31, 2015. The median follow-up was 43 months. Data analyses were conducted from June 1, 2016, to October 1, 2018. Exposures: Patients had a clinical complete response after completing neoadjuvant therapy and agreed to a WW strategy of active surveillance and possible salvage surgery (n = 113), or patients underwent total mesorectal excision and were found to have a pathologic complete response (pCR) at resection (n = 136). Main Outcomes and Measures: Kaplan-Meier estimates were used for analyses of local regrowth and 5-year rates of overall survival, disease-free survival, and disease-specific survival. Results: Compared with the 136 patients in the pCR group, the 113 patients in the WW group were older (median [range], 67.2 [32.1-90.9] vs 57.3 [25.0-87.9] years, P <.001) with cancers closer to the anal verge (median [range] height from anal verge, 5.5 [0.0-15.0] vs 7.0 [0.0-13.0] cm). All 22 local regrowths in the WW group were detected on routine surveillance and treated by salvage surgery (20 total mesorectal excisions plus 2 transanal excisions). Pelvic control after salvage surgery was maintained in 20 of 22 patients (91%). No pelvic recurrences occurred in the pCR group. Rectal preservation was achieved in 93 of 113 patients (82%) in the WW group (91 patients with no local regrowths plus 2 patients with local regrowths salvaged with transanal excision). At 5 years, overall survival was 73% (95% CI, 60%-89%) in the WW group and 94% (95% CI, 90%-99%) in the pCR group; disease-free survival was 75% (95% CI, 62%-90%) in the WW group and 92% (95% CI, 87%-98%) in the pCR group; and disease-specific survival was 90% (95% CI, 81%-99%) in the WW group and 98% (95% CI, 95%-100%) in the pCR group. A higher rate of distant metastasis was observed among patients in the WW group who had local regrowth vs those who did not have local regrowth (36% vs 1%, P <.001). Conclusions and Relevance: A WW strategy for select rectal cancer patients who had a clinical complete response after neoadjuvant therapy resulted in excellent rectal preservation and pelvic tumor control; however, in the WW group, worse survival was noted along with a higher incidence of distant progression in patients with local regrowth vs those without local regrowth.
AB - Importance: The watch-and-wait (WW) strategy aims to spare patients with rectal cancer unnecessary resection. Objective: To analyze the outcomes of WW among patients with rectal cancer who had a clinical complete response to neoadjuvant therapy. Design, Setting, and Participants: This retrospective case series analysis conducted at a comprehensive cancer center in New York included patients who received a diagnosis of rectal adenocarcinoma between January 1, 2006, and January 31, 2015. The median follow-up was 43 months. Data analyses were conducted from June 1, 2016, to October 1, 2018. Exposures: Patients had a clinical complete response after completing neoadjuvant therapy and agreed to a WW strategy of active surveillance and possible salvage surgery (n = 113), or patients underwent total mesorectal excision and were found to have a pathologic complete response (pCR) at resection (n = 136). Main Outcomes and Measures: Kaplan-Meier estimates were used for analyses of local regrowth and 5-year rates of overall survival, disease-free survival, and disease-specific survival. Results: Compared with the 136 patients in the pCR group, the 113 patients in the WW group were older (median [range], 67.2 [32.1-90.9] vs 57.3 [25.0-87.9] years, P <.001) with cancers closer to the anal verge (median [range] height from anal verge, 5.5 [0.0-15.0] vs 7.0 [0.0-13.0] cm). All 22 local regrowths in the WW group were detected on routine surveillance and treated by salvage surgery (20 total mesorectal excisions plus 2 transanal excisions). Pelvic control after salvage surgery was maintained in 20 of 22 patients (91%). No pelvic recurrences occurred in the pCR group. Rectal preservation was achieved in 93 of 113 patients (82%) in the WW group (91 patients with no local regrowths plus 2 patients with local regrowths salvaged with transanal excision). At 5 years, overall survival was 73% (95% CI, 60%-89%) in the WW group and 94% (95% CI, 90%-99%) in the pCR group; disease-free survival was 75% (95% CI, 62%-90%) in the WW group and 92% (95% CI, 87%-98%) in the pCR group; and disease-specific survival was 90% (95% CI, 81%-99%) in the WW group and 98% (95% CI, 95%-100%) in the pCR group. A higher rate of distant metastasis was observed among patients in the WW group who had local regrowth vs those who did not have local regrowth (36% vs 1%, P <.001). Conclusions and Relevance: A WW strategy for select rectal cancer patients who had a clinical complete response after neoadjuvant therapy resulted in excellent rectal preservation and pelvic tumor control; however, in the WW group, worse survival was noted along with a higher incidence of distant progression in patients with local regrowth vs those without local regrowth.
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U2 - 10.1001/jamaoncol.2018.5896
DO - 10.1001/jamaoncol.2018.5896
M3 - Article
C2 - 30629084
AN - SCOPUS:85059965477
SN - 2374-2437
VL - 5
JO - JAMA Oncology
JF - JAMA Oncology
IS - 4
ER -