Assessment of a watch-and-wait strategy for rectal cancer in patients with a complete response after neoadjuvant therapy

Smith, J. J. et al. (2019) Assessment of a watch-and-wait strategy for rectal cancer in patients with a complete response after neoadjuvant therapy. JAMA Oncology, 5(4), e185896. (doi: 10.1001/jamaoncol.2018.5896) (PMID:30629084) (PMCID:PMC6459120)

Abstract

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.

• Item Type: Articles
• Status: Published
• Refereed: Yes
• Glasgow Author(s) Enlighten ID: Roxburgh, Professor Campbell
• Authors: Smith, J. J., Strombom, P., Chow, O. S., Roxburgh, C. S., Lynn, P., Eaton, A., Widmar, M., Ganesh, K., Yaeger, R., Cercek, A., Weiser, M. R., Nash, G. M., Guillem, J. G., Temple, L. K. F., Chalasani, S. B., Fuqua, J. L., Petkovska, I., Wu, A. J., Reyngold, M., Vakiani, E., Shia, J., Segal, N. H., Smith, J. D., Crane, C., Gollub, M. J., Gonen, M., Saltz, L. B., Garcia-Aguilar, J., and Paty, P. B.
• College/School: College of Medical Veterinary and Life Sciences > School of Medicine, Dentistry & Nursing
• Journal Name: JAMA Oncology
• Publisher: American Medical Association
• ISSN: 2374-2437
• ISSN (Online): 2374-2445
• Published Online: 10 January 2019