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Original Investigation
Association of VA Surgeons
March 2018

Management of Stage I Squamous Cell Carcinoma of the Anal Canal

Author Affiliations
  • 1Michael E. DeBakey Department of Surgery, Baylor College of Medicine, Houston, Texas
  • 2Houston Veterans Affairs (VA) Center for Innovations in Quality, Effectiveness, and Safety, Michael E. DeBakey VA Medical Center, Houston, Texas
JAMA Surg. 2018;153(3):209-215. doi:10.1001/jamasurg.2017.3151
Key Points

Question  Is concurrent chemotherapy and radiotherapy required to treat patients with T1N0 (stage I) squamous cell carcinoma of the anal canal?

Findings  In this cohort study of 2243 patients with squamous cell carcinoma of the anal canal, 503 were treated with local excision with increasing proportional use over time. Relative to concurrent chemotherapy and radiotherapy, local excision alone was not associated with an increased risk of death.

Meaning  This study suggests the need for future trials to confirm the oncologic efficacy of local excision for the management of select patients with squamous cell carcinoma of the anal canal.


Importance  The incidence of squamous cell carcinoma of the anal canal (SCCAC) is increasing. Although standard management of SCCAC includes the use of concurrent chemotherapy and radiotherapy (chemoradiotherapy), data are lacking on potentially less morbid, alternative management strategies, such as local excision, among patients with node-negative T1 disease.

Objectives  To examine the use of local excision among patients with T1 SCCAC and to compare overall survival relative to those who received standard treatment with chemoradiotherapy.

Design, Setting, and Participants  This retrospective cohort study assessed 2243 patients in the National Cancer Database (2004-2012) between 18 and 80 years of age with T1N0M0 SCCAC. The association between the type of treatment received and overall risk of death was evaluated using multivariable Cox proportional hazards regression models. Data analysis was performed from June 29, 2016, to April 17, 2017.

Main Outcomes and Measures  Overall survival.

Results  Among 2243 patients with T1N0 SCCAC, 503 (22.4%) were treated with local excision alone (mean [SD] age, 54.5 [12.1] years; 240 [47.7%] male; 419 [83.3%] white) and 1740 with chemoradiotherapy (mean [SD] age, 57.0 [10.6] years; 562 [32.3%] male; 1547 [88.9%] white). Among those treated with chemoradiotherapy, 12 patients underwent a subsequent abdominoperineal resection. There was a statistically significant increase in the use of local excision during the study period (34 [17.3%] in 2004 to 68 [30.8%] in 2012; trend test, P < .001). This increase in use was observed among patients with primary tumors that measured 1 cm or smaller and greater than 1 cm to 2 cm or smaller (trend test, P < .001 for both). Overall survival at 5 years was not significantly different for the 2 management strategies (85.3% in the local excision cohort and 86.8% in the chemoradiotherapy cohort; log-rank test, P = .93). Overall risk of death was not significantly different for local excision alone relative to that for treatment with chemoradiotherapy (hazard ratio, 1.06; 95% CI, 0.78-1.44). These findings were robust when stratified by tumor size and when patients who underwent abdominoperineal resection after chemoradiotherapy were excluded.

Conclusions and Relevance  The use of local excision alone for the management of T1N0 SCCAC has significantly increased over time, with no clear decrement in overall survival. Because local excision may represent a lower-cost, less morbid treatment option for select patients with SCCAC, future studies are needed to better delineate its role and efficacy relative to the current standard of chemoradiotherapy.