Association of Adjuvant Therapy with Improved Survival in Ampullary Cancer: A National Cohort Study.

Background: There are limited data on the efficacy of adjuvant therapy in ampullary cancer. The aim of this study was to determine whether adjuvant therapy was associated with improved survival for patients with ampullary cancer.

Methods: From the National Cancer Database, we identified ampullary cancer patients who underwent resection between 2004 and 2013. We performed 1:1 propensity score matching, comparing patients who had postoperative observation to patients who received adjuvant chemotherapy (ACT) or adjuvant chemoradiotherapy (ACRT).

Results: We identified 4190 patients who fit our inclusion criteria; 63% had postoperative observation, 21% received ACT, and 16% underwent ACRT. In the matched cohorts, the use of ACT was associated with improved overall survival (HR = 0.82, 95% CI = 0.71 to 0.95). The median overall survival was 47.2 months for the ACT group and 35.5 months for the observation group. In a separate matched analysis, ACRT was also associated with improved survival (HR = 0.84, 95% CI = 0.72 to 0.98) as compared to observation. The median overall survival was 38.1 months for the ACRT group and 31.0 months for the observation group. The benefit was more pronounced in high-risk patients, such as ones with higher T and N categories.

Conclusions: In this retrospective study, the use of adjuvant therapy in ampullary cancer was associated with significantly improved overall survival. The benefit of adjuvant therapy for this disease should be confirmed in a more rigorous fashion via randomized controlled trials.