The optimal treatment approach for limited-stage small cell carcinoma of the esophagus remains uncertain. This study aimed to evaluate the efficacy and safety of preoperative chemotherapy in combination with surgery vs upfront surgery in those patients.

From June 2001 to June 2015, a total of 280 patients with limited-stage small cell carcinoma of the esophagus were screened from 60 131 patients with esophageal cancer. Outcome analysis of those patients who underwent preoperative chemotherapy in combination with surgery or upfront surgery was conducted. The primary end point was overall survival, and secondary end points included progression-free survival and safety.

Of the 280 patients, 200 were men (71.4%), the median age was 64 years (range, 42-75 years), 171 patients (61.1%) patients had preoperative chemotherapy in combination with surgery, and 109 patients (38.9%) underwent upfront surgery. A pathologic complete response rate of 8.8% was noted in patients who received preoperative chemotherapy. Compared with the upfront surgery group, the preoperative chemotherapy group had a better median overall survival (26.0 months vs 19.5 months, respectively; hazard ratio, 0.69; 95% CI, 0.51 to 0.92; P = .011) and a prolonged progression-free survival (16.0 months vs 13.0 months, respectively; hazard ratio, 0.75; 95% CI, 0.57 to 0.99; P = .039). Postoperative complications and peritreatment mortality were comparable between both groups.

Compared with upfront surgery, preoperative chemotherapy in combination with surgery improves overall survival in patients with limited-stage small cell carcinoma of the esophagus.