A multicenter phase II study of gemcitabine (GEM) in patients with unresectable biliary tract cancer

Abstract

4145 Background: Biliary tract cancer is one of the most malignant tumors, progresses in a short time period, and has a bleak prognosis. Moreover, early detection is difficult, and there is no standard therapy for patients with unresectable biliary tract cancer. The primary objectives of this study were to evaluate the response rate and toxicities of GEM in patients with unresectable biliary tract cancer; secondary objectives included response duration, progression-free survival, and overall survival. Methods: Patients with histologically or cytologically confirmed biliary tract cancer and measurable lesions were enrolled. GEM 1000 mg/m2 was administered once weekly for 3 consecutive weeks followed by a week of rest. This 4-week schedule was then repeated. Results: From December 2001 to April 2004, 40 patients with a median age of 61 (range, 33–73) were enrolled; the majority of patients had a PS of 0 (60.0%), and primary lesions were extra hepatic bile duct (30.0%), gallbladder (55.0%), and ampulla of Vater (15.0%). All 40 patients were evaluable for efficacy and safety. Seven patients had partial responses for an overall response rate of 17.5% (95% CI, 7.3%–32.8%); 15 patients had stable disease; 17 had progressive disease; and 1 was not evaluable. The median response duration was 9.4 months (range, 2.6–9.4; 95% CI, 3.8–9.4); the median progression-free survival was 2.6 months (range, 0.7–13.5; 95% CI, 1.7–3.8). The median survival was 7.6 months (range, 1.6–27.1+; 95% CI, 5.4–9.3), and the 1-year survival rate was 25.0%. The most common grade 3/4 hematologic toxicities were neutropenia (30.0% of patients) and ALT elevation (15.0%). The most common grade 3/4 nonhematologic toxicities were constipation (7.5%), vomiting (7.5%), and anorexia (7.5%). For 34 patients (85.0%), GEM was received on an outpatient basis. Conclusions: GEM monotherapy had good survival benefits and manageable toxicities. The safety profile was not different between patients with biliary drainage or a stent for jaundice and those without jaundice. GEM is also likely to contribute to improvement of patients’ quality of life based on its ease of administration. No significant financial relationships to disclose.