Multicenter phase II study of gemcitabine (GEM) plus S-1 in patients (pts) with advanced biliary tract cancer (BTC)

Abstract

e15516 Background: Pts with advanced BTC have a poor prognosis and no standard palliative chemotherapy has been defined. The purpose of this prospective multicenter phase II study was to evaluate the efficacy and safety of GEM plus S-1 combination therapy in pts with advanced BTC. Methods: Pts with advanced BTC, with at least one measurable lesion were eligible for this study. Other eligibility criteria included: no previous palliative chemotherapy, ECOG performance status ≤ 2, and adequate organ function. S-1 was given orally at a dose of 80mg/m2 daily from day 1 to day 14. GEM was given intravenously at a dose of 1,000mg/m2 over 30 min on day 1 and day 15, repeated every 4 weeks. Tumor response was the primary endpoint and was assessed according to RECIST every 8 weeks. Planned sample size was 35 according to Simon's two stage method. Results: 35pts were enrolled: median age 67; M/F (22/13); ECOG performance status 0/1/2 (16/18/1). The primary tumor site was; 14 pts in gallbladders, 14 pts in intrahepatic bile ducts, 6 pts in extrahepatic bile ducts, and 1 pt in Vater of papilla. Seven pts had previous surgical resection. Complete and partial responses were achieved in 2 and 9 pts, respectively. Eighteen pts had stable disease. Overall response rate was 31.4% and overall disease control rate was 82.9%. The median TTP was 6.1 months (95%CI, 4.0–8.1 months) and the overall survival was 11.7 months (95%CI, 7.3- months). The grade 3/4 toxicities were: neutropenia (31.4%), anemia (17.1%), thrombocytopenia (2.9%), anorexia (2.9%). No treatment-related deaths occurred during the study. Conclusions: GEM plus S- 1 combination therapy showed promising efficacy and good tolerability in pts with advanced BTC. No significant financial relationships to disclose.