Randomized phase III study of gemcitabine, cisplatin plus S-1 (GCS) versus gemcitabine, cisplatin (GC) for advanced biliary tract cancer (KHBO1401-MITSUBA)

Abstract

Methods: Eligibility criteria included chemotherapy-naïve patients with advanced biliary tract adenocarcinoma (gallbladder, intrahepatic/ extrahepatic biliary tract, or ampulla of Vater), PS of 0–2, and adequate organ function. Enrolled patients were randomly allocated to either GCS arm or GC arm. In the GCS arm, gemcitabine and cisplatin were administered intravenously at doses of 1,000 or 25 mg/m2, respectively, on day 1, and oral S-1 were administered daily at a dose of 80 mg/m2 on days 1-7 every 2 weeks. In the GC arm, 1000 mg/m2 of gemcitabine and 25 mg/m2 of cisplatin were infused on days 1 and 8 and repeated every 3 weeks. The primary endpoint was overall survival (OS) and the secondary endpoints were progression-free survival (PFS), response rate (RR), and adverse events (AEs). The sample size was calculated to be 240, assumed 1 year OS rate of 43 % in GC and of 55 % in GCS (one-sided α 0.05, and power 0.8). A total of 39 institutions in Japan participated in this study.