Single-agent Gemcitabine in the Treatment of Advanced Biliary Tract Cancers: a Phase II Study

Objective: Patients with advanced biliary tract cancers have a dismal prognosis. The aim of this study was to evaluate the efficacy and safety of gemcitabine as a single agent in the treatment of patients with unresectable biliary tract cancers. Methods: From May 2002 to April 2004, 23 chemotherapy-naı̈ve patients with locally advanced or metastatic biliary tract adenocarcinomas were enrolled. The median age was 59 years (range 37–76). Fifteen patients (65.2%) had cholangiocarcinomas and eight (34.8%) had gallbladder adenocarcinomas. Patients received gemcitabine 1000 mg/m2 over 60 min once a week for 2weeks followedbyaweekoff therapy.Treatmentwasdiscontinuedwhenunacceptable toxicities occurred or there was evidence of disease progression. Results:A total of 110cyclesof chemotherapywereperformedwithamedianof four cycles (range 1–10). The median follow-up was 13.4 months. Among the 23 patients, six (26.1%) had a partial response, eight (34.8%) had stable disease and nine (39.1%) had disease progression despite treatment. The overall response rate was 26.1 % [95 % confidence interval (CI) 22.08–30.12]. The median time to disease progression was 8.1months (95%CI 3.33–12.87) and themedian overall survival was 13.1 months (95 % CI 1.64–24.56). Toxicities were generally mild and treatment was well tolerated. Of the 23 patients, one patient experienced a grade 3–4 neutropenia and one a grade 3–4 thrombocytopenia; however, no cases of febrile neutropenia or treatment-related deaths were noted. Conclusion: In this phase II trial, therapywith gemcitabinewaswell tolerated and clinically active in patients with locally advanced or metastatic biliary tract cancers. Key words: gemcitabine – biliary tract cancer – cholangiocarcinom