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-naïve 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/m(2) over 60 min once a week for 2 weeks followed by a week off therapy. Treatment was discontinued when unacceptable toxicities occurred or there was evidence of disease progression.

RESULTS: A total of 110 cycles of chemotherapy were performed with a median of 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 8.1 months (95% CI 3.33-12.87) and the median 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, therapy with gemcitabine was well tolerated and clinically active in patients with locally advanced or metastatic biliary tract cancers.