Phase II study of single-agent gemcitabine in patients with advanced biliary tract cancer.

PURPOSE: This phase II study was conducted to evaluate the efficacy and toxicity of single-agent gemcitabine in patients with advanced or metastatic biliary tract cancer. PATIENTS AND METHODS: Gemcitabine 1,000 mg/m(2) was administered as an intravenous 30-min infusion on days 1, 8, and 15 for every 28 days. RESULTS: Forty chemonaive patients with a median age of 61 (range 33-73) were enrolled, and all 40 patients were involved in efficacy and safety analyses. Seven (17.5%) achieved partial response; 15 (37.5%) had stable disease; 17 (42.5%) had progressive disease; and 1 (2.5%) was not evaluated. The median survival time was 7.6 months, and the 1-year survival rate was 25.0%. Grade 3/4 neutropenia occurred in 12 patients (30.0%), leukopenia in five patients (12.5%), and anemia in four patients (10.0%). The most common grade 3/4 nonhematologic toxicities were elevated ALT (15.0%) and elevated gamma-GTP (12.5%). One patient had grade 4 hemolytic uremic syndrome and recovered after discontinuation of gemcitabine. CONCLUSIONS: In single-agent therapy, gemcitabine demonstrated moderate efficacy with manageable toxicity in patients with advanced or metastatic biliary tract cancer. Further evaluations are warranted, including the exact impact of gemcitabine on the management of advanced or metastatic biliary tract cancer.