A phase II trial of gemcitabine in the treatment of advanced bile duct and periampullary carcinomas

Background: Gemcitabine is a novel nucleoside analogue with clinical anticancer activity in several malignancies. From September 1998 to April 2000, we treated patients with advanced bile duct and periampullary carcinomas with gemcitabine alone. Methods: Gemcitabine 1,000 mg/m²/day was administered in 200 ml of normal saline as a 30-min intravenous infusion on day 1 weekly for 3 weeks, followed by a 1-week rest. Results: A total of 24 consecutive patients (15 men, 9 women), with a median age of 59.5 years (range 40-72 years), were enrolled. All patients were evaluable for response: 1 patient achieved complete remission (CR); 2 patients had partial remission (PR); 8 patients remained stable (SD), and 13 patients had progressive disease (PD). The overall response rate (CR + PR) was 12.5% with a 95% confidence interval (CI) of 2.7-32.4%. The median progression-free survival (PFS) was 2.5 months (95% CI 1.6-5.5 months), and the median overall survival (OS) was 7.2 months (95% CI 3.8-8.9 months). Patients with disease control (CR + PR + SD) had better PFS and OS than those with PD. There were no treatment-related deaths. Few patients encountered grade 3/4 toxicity. Conclusion: Chemotherapy with gemcitabine demonstrated notable activity and was associated with a well-tolerable toxicity profile in patients with advanced biliary tract malignancies.