A phase II trial of uracil-tegafur (UFT) in patients with advanced biliary tract carcinoma

Background: Uracil-tegafur (UFT) has been reported to have broad antitumor activity in a variety of malignancies. However, its activity in biliary tract carcinoma has not been fully evaluated. The aim of this study was to evaluate the antitumor activity and toxicity of UFT in chemotherapy-naive patients with advanced biliary tract carcinoma. Methods: Nineteen patients with advanced biliary tract carcinoma that was histologically confirmed as adenocarcinoma were enrolled in this phase II trial of UFT. A dose of 360 mg/m(2)/day of UFT was administered orally if there was no evidence of tumor progression or there was unacceptable toxicity. Results: Of the 19 patients evaluable for response, one patient (5%) achieved a partial response with a duration of 2.0 months. Six patients (32%) showed no change and the remaining 12 (63%) had progressive disease. The median survival, 6-month survival rate and 1-year survival rate for all patients were 8.8 months, 52.6 and 21.1%, respectively. The chemotherapy was well tolerated, because grades 3 or 4 toxicity were not observed. Conclusion: UFT appears to have little activity as a single agent in treating patients with advanced biliary tract carcinoma. These findings do not support its use in practice, and further trials with this regimen in patients with biliary tract carcinoma are not recommended.