Phase II trial of gemcitabine combined with 5-fluorouracil and cisplatin (GFP) chemotherapy in patients with advanced biliary tree cancers.

Objective: Advanced biliary tree cancers are often diagnosed at an advanced or metastatic stage and have poor prognoses. We reported the promising anti-tumor activity of gemcitabine/5-fluorouracil (5-FU)/cisplatin (CDDP) therapy, called 'GFP chemotherapy' in a pilot study.

Methods: Twenty-one patients with advanced or metastatic biliary tree cancers with no prior chemotherapy were enrolled in this Phase II trial. Patients were treated on 4-week cycle GFP chemotherapy consisting of gemcitabine at 1000 mg/m(2) on days 1, 8 and 15, and 5-FU at 150 mg/m(2) and CDDP at 3 mg/m(2) on days 1-5, 8-12 and 15-19. After two cycles, a 4-week outpatient treatment of gemcitabine (1000 mg/m(2)) on days 1 and 15 combined with 5-FU (500 mg/m(2)) and CDDP (7 mg/m(2)) on days 1 and 15 was commenced. Treatment was repeated until tumor progression or remission allowing curative operation, or unacceptable toxicity occurred.

Results: Of these 21 patients, no complete responses were observed, but 7 patients (33.3%) demonstrated partial responses (PRs) with an additional 12 patients (57.2%) having stable diseases, as assessed by RECIST. Three patients with PRs were treated by curative operation after GFP chemotherapy, and all of them survived with no recurrence for over 3 years. The median overall survival time was 18.8 months, and median time to progression was 13.4 months. Grade 3 side effects such as leukopenia, thrombocytopenia and anemia were found in six patients (28.6%), but no patients dropped out because of toxicity.

Conclusions: This GFP chemotherapy has promising anti-tumor activity and is well tolerated in patients with advanced biliary tree cancers.