A phase II FOLFOX-4 regimen as second-line treatment in advanced biliary tract cancer refractory to gemcitabine/cisplatin

OBJECTIVE: We aimed to evaluate the efficacy and safety of oxaliplatin, 5-fluorouracil (5-FU), and leucovorin (LV) (FOLFOX-4) as second-line treatment in patients with advanced biliary tract cancer (BTC) failing gemcitabine/cisplatin first-line chemotherapy. METHODS: Thirty-seven patients with advanced BTC refractory to gemcitabine/cisplatin chemotherapy were included in the study. FOLFOX-4 regimen consisted of oxaliplatin (85 mg/m(2)) as a 2-hour infusion on day 1 and 2-hour infusion of LV (200 mg/m(2)/day) followed by a 5-FU bolus (400 mg/m(2)/day) and 22-hour infusion of 5-FU (600 mg/m(2)/day) for two consecutive days every 2 weeks. The primary end point was the time to progression (TTP). RESULTS: Between January 2009 and January 2012, a total of 37 patients were enrolled. The median age was 57 years (range 32-70) and male to female ratio was 21:16. Median TTP was 3·1 months (95% CI 2·3-3·6). The objective response rate was 21·6% (eight partial responses), and disease control rate was 62·2% (15 stable disease). Grade 3-4 toxicities were observed in 37·8% of the patients with neutropenia and fatigue being the most frequent (21·6%). CONCLUSIONS: FOLFOX-4 regimen is a feasible and moderately efficacious second-line chemotherapy for advanced BTC.