First-line treatment of advanced biliary ducts carcinoma: A randomized phase II study evaluating 5-FU/LV Plus oxaliplatin (Folfox 4) versus 5-FU/LV (de gramont regimen)

Background/Aim: Few clinical trials are available for advanced biliary tract carcinoma (BTC). We conducted this randomized phase II clinical trial to explore efficacy and safety of 5-fluorouracil/leucovorin (5-FU/LV-de Gramont) or the same regimen plus oxaliplatin (Folfox 4) as first-line treatment of advanced BTC. Patients and Methods: Primary endpoint was overall survival (OS); secondary endpoints were progression-free survival (PFS), response and toxicity. Results: A total of 48 patients were enrolled, 23 in de Gramont arm and 25 in the Folfox arm. Disease control rate was 56.5% for de Gramont vs. 72% for Folfox. RR was 21.7% for de Gramont arm and 28% for Folfox arm (p=0.12). PFS was in favor of Folfox (5.2 vs. 2.8 months; p=0.031). OS was 7.5 and 13.0 months for de Gramont and Folfox arm respectively (p=0.0010). Toxicity was generally mild in both arms. Conclusion: Folfox 4 could be considered a valid option as first-line treatment of BTC due to its efficacy and tolerability.