FOLFIRINOX is a safe and potentially effective option for first-line chemotherapy in patients suffering from advanced biliary tract cancer (BTC), a recent study published in the Journal of Investigational New Drugs showed.

The study assessed the safety and efficacy of FOLFIRINOX in patients with chemotherapy-naïve, advanced, or recurrent BTC. This multicenter phase 2 study, the researchers enrolled 35 patients. Of the patients, 21 cases were intrahepatic, 2 gall bladder, 2 ampulla, and 10 extrahepatic. This also included 26 unresectable and 9 recurrent diseases between June 2016 and March 2020. All patients had cytologically or histologically proven advanced or recurrent BTC with at least 1 measurable lesion.

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The study findings suggest that FOLFIRINOX is a well-tolerable and potentially effective first-line of treatment for advanced BTC, with a median progression-free survival of 7.4 months and overall survival of 14.7 months, and an objective response rate and disease control rate of 31.4% and 74.3%, respectively.

Study results also suggest 3–4 major grade adverse events in 88.6% of patients including neutropenia (54.3%), febrile neutropenia (17.1%), thrombocytopenia (8.6%), and cholangitis (8.6%), however the reported adverse events were all feasible with no treatment-related deaths. Moreover, in another study, the modified FOLFIRINOX reported lower incidence of all grade 3–4 adverse events (72.8%) compared to the current study, possibly due to dose modification and repeated use of primary prophylactic granulocyte colony stimulating factor (G-CSF). Keeping in mind the low dose intensity, the modified procedure with G-CSF support can prove to be a better alternative to the original full dose FOLFIRINOX.

Biliary tract cancer is composed of heterogenous neoplasms acquired from epithelial cells in the biliary system containing either intrahepatic or extrahepatic cholangiocarcinoma and cancers of the gallbladder and ampulla of Vater, the study team noted. BTC accounts for 3% of all the adult cancers, however its incidence rate has been reported to be increasing worldwide, mainly due to the increase in intrahepatic cholangiocarcinoma, the researchers added.

The authors mentioned few limitations in their study including the nonrandomized nature of the study, small sample size and longer time to enroll patients than expected because of the slow recruitment.

“In conclusion, FOLFIRINOX was safe and potentially effective an option for first-line chemotherapy in patients with advanced BTC, but further explorations are required to find a subset of patients and/or certain clinical scenario which might be beneficial from FOLFIRINOX,” the authors concluded.


Takahara N, Nakai Y, Isayama H, et al. A prospective multicenter phase II study of FOLFIRINOX as a first-line treatment for patients with advanced and recurrent biliary tract cancer. Invest New Drugs. Published online December 2, 2022. doi:10.1007/s10637-022-01322-7