Second-Line 5-FU/Irinotecan Is Effective in Biliary Cancer

Metastatic biliary cancers are treated with first-line gemcitabine and cisplatin combined with durvalumab. FOLFOX is a second-line option with limited benefit. Next generation sequencing in patients with metastatic biliary cancers is mandatory given the advent of active agents targeting specific mutations, gene amplifications, or gene rearrangements.

Investigators report updated results from the NIFTY trial, an industry-sponsored, randomized, open-label, phase 2 trial comparing second-line treatment with 5-FU/leucovorin with or without the addition of liposome-encapsulated irinotecan in patients with biliary cancer progression on gemcitabine and cisplatin. Of 174 patients, 57% had extrahepatic or gallbladder primaries, 64% had disease progression after less than 6 months of prior gemcitabine and cisplatin treatment, and 71% had liver metastasis.

At a median follow-up of 33.2 months, median progression-free survival as assessed by masked independent central review (the primary endpoint) was 4.2 months in the irinotecan arm compared to 1.7 months in the control arm (hazard ratio, 0.61; P=0.004). Median overall survival was also longer in the irinotecan arm compared with the control arm (8.6 vs. 5.3 months; HR, 0.68; P=0.02), and the objective response rate was higher (12.5% vs. 3.5%; P=0.04).