Biliary Tract Adenocarcinoma (Gallbladder, Bile Duct)

    Actionable Target Abnormality Prevalence Clinical Experience with Targeted Agent
    IDH1 R132 mutation 20% of IHCCA AG-120 (IDH1 inhibitor):
    • Phase I trial, for IDH1R132 mutant pts: 1/20 pt had PR, 11/20 pts had SD, with the 1 PR and 5 SDs >6 mo (1)
    FGFR2 fusions FGFR2-BICC1 and several other fusions 10%–15% of IHCCA BGJ398 (FGFR inhibitor):
    • Phase 2: RR 15%, disease control rate (ORR + SD) 75%, PFS 25 wk. Among 61 pts, most had FGFR2 alterations, including translocation (n = 48), mutation (n = 8), and amplification (n 3) (2)
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