Cholangiocarcinoma therapy reference (2026)
Reference snapshot of cholangiocarcinoma therapy across resectable and advanced disease and biomarker-defined targeted tiers.
Cholangiocarcinoma therapy in 2026 organises around stage and molecular profile.
Resectable disease: surgical resection is the only curative option; capecitabine adjuvant therapy improves outcomes in resected disease.
Advanced disease first-line: gemcitabine plus cisplatin remains the chemotherapy backbone; durvalumab and pembrolizumab have approvals in combination with gemcitabine-cisplatin based on TOPAZ-1 and KEYNOTE-966; the combination of IO plus chemotherapy is first-line standard.
Molecularly-defined targeted tier (intrahepatic cholangiocarcinoma in particular): FGFR2-fusion-positive disease has pemigatinib, infigratinib, futibatinib; IDH1-mutant disease has ivosidenib; HER2-amplified disease has trastuzumab deruxtecan and zanidatamab; BRAF V600E-mutant disease has dabrafenib plus trametinib; NTRK-fusion-positive disease has larotrectinib or entrectinib; MSI-high disease has pembrolizumab and other IO options.
Later-line and emerging: KRAS G12C-targeted programs in late-stage trials; novel FGFR2 follow-on programs; second-generation IDH inhibitors; combination strategies.
The diagnostic-pathway question matters. Comprehensive molecular profiling (DNA panel, RNA fusion testing) is the gate to the targeted-therapy tier; testing rate trajectory in newly diagnosed advanced cholangiocarcinoma defines the addressable population.
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