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Ref Type Journal Article
PMID (39864891)
Authors Vogel A, Ducreux M, ESMO Guidelines Committee. Electronic address: clinicalguidelines@esmo.org
Title ESMO Clinical Practice Guideline interim update on the management of biliary tract cancer.
Journal Vol Issue Date Pages Journal Short Title
ESMO open 10 1 2025 Jan 104003 ESMO Open
URL
Abstract Text • This ESMO Clinical Practice Guideline update addresses new developments in the management of biliary tract cancer. • Recommendations are given for first-line treatment with immune checkpoint inhibitors. • Key recommendations are also provided for second-line treatment with targeted therapies. • The update also covers the latest developments in molecular testing and intra-arterial therapies. • A management algorithm for early-stage, locally advanced and advanced/metastatic disease is provided.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
IDH1 mutant cholangiocarcinoma sensitive Ivosidenib Guideline Actionable Tibsovo (ivosidenib) is included in guidelines as second or later-line therapy for patients with cholangiocarcinoma harboring IDH1 mutations (PMID: 39864891; ESMO.org). detail... 39864891
FGFR2 rearrange cholangiocarcinoma sensitive Pemigatinib Guideline Actionable Pemazyre (pemigatinib) is included in guidelines as second or later-line therapy for patients with cholangiocarcinoma harboring FGFR2 fusions or rearrangements (PMID: 39864891; ESMO.org). detail... 39864891
FGFR2 fusion cholangiocarcinoma sensitive Futibatinib Guideline Actionable Lytgobi (futibatinib) is included in guidelines as second or later-line therapy for patients with cholangiocarcinoma harboring FGFR2 fusions and rearrangements (PMID: 39864891; ESMO.org). detail... 39864891
MLH1 negative biliary tract cancer sensitive Pembrolizumab Guideline Actionable Keytruda (pembrolizumab) is included in guidelines as second or late-line therapy for biliary tract cancer patients with high microsatellite instability (MSI-H) or deficient mismatch repair (dMMR, often defined by the loss of MLH1, PMS2, MSH2, or MSH6 expression by IHC) who have not received first-line immunotherapy (PMID: 39864891; ESMO.org). detail... 39864891
MSH2 negative biliary tract cancer sensitive Pembrolizumab Guideline Actionable Keytruda (pembrolizumab) is included in guidelines as second or late-line therapy for biliary tract cancer patients with high microsatellite instability (MSI-H) or deficient mismatch repair (dMMR, often defined by the loss of MLH1, PMS2, MSH2, or MSH6 expression by IHC) who have not received first-line immunotherapy (PMID: 39864891; ESMO.org). detail... 39864891
FGFR2 fusion cholangiocarcinoma sensitive Pemigatinib Guideline Actionable Pemazyre (pemigatinib) is included in guidelines as second or later-line therapy for patients with cholangiocarcinoma harboring FGFR2 fusions or rearrangements (PMID: 39864891; ESMO.org). 39864891 detail...
BRAF V600E biliary tract cancer sensitive Dabrafenib + Trametinib Guideline Actionable Tafinlar (dabrafenib) in combination with Mekinist (trametinib) is included in guidelines as second or later-line therapy for patients with biliary tract cancer harboring BRAF V600E (PMID: 39864891; ESMO.org). detail... 39864891
RET fusion biliary tract cancer sensitive Selpercatinib Guideline Actionable Retevmo (selpercatinib) is included in guidelines as second or later-line therapy for patients with biliary tract cancer harboring RET fusions (PMID: 39864891; ESMO.org). detail... 39864891
FGFR2 rearrange cholangiocarcinoma sensitive Futibatinib Guideline Actionable Lytgobi (futibatinib) is included in guidelines as second or later-line therapy for patients with cholangiocarcinoma harboring FGFR2 fusions and rearrangements (PMID: 39864891; ESMO.org). detail... 39864891
PMS2 negative biliary tract cancer sensitive Pembrolizumab Guideline Actionable Keytruda (pembrolizumab) is included in guidelines as second or late-line therapy for biliary tract cancer patients with high microsatellite instability (MSI-H) or deficient mismatch repair (dMMR, often defined by the loss of MLH1, PMS2, MSH2, or MSH6 expression by IHC) who have not received first-line immunotherapy (PMID: 39864891; ESMO.org). detail... 39864891
MSH6 negative biliary tract cancer sensitive Pembrolizumab Guideline Actionable Keytruda (pembrolizumab) is included in guidelines as second or late-line therapy for biliary tract cancer patients with high microsatellite instability (MSI-H) or deficient mismatch repair (dMMR, often defined by the loss of MLH1, PMS2, MSH2, or MSH6 expression by IHC) who have not received first-line immunotherapy (PMID: 39864891; ESMO.org). detail... 39864891