ESMO Clinical Practice Guideline interim update on the management of biliary tract cancer
- Study type
- Clinical guideline
- Study name
- ESMO BTC interim update 2025
- Design
- Interim update of the ESMO clinical guideline
- Source
- ESMO Open · 2025
In plain words
This update of the European guideline says that in advanced bile duct cancer, chemotherapy with gemcitabine and cisplatin together with an immunotherapy drug (pembrolizumab or durvalumab) is the standard first-line treatment, and that genetic testing of the tumour should be done from the start so that, if specific changes are found, targeted drugs can be used at later stages.
Key guideline recommendations
- I, A
Molecular testing is recommended at the start of first-line systemic therapy in patients with locally advanced, advanced or metastatic disease, particularly in those at high risk of progression or recurrence. (According to the guideline algorithm, the gene panel should include IDH1, FGFR2, BRAF, HER2, NTRK, RET, BRCA1/2 and PALB2.)
- I, A
Cisplatin–gemcitabine–durvalumab and cisplatin–gemcitabine–pembrolizumab are recommended as first-line treatment.
- III, A
Futibatinib (ESMO-MCBS v1.1: score 3) and pemigatinib (score 2) are recommended in patients with FGFR2 fusions or rearrangements who have progressed after one or more lines of systemic therapy (ESCAT: I-B).
- III, A
Trastuzumab deruxtecan should be considered in patients with HER2 overexpression and/or HER2 amplification who have progressed on or are intolerant of prior therapy (ESMO-MCBS v1.1: score 3; ESCAT: I-C; FDA approved, not EMA).
- III, A
Zanidatamab is recommended in previously treated HER2-positive patients (ESMO-MCBS v1.1: score 3; ESCAT: I-C; FDA approved, not EMA).
- III, A
Entrectinib (ESMO-MCBS: score 3), larotrectinib (score 3) and repotrectinib (not approved by EMA or FDA) are recommended in patients with NTRK fusions who have progressed on or are intolerant of prior therapy (ESCAT: I-C).
- III, A
Selpercatinib should be considered in patients with RET fusions who have progressed on or are intolerant of prior therapy (ESMO-MCBS v1.1: score 3; ESCAT: I-C).
Study population
Patients with biliary tract cancer (intrahepatic and extrahepatic cholangiocarcinoma and gallbladder cancer).
Endpoints
This article is not a trial and has no endpoints.
Safety and side effects
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Clinical significance and place in guidelines
Turns the results of KEYNOTE-966 and TOPAZ-1 into a level I, A recommendation and gives greater prominence to the role of molecular testing.
Limitations
It is an interim update and covers only the changed sections of the 2023 guideline.
This summary was prepared by the editorial team from the published paper; the original paper is the final reference. Read the original abstract on PubMed.