Biliary tract cancer: ESMO Clinical Practice Guideline for diagnosis, treatment and follow-up
A newer version of this guideline has been published: ESMO Clinical Practice Guideline interim update on the management of biliary tract cancer (2025)
- Study type
- Clinical guideline
- Study name
- ESMO BTC 2023
- Design
- Clinical guideline of the European Society for Medical Oncology (ESMO); levels of evidence I to V and grades of recommendation A to E; with ESMO-MCBS and ESCAT scores
- Source
- Ann Oncol · 2023
In plain words
This guideline from the European Society for Medical Oncology (ESMO) explains to doctors how to diagnose and treat bile duct cancer. Key points for patients: before starting drug treatment, a biopsy and genetic testing of the tumour are very important, because some gene changes (such as FGFR2, IDH1 or BRAF) can be treated with targeted drugs; after surgery, capecitabine tablets are considered to reduce the risk of the cancer coming back; and in advanced disease, chemotherapy together with the immunotherapy drug durvalumab is a first-line option.
Key guideline recommendations
- III, A
Before any non-surgical treatment, a core biopsy should be obtained for pathological diagnosis and molecular profiling.
- I, A
In advanced disease suitable for systemic therapy, molecular analysis is recommended.
- III, A
MRI is the reference test for assessing the local extent of perihilar and distal cholangiocarcinoma and for detecting liver metastases; CT of the chest, abdomen and pelvis is the reference test for assessing nodal spread and metastases.
- II, A
Adjuvant chemotherapy with capecitabine should be considered for patients with cholangiocarcinoma or gallbladder cancer after resection.
- III, D
Liver transplantation is not considered standard treatment for perihilar cholangiocarcinoma, and participation in clinical trials should be encouraged.
- I, A
Cisplatin-gemcitabine is recommended as the standard first-line treatment for patients with performance status 0 to 1.
- I, A
Cisplatin-gemcitabine combined with durvalumab should be considered in the first-line treatment of biliary tract cancer (ESMO-MCBS v1.1: score 4; at the time of writing: FDA approved, not EMA approved).
- I, A
FOLFOX is the standard second-line treatment after cisplatin-gemcitabine (ESMO-MCBS v1.1: score 1).
- I, A
Ivosidenib is recommended for patients with IDH1-mutated cholangiocarcinoma who have progressed after at least one line of systemic therapy (ESMO-MCBS: 2; ESCAT: I-A).
- III, A
FGFR inhibitors are recommended for patients with FGFR2 fusions who have progressed after at least one line of systemic therapy (ESMO-MCBS: 3; ESCAT: I-B).
- III, A
Pembrolizumab is recommended in patients with MSI-H/dMMR disease who have progressed on or are intolerant of prior therapy (ESCAT: I-C); dabrafenib–trametinib is recommended for BRAF V600E mutations after at least one line of therapy [I, A; ESCAT: I-B].
Study population
Patients with biliary tract cancer, including intrahepatic, perihilar and distal cholangiocarcinoma and gallbladder cancer.
Clinical significance and place in guidelines
This guideline replaces the 2016 ESMO version and is the first ESMO guideline to include cisplatin-gemcitabine with durvalumab (based on TOPAZ-1) in first line and to require molecular profiling in advanced disease. ESMO has since also published an interim update (ESMO Open 2025), which should be checked for the latest recommendations.
Limitations
Many of the surgical and locoregional recommendations rely on level III–IV evidence; drug approval status refers to the time of writing (2022), and some approvals (such as EMA approval of durvalumab) have changed since. The recommendations were translated from the pre-print version (Journal Pre-proof) of the paper.
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.