How I treat biliary tract cancer

ESMO Open. 2022 Feb;7(1):100378. doi: 10.1016/j.esmoop.2021.100378. Epub 2022 Jan 13.


Management of biliary tract cancers (BTCs) is rapidly evolving. Curative management relies on surgical resection followed by adjuvant capecitabine for cholangiocarcinoma and gallbladder cancers. Unfortunately relapse rate remains high, and better adjuvant strategies are urgently required. A majority of patients are diagnosed with advanced disease, when chemotherapy with cisplatin and gemcitabine followed by second-line 5-FU and oxaliplatin /irinotecan is the cornerstone of treatment for most patients in the absence of targetable alterations. Targeted therapies, including therapies for tumours with fibroblast growth factor receptor-2 (FGFR-2) fusions, isocitrate dehydrogenase-1 (IDH-1) mutations, B-Raf proto-oncogene serine/threonine kinase (BRAF) V600E mutations, neurotrophic tyrosine receptor kinase (NTRK) fusions, Human epidermal growth factor-2 (HER-2) amplifications, and/or microsatellite instability are rapidly changing the treatment paradigm for many patients with advanced BTC, especially for patients with intrahepatic cholangiocarcinoma. Because of this, molecular profiling should be considered early on patients pathway to allow adequate planning of therapy. Ongoing research is likely to clarify the role of immunotherapy, liver-directed therapy, and liver transplant for BTCs in the future.

Keywords: ampullary cancer; biliary tract cancer; chemotherapy; cholangiocarcinoma; gallbladder cancer; surgery; targeted; treatment.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Bile Duct Neoplasms* / diagnosis
  • Bile Duct Neoplasms* / genetics
  • Bile Duct Neoplasms* / therapy
  • Bile Ducts, Intrahepatic / metabolism
  • Bile Ducts, Intrahepatic / pathology
  • Biliary Tract Neoplasms* / diagnosis
  • Biliary Tract Neoplasms* / genetics
  • Biliary Tract Neoplasms* / therapy
  • Cholangiocarcinoma* / diagnosis
  • Cholangiocarcinoma* / genetics
  • Cholangiocarcinoma* / therapy
  • Humans
  • Neoplasm Recurrence, Local