Immune checkpoint inhibitors in cholangiocarcinoma

Immunotherapy. 2023 May;15(7):541-551. doi: 10.2217/imt-2022-0288. Epub 2023 Apr 25.


Cholangiocarcinoma is an epithelial malignancy originating in the biliary tracts and frequently recurs even with surgical resection. Unresectable disease has a 5-year overall survival of less than 10%. Given this poor prognosis, additional therapies are urgently needed. Chemotherapy has been the mainstay of treatment for many years. However, with the incorporation of immunotherapy into the treatment of other malignancies, there has been a great deal of interest in immunotherapy for biliary cancers. Recently, durvalumab was approved in combination with gemcitabine and cisplatin for the treatment of unresectable cholangiocarcinoma in the first-line setting. However, predicting which patients may respond to immunotherapy remains a challenge due to the lack of a reliable biomarker.

Keywords: bile duct cancer; biliary cancer; checkpoint inhibitors; cholangiocarcinoma; immunotherapy.

Plain language summary

Cholangiocarcinoma is a type of cancer that originates in the bile ducts. Unfortunately, this tumor has a poor prognosis and frequently recurs despite surgical resection. Therefore, there is an urgent need to develop additional therapies. Immunotherapy has been integrated into the treatment of multiple types of cancers, and there is interest in integrating immunotherapy into the treatment of biliary cancers as well. Most recently, the TOPAZ-1 study led to the approval of durvalumab added to gemcitabine and cisplatin chemotherapy. There are additional ongoing studies evaluating the use of combination immunotherapies, local therapies such as radiation plus immunotherapy as well as other multimodality treatments.

Publication types

  • Review

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Bile Duct Neoplasms* / drug therapy
  • Bile Ducts, Intrahepatic / pathology
  • Cholangiocarcinoma* / drug therapy
  • Humans
  • Immune Checkpoint Inhibitors / therapeutic use
  • Neoplasm Recurrence, Local / drug therapy


  • Immune Checkpoint Inhibitors