Advances in targeted therapy of cholangiocarcinoma

Ann Med. 2024 Dec;56(1):2310196. doi: 10.1080/07853890.2024.2310196. Epub 2024 Feb 15.


Cholangiocarcinoma (CCA) is a malignant tumor originating in the bile duct and its branching epithelium. Due to its high heterogeneity, there are no specific clinical indications at the early stage, the diagnosis is often in advanced CCA. With surgical resection, the 5-year postoperative survival rate (long-term survival rate) is very poor. The regimen of gemcitabine combined with platinum has been used as the first-line chemotherapy for advanced patients. In recent years, targeted therapy for a variety of malignant tumors has made great progress, showing good efficacy and safety in advanced CCA. However, the current targeted therapy of CCA still has many challenges, such as adverse reactions, drug resistance, and individual differences. Therefore, the researches need to further explore the targeted therapy mechanism of CCA malignancies in depth, develop more effective and safe drugs, and accurately formulate plans based on patient characteristics to further improve patient prognosis in the future. This article reviews the recent progress of targeted therapy for CCA, aiming to provide a strategy for the research and clinical work of targeted therapy for CCA.

Keywords: Cholangiocarcinoma; targeted therapy; treatment.

Plain language summary

For these patients without surgical indications, chemotherapy and radiation therapy are the main treatment options, among which gemcitabine combined with cisplatin is the standard recognized chemotherapy regimen.With the gradual maturity of gene detection technology, the molecular pathology of CCA has gradually been revealed and precision oncology has become a promising method for the treatment of CCA.

Publication types

  • Review

MeSH terms

  • Bile Duct Neoplasms* / drug therapy
  • Bile Duct Neoplasms* / pathology
  • Bile Ducts, Intrahepatic / pathology
  • Cholangiocarcinoma* / drug therapy
  • Cholangiocarcinoma* / pathology
  • Gemcitabine
  • Humans
  • Prognosis


  • Gemcitabine

Grants and funding

The author(s) reported there is no funding associated with the work featured in this article.