New advances in the treatment of biliary tract cancer: a narrative review
- Author:
Ilhwan KIM
1
Author Information
- Publication Type:Focused Issue of This Month
- From:Journal of the Korean Medical Association 2025;68(12):824-836
- CountryRepublic of Korea
- Language:Korean
- Abstract: Radical surgical resection for biliary tract cancers (BTCs) remains the only curative treatment option; however, most BTCs are diagnosed at advanced, unresectable, or metastatic stages, at which point palliative chemotherapy is required. Accordingly, effective systemic therapies play a central role in disease management. This review summarizes recent advances in BTC treatment.Current Concepts: Several recent studies have demonstrated improved overall survival with the introduction of new treatment options built upon the standard regimen of gemcitabine and cisplatin (Gem/Cis). Immune checkpoint inhibitors have recently demonstrated overall survival benefits across various tumor types, and in BTCs specifically, the TOPAZ-1 study combining durvalumab with Gem/Cis and the KEYNOTE-966 study combining pembrolizumab with Gem/Cis have shown survival benefits. In cases where BTCs progress because of resistance to standard Gem/Cis-based chemotherapy, oncogene-targeted therapy may be considered as a second-line or later treatment option. When actionable genomic alterations are present, such as FGFR fusion mutations, agents including infigratinib and pemigatinib may be considered. For tumors harboring IDH1 mutations, ivosidenib is approved as a second-line treatment. Furthermore, in cases of HER2 overexpression, treatment options include monoclonal antibodies or antibody–drug conjugates targeting HER2, either alone or in combination with cytotoxic chemotherapy. When targeted therapy is not feasible, cytotoxic chemotherapy remains an option, with regimens such as FOLFOX and liposomal irinotecan with 5-FU/leucovorin (NIFTY) demonstrating improvements in overall survival and progression-free survival. Nevertheless, decisions regarding second-line cytotoxic chemotherapy should be made after comprehensive consideration of the patient’s performance status and prior treatment history.Discussion and Conclusion: The establishment of integrated and personalized treatment strategies that combine immunotherapy, targeted agents, and chemotherapy will be essential for improving clinical outcomes in patients with BTCs.
