Targeting BRCA-Mutant Biliary Tract Cancer: Current Evidences and Future Perspectives.

Biliary tract cancers (BTCs) represent aggressive tumors of the bile ducts, which are often diagnosed at the advanced stages with a dismal prognosis. Among BTCs patients, breast cancer gene (BRCA 1/2) has been reported with germline or somatic mutation and the use of poly (ADP-ribose) polymerase inhibitors (PARPi) has achieved a certain effect with no obvious severe side effects. We find that the frequency and mutated type of BRCA 1/2 in advanced BTCs vary among studies. In the development of tumors, BRCA1 and BRCA2 play distinct roles with no age or gender differences. Surprisingly, some BTCs patients with germline BRCA mutation can get better therapeutic effects than those with somatic mutation, and patients who carry BRCA mutation are more likely to benefit from immunotherapy alone or in combination. Herein, we provide an overview of the targeted therapies in BRCA-mutant BTCs, with a particular focus on the differences between the germline and somatic mutations, as well as reviewing the current status and future steps. This article is protected by copyright. All rights reserved.