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Systemic treatment options for advanced biliary tract carcinoma

Authors
  • Xie, Changqing1
  • McGrath, Nicole A.1
  • Monge Bonilla, Cecilia1
  • Fu, Jianyang1
  • 1 Center for Cancer Research, National Cancer Institute, National Institutes of Health,
Type
Published Article
Journal
Journal of Gastroenterology
Publisher
Springer Singapore
Publication Date
Aug 03, 2020
Volume
55
Issue
10
Pages
944–957
Identifiers
DOI: 10.1007/s00535-020-01712-9
PMID: 32748173
PMCID: PMC7519922
Source
PubMed Central
Keywords
License
Unknown

Abstract

Advanced biliary tract cancers (BTC) include a diverse collection of rare and heterogenous tumors with poor prognosis. The combination of gemcitabine and cisplatin is the established first-line therapy for advanced BTC. There are no accepted standard treatments in the second line setting, though there are several ongoing clinical trials that implement chemotherapy as a therapeutic strategy. The understanding of the molecular landscape of BTC has offered hope of targeted therapies to the identified actionable genomic aberrations, such as FGFR2 gene fusions, mutations of IDH1/2, HER2, BRAC1/2 and BRAF. Pembigatinib has become the first approved targeted therapy for BTC with FGFR2 fusion or other rearrangements. Recent immunotherapy has opened new therapy avenues in BTC with pembrolizumab approved for either microsatellite instability high (MSI-H) or DNA mismatch repair deficient (dMMR) advanced solid tumors, including BTC. The combination of immunotherapy with other modalities is currently being evaluated in different clinical trials, since single agent immunotherapy appears to provide modest benefits in advanced BTC. In this review, we summarize the current status of treatment options, including systemic chemotherapy, targeted therapy, immunotherapy, and various combinations in advanced BTC.

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