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Systemic treatment options for advanced biliary tract carcinoma
Changqing Xie1, Nicole A McGrath2, Cecilia Monge Bonilla2
1Thoracic and Gastrointestinal Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA. changqing.xie@nih.gov.
Journal of Gastroenterology
|August 5, 2020
Summary
Treatment options for advanced biliary tract cancers (BTC) are evolving. While chemotherapy is standard, targeted therapies and immunotherapies show promise, with combinations under investigation for improved outcomes in rare BTC tumors.
Area of Science:
- Oncology
- Gastroenterology
- Genetics
Background:
- Advanced biliary tract cancers (BTC) represent a group of rare, heterogeneous tumors with a generally poor prognosis.
- Gemcitabine and cisplatin combination chemotherapy is the current first-line standard treatment for advanced BTC.
- Limited second-line treatment options exist, highlighting the need for novel therapeutic strategies.
Purpose of the Study:
- To review the current landscape of treatment options for advanced biliary tract cancers (BTC).
- To summarize systemic chemotherapy, targeted therapy, immunotherapy, and combination approaches in advanced BTC.
- To discuss emerging therapeutic strategies based on the molecular understanding of BTC.
Main Methods:
- Comprehensive literature review of current treatment options for advanced BTC.
- Analysis of established therapies, including chemotherapy, targeted agents, and immunotherapies.
- Evaluation of ongoing clinical trials exploring novel combinations and therapeutic strategies.
Main Results:
- Pembigatinib is the first approved targeted therapy for BTC harboring FGFR2 gene fusions or rearrangements.
- Pembrolizumab is approved for advanced solid tumors, including BTC, with high microsatellite instability (MSI-H) or DNA mismatch repair deficiency (dMMR).
- Combination therapies, particularly immunotherapy with other modalities, are under active investigation due to modest single-agent immunotherapy benefits.
Conclusions:
- The molecular characterization of BTC has identified actionable targets, leading to the development of novel therapies.
- Targeted therapies and immunotherapies represent significant advancements in managing advanced BTC.
- Ongoing research into combination treatments holds promise for improving therapeutic efficacy in advanced BTC.
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