Background: Biliary tract carcinoma (BTC) represents a heterogeneous group of tumors,
and the overall prognosis is poor. Limited progress has been made in systemic therapy of this disease.
Recent advances have been made in understanding the molecular biology of biliary tract tumor, development
of targeted therapeutics, and molecular profiling of tumors. Methods: The currently used systemic
chemotherapy and emerging therapeutic regimens in BTC are reviewed. An overview of the
signaling pathways in biliary tract neoplasia and the sites for targeted agents is provided. The pre-clinical and clinical data
obtained from the literature in PubMed as well as the ongoing clinical studies as displayed in www.ClinicalTrials.gov are
discussed. The potential utility of molecular profiling of tumors as an approach to personalize treatment for patients with
BTC is explored. Results: The combination of gemcitabine and cisplatin produced significant though limited benefit in
prolonging survival. This regimen has become the standard of care for advanced BTC as well as the backbone for further
development of systemic therapy. The genetic alterations involved in BTC have been explored as targets for the development
of therapeutic agents. Combination of targeted therapeutics with cytotoxic chemotherapy potentially improves the
treatment response in patients with BTC. Multiplatform analysis of biliary tract tumors provides new opportunity for personalizing
treatment of individual patients. Conclusion: Therapeutic regimens are evolving with the hope of improving the
clinical outcomes for patients with BTC. Future strategies by integration of targeted therapeutics and molecular profiling
are expected to help accomplish the goal of precision therapy in BTC.
Keywords: Biliary tract carcinoma, cholangiocarcinoma, gallbladder carcinoma, molecular profiling, personalized therapy,
precision treatment, systemic treatment, targeted therapy.
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