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Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials

Department of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, Italy
Author to whom correspondence should be addressed.
Medicina 2019, 55(2), 42;
Received: 15 December 2018 / Revised: 28 January 2019 / Accepted: 1 February 2019 / Published: 8 February 2019
(This article belongs to the Special Issue Liver Cancer: Molecular Mechanisms and Targeted Therapies)
PDF [1560 KB, uploaded 18 February 2019]


Cholangiocarcinoma (CCA) is a highly-aggressive malignancy arising from the biliary tree, characterized by a steady increase in incidence globally and a high mortality rate. Most CCAs are diagnosed in the advanced and metastatic phases of the disease, due to the paucity of signs and symptoms in the early stages. This fact, along with the poor results of the local and systemic therapies currently employed, is responsible for the poor outcome of CCA patients and strongly supports the need for novel therapeutic agents and strategies. In recent years, the introduction of next-generation sequencing technologies has opened new horizons for a better understanding of the genetic pathophysiology of CCA and, consequently, for the identification and evaluation of new treatments tailored to the molecular features or alterations progressively elucidated. In this review article, we describe the potential targets under investigation and the current molecular therapies employed in biliary tract cancers. In addition, we summarize the main drugs against CCA under evaluation in ongoing trials and describe the preliminary data coming from these pioneering studies. View Full-Text
Keywords: cancer; biliary tree; cholangiocarcinoma; molecular pathways; genomics; targeted therapies cancer; biliary tree; cholangiocarcinoma; molecular pathways; genomics; targeted therapies

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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).

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Simile, M.M.; Bagella, P.; Vidili, G.; Spanu, A.; Manetti, R.; Seddaiu, M.A.; Babudieri, S.; Madeddu, G.; Serra, P.A.; Altana, M.; Paliogiannis, P. Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials. Medicina 2019, 55, 42.

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