PMID- 25678238 OWN - NLM STAT- MEDLINE DCOM- 20160404 LR - 20200923 IS - 1440-1746 (Electronic) IS - 0815-9319 (Linking) VI - 30 IP - 7 DP - 2015 Jul TI - Role of the fibroblast growth factor receptor axis in cholangiocarcinoma. PG - 1116-22 LID - 10.1111/jgh.12916 [doi] AB - Advanced cholangiocarcinoma (CCA) is a highly lethal disease with limited therapeutic options beyond cytotoxic chemotherapy. Molecular profiling of CCA has provided insights into the pathogenesis of this disease and identified potential therapeutic targets. The fibroblast growth factor receptor (FGFR) axis is important for maintaining tissue homeostasis. Aberrations in FGFR activity have been implicated in the development and progression of CCA and other malignancies, which has generated significant interest in exploring FGFR's therapeutic potential. FGFR2 fusion events are present in up to 17% of intrahepatic CCAs and appear to predict sensitivity to FGFR inhibitors even after progression on chemotherapy. These observations have led to a clinical trial evaluating FGFR inhibition in patients with CCA enriched for FGFR alterations. This review summarizes current knowledge about the role of the FGFR pathway in cholangiocarcinogenesis and ongoing work in developing FGFR-directed therapies as an antineoplastic strategy for CCA. CI - (c) 2015 Journal of Gastroenterology and Hepatology Foundation and Wiley Publishing Asia Pty Ltd. FAU - Ang, Celina AU - Ang C AD - Division of Medicine, Hematology, and Medical Oncology, Mount Sinai School of Medicine, New York, New York, USA. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't PT - Review PL - Australia TA - J Gastroenterol Hepatol JT - Journal of gastroenterology and hepatology JID - 8607909 RN - 0 (Antineoplastic Agents) RN - 0 (Phenylurea Compounds) RN - 0 (Protein Kinase Inhibitors) RN - 0 (Pyrimidines) RN - 0 (Receptors, Fibroblast Growth Factor) RN - 0 (Triazines) RN - A4055ME1VK (infigratinib) RN - DDU33B674I (brivanib) RN - EC 2.7.10.1 (FGFR2 protein, human) RN - EC 2.7.10.1 (Receptor, Fibroblast Growth Factor, Type 2) RN - OF5P57N2ZX (Alanine) SB - IM MH - Alanine/analogs & derivatives MH - Antineoplastic Agents MH - Bile Duct Neoplasms/drug therapy/*genetics MH - Cholangiocarcinoma/drug therapy/*genetics MH - Chromosome Aberrations MH - Drug Discovery MH - Gene Fusion MH - Humans MH - *Molecular Targeted Therapy MH - Phenylurea Compounds MH - Protein Kinase Inhibitors MH - Pyrimidines MH - Receptor, Fibroblast Growth Factor, Type 2/genetics MH - Receptors, Fibroblast Growth Factor/antagonists & inhibitors/*genetics/*physiology MH - Signal Transduction/genetics/physiology MH - Triazines OTO - NOTNLM OT - cholangiocarcinoma OT - fibroblast growth factor receptor OT - tyrosine kinase inhibitor EDAT- 2015/02/14 06:00 MHDA- 2016/04/05 06:00 CRDT- 2015/02/14 06:00 PHST- 2015/01/22 00:00 [accepted] PHST- 2015/02/14 06:00 [entrez] PHST- 2015/02/14 06:00 [pubmed] PHST- 2016/04/05 06:00 [medline] AID - 10.1111/jgh.12916 [doi] PST - ppublish SO - J Gastroenterol Hepatol. 2015 Jul;30(7):1116-22. doi: 10.1111/jgh.12916.