PMID- 34066684 OWN - NLM STAT- MEDLINE DCOM- 20210603 LR - 20230920 IS - 1648-9144 (Electronic) IS - 1010-660X (Print) IS - 1010-660X (Linking) VI - 57 IP - 5 DP - 2021 May 8 TI - Targeted Therapies in Advanced Cholangiocarcinoma: A Focus on FGFR Inhibitors. LID - 10.3390/medicina57050458 [doi] LID - 458 AB - Despite advanced diseases continuing to be associated with grim prognoses, the past decade has witnessed the advent of several novel treatment options for cholangiocarcinoma (CCA) patients. In fact, CCA has emerged as a heterogeneous group of malignancies harboring potentially druggable mutations in approximately 50% of cases, and thus, molecularly targeted therapies have been actively explored in this setting. Among these, fibroblast growth factor receptor (FGFR) inhibitors have reported important results, as witnessed by the FDA approval of pemigatinib in previously treated metastatic CCA patients harboring FGFR2 fusion or other rearrangements. Herein, we provide an overview of available evidence on FGFR inhibitors in CCA, especially focusing on the development, pitfalls and challenges of emerging treatments in this setting. FAU - Rizzo, Alessandro AU - Rizzo A AUID- ORCID: 0000-0002-5257-8678 AD - Department of Experimental, Diagnostic and Specialty Medicine, S. Orsola-Malpighi University Hospital, 40138 Bologna, Italy. LA - eng PT - Journal Article PT - Review DEP - 20210508 PL - Switzerland TA - Medicina (Kaunas) JT - Medicina (Kaunas, Lithuania) JID - 9425208 RN - 0 (Morpholines) RN - 0 (Pyrimidines) RN - 0 (Pyrroles) RN - EC 2.7.10.1 (Receptor, Fibroblast Growth Factor, Type 2) RN - Y6BX7BL23K (pemigatinib) SB - IM MH - *Bile Duct Neoplasms/drug therapy/genetics MH - Bile Ducts, Intrahepatic MH - *Cholangiocarcinoma/drug therapy/genetics MH - Humans MH - Morpholines MH - Pyrimidines MH - Pyrroles MH - Receptor, Fibroblast Growth Factor, Type 2/genetics PMC - PMC8151905 OTO - NOTNLM OT - FGFR OT - cholangiocarcinoma OT - intrahepatic cholangiocarcinoma OT - pemigatinib OT - targeted therapies COIS- The author declares no conflict of interest. EDAT- 2021/06/03 06:00 MHDA- 2021/06/04 06:00 PMCR- 2021/05/08 CRDT- 2021/06/02 01:17 PHST- 2021/03/27 00:00 [received] PHST- 2021/04/26 00:00 [revised] PHST- 2021/05/04 00:00 [accepted] PHST- 2021/06/02 01:17 [entrez] PHST- 2021/06/03 06:00 [pubmed] PHST- 2021/06/04 06:00 [medline] PHST- 2021/05/08 00:00 [pmc-release] AID - medicina57050458 [pii] AID - medicina-57-00458 [pii] AID - 10.3390/medicina57050458 [doi] PST - epublish SO - Medicina (Kaunas). 2021 May 8;57(5):458. doi: 10.3390/medicina57050458.