Efficacy of 2 different fibroblast growth factor receptor-inhibitors in a patient with extrahepatic cholangiocarcinoma harboring an FGFR2 mutation: a case report.

Cholangiocarcinoma (CCA) is a type of cancer with few effective systemic therapies. Elucidation of the molecular landscape of the disease from genomic studies based on next-generation sequencing (NGS) has contributed to the introduction of new targeted therapies. One of these treatments consists of a class of small molecules that target members of the fibroblast growth factor receptors (FGFRs) family of receptor tyrosine kinases. We report here on a patient with a cholangiocarcinoma bearing an FGFR2 mutation. The patient was treated with 2 different FGFR inhibitors, as the first caused ocular toxicity. She obtained clinical benefits from both. This case illustrates the efficacy of FGFR inhibitors on cholangiocarcinoma with specific point mutations.