Galli-Vareia Ilianna, Szturz Petr, Voutsadakis Ioannis A, Villard Nicolas, Tsoumakidou Georgia, Fleury Mapi, Herrera Gabriela, Fasquelle Francois, Godat Sebastien, Digklia Antonia
Department of Oncology, Centre Hospitalier Universitaire Vaudois, Lausanne 1011, Switzerland.
Algoma District Cancer Program, Sault Area Hospital, Sault Ste Marie, ON P3E 2C6, Canada.
Oncologist. 2025 May 8;30(5). doi: 10.1093/oncolo/oyae294.
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.
胆管癌（CCA）是一种有效的全身治疗方法较少的癌症。基于下一代测序（NGS）的基因组研究对该疾病分子图谱的阐明，推动了新靶向治疗方法的引入。其中一种治疗方法是一类靶向受体酪氨酸激酶成纤维细胞生长因子受体（FGFRs）家族成员的小分子。我们在此报告一名患有FGFR2突变胆管癌的患者。该患者先后接受了2种不同的FGFR抑制剂治疗，因为第一种药物导致了眼部毒性。她从两种药物治疗中均获得了临床益处。该病例说明了FGFR抑制剂对具有特定点突变的胆管癌的疗效。
