FDFT1/FGFR2 rearrangement: A newly identified anlotinib-sensitive FGFR2 variant in cholangiocarcinoma

According to previous reports,10-16% of patients with clinically advanced cholangiocarcinoma develop FGFR2 fusions or rearrangements. Treatment with FGFR2-specific inhibitors (tyrosine kinase inhibitors, TKIs) has proven effective for patients with cholangiocarcinoma. In this study, we report a case of advanced cholangiocarcinoma, in which the patient was unable to tolerate the adverse effects of standard first-line chemotherapy. Genetic testing suggested the presence of a novel variant resulting from FDFT1/FGFR2 rearrangement. Owing to poor accessibility and high price, only a limited number of patients with advanced cholangiocarcinoma have access to TKIs and precision targeted therapy in China. Anlotinib is a novel small-molecule multi-target TKI developed independently in China. It has a broad target spectrum, including FGFR, and can effectively inhibit tumor angiogenesis and tumor cell proliferation, thereby achieving an anti-tumor effect. Here, the patient was prescribed anlotinib. After treatment, the tumor size continued to shrink, and no significant adverse effects were reported. The finding suggested that anlotinib may be effective in patients with FDFT1/FGFR2 rearrangement and could serve as a novel treatment option for affected patients in future.