Abstract
Alterations in fibroblast growth factor receptor 2 (FGFR2) represent potential therapeutic targets in intrahepatic cholangiocarcinoma (ICC), yet they occur in only about 10% of patients. In this study, patients with advanced ICC are tested for FGFR2 alterations via pre-enrollment biopsy; those with alterations are excluded. Eligible patients receive locoregional gemcitabine combined with camrelizumab and surufatinib until disease progression or intolerable adverse events (AEs). Between July 2022 and June 2024, 23 eligible patients are enrolled. Twelve patients achieve partial response, resulting in an objective response rate of 52.2%. The median progression-free survival is 11.3 months, and the median overall survival is 20.3 months. Eighteen patients experience at least one AE, including one grade 3 event. Exploratory analysis indicates that responders show significantly higher tumor PD-L1 expression than non-responders do, with median tumor proportion scores of 8% and 2%, respectively. The study is registered at ClinicalTrials.gov (NCT05236699).