What question did this study set out to answer?

This research aims to evaluate the pooled efficacy of FGFR inhibitors in treating advanced cholangiocarcinoma with FGFR alterations.

April 22, 2026

Efficacy of Fibroblast Growth Factor Receptor Inhibitors (FGFRi) in Locally Advanced or Metastatic Cholangiocarcinoma With FGFR Fusions or Rearrangements

Key Points

This research aims to evaluate the pooled efficacy of FGFR inhibitors in treating advanced cholangiocarcinoma with FGFR alterations.
Systematic search of PubMed, Cochrane, and Embase databases for FGFRi efficacy studies in cholangiocarcinoma patients
Proportional meta-analysis using random-effects model
Assessment of heterogeneity via I 2 statistics.
Objective response rate of 40.4% observed in the pooled analysis
Disease control rate reported at 92.48%
Overall survival rates at 91.45% at 6 months and 70.67% at 12 months.

Abstract

Objectives: The efficacy of various FGFR inhibitors (FGFRi) has been reported for advanced cholangiocarcinoma patients harboring FGFR fusion genes or rearrangements. However, the pooled efficacy of FGFRi is still lacking. Methods: PubMed, Cochrane, and Embase databases were systematically searched from inception to Jan 2026 for studies evaluating the efficacy of FGFRi in advanced cholangiocarcinoma patients previously treated with chemotherapy. The primary outcomes were the objective response rate, disease control rate, progression-free survival, and overall survival. Secondary outcomes included safety parameters. A proportional meta-analysis was performed using a random-effects model.Heterogeniety was assessed using I 2 statistics. Results: Eight studies with 463 patients were included. In the pooled analysis, the objective response rate was 40.4% (n=171/463; 95% CI: 31.64%-49.81%), and the disease control rate was 92.48% (n=368/435; 95% CI: 81.68%-98.72%). Progression-free survival was 64.3% (n=164/255; 95% CI: 58.24%-69.96%) and 37.33% (n=84/255; 95% CI: 31.26%-43.84%) at 6 and 12 months, respectively. Overall survival rates were 91.45% (n=139/152; 95% CI: 85.83%-94.97%) and 70.67% (n=159/225; 95% CI: 64.39%-76.25%) at 6 and 12 months, respectively. The common adverse events were hyperphosphatemia (75.4%), alopecia (44.53%), stomatitis (33.20%), diarrhea (32.15%), fatigue (33.64%), dry eyes (25.20%), nail discolouration (17.38%), ALT elevation (14.53%), and AST elevation (16.93%). Conclusions: Our analysis suggests that FGFR alteration testing and the use of FGFRi as an effective alternative option in advanced cholangiocarcinoma patients previously treated with chemotherapy.

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