Successful Long-term Treatment with Modified Dose Pemigatinib in FGFR2-BICC1 Intrahepatic Cholangiocarcinoma: A Case Report

Abstract

Patients with advanced intrahepatic cholangiocarcinoma (iCCA) have a poor prognosis. Recent advances in molecular profiling offer hope by enabling targeted treatment for those with specific mutations, potentially increasing survival rates. Pemigatinib targets fibroblast growth factor receptor 2 (FGFR2) fusions or rearrangements, providing a promising avenue for treatment. Here, we report the case of a 70-year-old woman diagnosed with advanced iCCA, characterized by FGFR2-Bicaudal family RNA binding protein 1 (BICC1) fusion, who received pemigatinib therapy. Despite adverse effects including mucositis and nail changes, she continued treatment following dose adjustments for over 18 months. This report highlights the importance of dose optimization in maintaining patients on therapy and preventing treatment discontinuation. Real-world patients are often more fragile than those in clinical trials, necessitating tailored dose adjustments. However, the literature on alternative dose modifications remains limited. This case represents the longest documented successful treatment of advanced iCCA with pemigatinib in Taiwan, emphasizing the potential efficacy of pemigatinib treatment in a real-world setting.

Keywords

• cholangiocarcinoma
• fibroblast growth factor receptor 2
• pemigatinib