From progression to complete remission – a case study of successful pemigatinib treatment in a patient with metastatic FGFR2+ cholangiocarcinoma

Overview

Background: Cholangiocarcinoma is a rare biliary tract cancer with a generally poor prognosis, and a 5-year survival rate of < 10%. In advanced or metastatic disease, the prognosis is even worse, with a median survival of around 11.7 months with standard chemotherapy. Effective treatment options have been limited, especially after first-line treatment failure. Pemigatinib is a selective inhibitor of FGFR1, 2, and 3, which has demonstrated significant efficacy as the first targeted therapy in patients with cholangiocarcinoma with FGFR2 gene fusions or rearrangements. Case: A patient was diagnosed with metastatic cholangiocarcinoma at the age of 45 years. Initially, he was treated with two lines of systemic chemotherapy, achieving temporary disease stabilization. Subsequently, however, the disease progressed to multiple metastatic processes in the liver. NGS testing revealed an FGFR: BICC gene fusion. After approval of drug reimbursement based on a request under § 16, treatment with pemigatinib was initiated. The first three cycles were administered from January to April 2024. The first restaging PET/CT showed complete disease regression. Treatment was well tolerated by the patient without significant adverse effects. Follow-up PET/CT in July 2024 confirmed ongoing complete remission. The patient continued pemigatinib treatment with regular approval of reimbursement for 3-month periods. The latest restaging CT in February 2025 showed persistent complete remission. The patient is in excellent clinical condition (PS 0 according to WHO), is working, and has no subjective complaints or laboratory abnormalities. Conclusion: This case demonstrates the significant therapeutic potential of pemigatinib in a patient with metastatic cholangiocarcinoma with FGFR2 fusion, where complete disease remission was achieved after previous progression on standard treatment.

Keywords:

cholangiocarcinoma – targeted therapy – pemigatinib – FGFR tyrosine kinase inhibitor