Abstract
BACKGROUND: Cholangiocarcinomas (CCAs) are aggressive hepatic tumors with poor prognoses. Of intrahepatic CCAs (iCCAs), ≤10% harbor fibroblast growth factor receptor-2 (FGFR2) fusions or rearrangements. We investigated the real-world use of pemigatinib, an oral FGFR1-3 inhibitor, for the treatment of patients with locally advanced/metastatic CCA in the United States of America. MATERIALS AND METHODS: This was a retrospective observational analysis of claims data from the Komodo Healthcare Map. Data were obtained for adult patients with CCA who had one or more claims for pemigatinib between 17 April 2020 and 31 May 2023. Demographics, clinical characteristics, treatments before pemigatinib initiation (baseline regimen), pemigatinib treatment patterns, adherence, health care resource utilization (HCRU), costs, and overall survival (OS) were assessed. RESULTS: Overall, 221 patients with CCA were included (median age 57 years); 90.5% had iCCA, and the population was racially diverse. The most common baseline regimen was gemcitabine + cisplatin (34.4%). Most patients (86.9%) initiated pemigatinib at the recommended dose (13.5 mg). During observation, 47.1% of patients discontinued pemigatinib; median [95% confidence interval (CI)] time to discontinuation was 7.0 (6.1-9.7) months. During 6-month follow-up, 68.4% of patients had a medication possession ratio ≥0.8. Median (95% CI) OS was 15.9 (13.4-23.2) months. HCRU mostly comprised ambulatory visits and pharmacy fills. Mean monthly health care costs per patient were $11 139 (all-cause) and $8889 (CCA-related). Treatment patterns, adherence, OS, HCRU, and costs demonstrated consistent trends across racial subgroups. CONCLUSIONS: Real-world pemigatinib treatment patterns and survival outcomes were consistent with findings from clinical trials and support continued use of pemigatinib across diverse patient groups as a key second-line treatment for CCA.