LAUSR

Simple Summary Cholangiocarcinoma is a rare and aggressive cancer of the bile ducts. For some patients, this cancer is linked to a genetic change in the FGFR2 protein. In 2021, Health Canada approved pemigatinib as a targeted therapy for patients with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement. However, there is little real-world data on the use of pemigatinib in these patients in the Canadian setting. This study included 18 patients across six provinces who received pemigatinib through a patient support program. Most had advanced disease, and many had already received several lines of chemotherapy. After starting pemigatinib, over half showed a measurable response in their cancer, and nearly 90% had some level of disease control. On average, pemigatinib delayed disease progression for approximately one year. Importantly, none of the patients stopped treatment because of side effects. These results are comparable to those from earlier clinical trials, suggesting pemigatinib is effective and well tolerated in real-world settings. These findings reinforce the clinical value of pemigatinib for Canadian patients with cholangiocarcinoma and underscore the need for timely access to both targeted therapies and comprehensive genetic testing to ensure patients receive the most effective, personalized care.