Abstract

•This report describes a patient with recurrent, metastatic anal squamous cell carcinoma (ASCC) who failed multiple lines of therapy followed by an impressive response to pemigatinib, an oral inhibitor of FGFR1, 2, and 3. •This case demonstrates how mutational analysis in the setting of advanced disease may elucidate actionable mutations when standard therapies have been exhausted. •Targeted FGFR inhibition may provide benefit to patients with advanced, refractory, FGFR-aberrant ASCC. Randomized trials would be helpful to better describe efficacy and durability of response. •This report describes a patient with recurrent, metastatic anal squamous cell carcinoma (ASCC) who failed multiple lines of therapy followed by an impressive response to pemigatinib, an oral inhibitor of FGFR1, 2, and 3. •This case demonstrates how mutational analysis in the setting of advanced disease may elucidate actionable mutations when standard therapies have been exhausted. •Targeted FGFR inhibition may provide benefit to patients with advanced, refractory, FGFR-aberrant ASCC. Randomized trials would be helpful to better describe efficacy and durability of response.

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