DOI: 10.1182/blood.2024024838 ISSN: 0006-4971
Cerebrospinal fluid distribution and pharmacokinetics of ponatinib in Ph1-positive acute lymphoblastic leukemia
Pierre Walczak, Sofiane Fodil, Nicolas Vignal, Aurelie Cabannes-Hamy, Nicolas Boissel, Emmanuel Raffoux, Jean-Michel Cayuela, Lauriane Goldwirt, Etienne LenglinéTyrosine kinase inhibitors have improved outcomes in Philadelphia-positive ALL, but their efficacy in CNS disease remains uncertain. Ponatinib was studied for CNS distribution in 22 samples from 16 patients. CSF concentrations fell below the 40nM threshold, suggesting suboptimal CNS exposure.