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Cerebrospinal Fluid Distribution and Pharmacokinetics of Ponatinib in Ph1-positive Acute Lymphoblastic Leukemia

Pierre Walczak,Sofiane Fodil,Nicolas Vignal,Aurélie Cabannes-Hamy,Nicolas Boissel,Emmanuel Raffoux,Jean-Michel Cayuela,Lauriane Goldwirt,Etienne Lengliné

Blood（2024）

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Abstract
Tyrosine 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.
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Key words
Ponatinib,ALL,CNS involvement,CSF,BCR::ABL1
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