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. 2024 Sep 5;144(10):1127-1130.
doi: 10.1182/blood.2024024838.

Cerebrospinal fluid distribution and pharmacokinetics of ponatinib in Ph1+ acute lymphoblastic leukemia

Pierre Walczak  1 , Sofiane Fodil  1   2 , Nicolas Vignal  3 , Aurélie Cabannes-Hamy  4 , Nicolas Boissel  1   2 , Emmanuel Raffoux  1   2 , Jean-Michel Cayuela  5 , Lauriane Goldwirt  3 , Etienne Lengliné  1
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Free article

Cerebrospinal fluid distribution and pharmacokinetics of ponatinib in Ph1+ acute lymphoblastic leukemia

Pierre Walczak et al. Blood. .
Free article

Abstract

Tyrosine kinase inhibitors efficacy in central nervous system (CNS) disease remains uncertain. Ponatinib was studied for CNS distribution in 16 patients with Philadelphia-positive acute lymphoblastic leukemia. Cerebrospinal fluid concentrations fell below the 40 nM threshold, suggesting suboptimal CNS exposure.

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