Pediatric PK/PD Phase I Trial of Pexidartinib in Relapsed and Refractory Leukemias and Solid Tumors Including Neurofibromatosis Type I-Related Plexiform Neurofibromas.
Lauren H BoalJohn GlodMelissa SpencerMiki KasaiJoanne DerdakEva DombiMark AhlmanDaniel W BeuryMelinda S MerchantChristianne PersenaireDavid J LiewehrSeth M SteinbergBrigitte C WidemannRosandra N KaplanPublished in: Clinical cancer research : an official journal of the American Association for Cancer Research (2020)
Pexidartinib in pediatric patients was well tolerated at all DL tested, achieved target inhibition, and resulted in a weight-based RPD2 dose.