Open AccessPediatric PK/PD Phase I Trial of Pexidartinib in Relapsed and Refractory Leukemias and Solid Tumors Including Neurofibromatosis Type I–Related Plexiform Neurofibromas
Author(s) -
Lauren Hittson Boal,
John Glod,
Melissa Spencer,
Miki Kasai,
Joanne Derdak,
Eva Dombi,
Mark Ahlman,
Daniel W. Beury,
Melinda S. Merchant,
Christianne Persenaire,
David J. Liewehr,
Seth M. Steinberg,
Brigitte C. Widemann,
Rosandra N. Kaplan
Publication year - 2020
Publication title -
clinical cancer research
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 5.427
H-Index - 324
eISSN - 1557-3265
pISSN - 1078-0432
DOI - 10.1158/1078-0432.ccr-20-1696
Subject(s) - medicine , pharmacokinetics , refractory (planetary science) , population , gastroenterology , pharmacodynamics , response evaluation criteria in solid tumors , oncology , chemotherapy , phases of clinical research , physics , environmental health , astrobiology
Simultaneously targeting the tumor and tumor microenvironment may hold promise in treating children with refractory solid tumors. Pexidartinib, an oral inhibitor of tyrosine kinases including colony stimulating factor 1 receptor (CSF-1R), KIT, and FLT3, is FDA approved in adults with tenosynovial giant cell tumor. A phase I trial was conducted in pediatric and young adult patients with refractory leukemias or solid tumors including neurofibromatosis type 1-related plexiform neurofibromas.