NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas.
Brian D WeissPamela L WoltersScott R PlotkinBrigitte C WidemannJames H TonsgardJaishri O BlakelyJeffrey C AllenElizabeth K SchorryBruce R KorfNathan J RobisonStewart GoldmanAlexander A VinksChie EmotoTsuyoshi FukudaCoretta T RobinsonGary R CutterLloyd J EdwardsEva DombiNancy RatnerRoger PackerMichael J FisherPublished in: Journal of clinical oncology : official journal of the American Society of Clinical Oncology (2021)
To our knowledge, this analysis represents the first characterization of the activity and pharmacokinetics of mirdametinib in patients with NF1 and PNs and is the first published response study for MAPK/ERK kinase inhibitors in adults with NF1 and PNs. Mirdametinib given at 2 mg/m2/dose (maximum dose, 4 mg) twice daily in a 3-week on/1-week off sequence resulted in a 42% partial response rate with preliminary evidence of reduction in pain.