Selumetinib in Children with Inoperable Plexiform Neurofibromas.
Andrea M GrossPamela L WoltersEva DombiAndrea BaldwinPatricia WhitcombMichael J FisherBrian WeissAeRang KimMiriam BornhorstAmish C ShahStaci MartinMarie C RoderickDominique C PichardAmanda CarbonellScott M PaulJanet TherrienOxana KapustinaKara HeiseyD Wade ClappChi ZhangCody J PeerWilliam Douglas FiggMalcolm SmithJohn GlodJaishri O BlakeleySeth M SteinbergDavid J VenzonL Austin DoyleBrigitte C WidemannPublished in: The New England journal of medicine (2020)
In this phase 2 trial, most children with neurofibromatosis type 1 and inoperable plexiform neurofibromas had durable tumor shrinkage and clinical benefit from selumetinib. (Funded by the Intramural Research Program of the National Institutes of Health and others; ClinicalTrials.gov number, NCT01362803.).