biostudies-literatureGross AMSporeNeurofibromatosis Therapeutic Acceleration ProgramDevelopmental and Hyperactive Ras TumorNational Cancer InstituteNCI NIH HHSAstraZenecaNCI Cancer Therapy Evaluation ProgramNational Institutes of HealthCenter for Cancer Research1978-1988https://www.ebi.ac.uk/biostudies/studies/S-EPMC9629448biostudies-literatureUnknown24(11)<h4>Background</h4>Selumetinib was recently approved for the treatment of inoperable symptomatic plexiform neurofibromas (PNs) in children with neurofibromatosis type 1 (NF1). This parallel phase II study determined the response rate to selumetinib in children with NF1 PN without clinically significant morbidity.<h4>Methods</h4>Children with NF1 and inoperable PNs, which were not yet causing clinically significant morbidity but had the potential to cause symptoms, received selumetinib at 25 mg/m2 orally twice daily (1 cycle = 28 days). Volumetric magnetic resonance imaging analysis and outcome assessments, including patient-reported (PRO), observer-reported, and functional outcome measures were performed every 4 cycles for 2 years, with changes assessed over time. A confirmed partial respoNeuro-oncologySelumetinib in children with neurofibromatosis type 1 and asymptomatic inoperable plexiform neurofibroma at risk for developing tumor-related morbidity.PMC9629448U54 CA196519U54 CA196519-04Fisher MJKim AWhitcomb PWidemann BCGross AMBornhorst MKapustina ODufek APaul SMTherrien JSteinberg SMDombi EBlakeley JOVenzon DJWeiss BDSmith MADerdak JGlassberg BBaldwin ACarbonell AMartin SHeisey KWolters PLfalseSelumetinib in children with neurofibromatosis type 1 and asymptomatic inoperable plexiform neurofibroma at risk for developing tumor-related morbidity.<h4>Background</h4>Selumetinib was recently approved for the treatment of inoperable symptomatic plexiform neurofibromas (PNs) in children with neurofibromatosis type 1 (NF1). This parallel phase II study determined the response rate to selumetinib in children with NF1 PN without clinically significant morbidity.<h4>Methods</h4>Children with NF1 and inoperable PNs, which were not yet causing clinically significant morbidity but had the potential to cause symptoms, received selumetinib at 25 mg/m2 orally twice daily (1 cycle = 28 days). Volumetric magnetic resonance imaging analysis and outcome assessments, including patient-reported (PRO), observer-reported, and functional outcome measures were performed every 4 cycles for 2 years, with changes assessed over time. A confirmed partial respo2022-01-01T00:00:00Z2022 Nov2026-07-14T16:59:22.643Z2025-02-19T04:16:06.081ZS-EPMC96294483546774910.1093/neuonc/noac109