{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Gross AM"],"funding":["Spore","Neurofibromatosis Therapeutic Acceleration Program","Developmental and Hyperactive Ras Tumor","National Cancer Institute","NCI NIH HHS","AstraZeneca","NCI Cancer Therapy Evaluation Program","National Institutes of Health","Center for Cancer Research"],"pagination":["1978-1988"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC9629448"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["24(11)"],"pubmed_abstract":["Background
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.Methods
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 respo"],"journal":["Neuro-oncology"],"pubmed_title":["Selumetinib in children with neurofibromatosis type 1 and asymptomatic inoperable plexiform neurofibroma at risk for developing tumor-related morbidity."],"pmcid":["PMC9629448"],"funding_grant_id":["U54 CA196519","U54 CA196519-04"],"pubmed_authors":["Fisher MJ","Kim A","Whitcomb P","Widemann BC","Gross AM","Bornhorst M","Kapustina O","Dufek A","Paul SM","Therrien J","Steinberg SM","Dombi E","Blakeley JO","Venzon DJ","Weiss BD","Smith MA","Derdak J","Glassberg B","Baldwin A","Carbonell A","Martin S","Heisey K","Wolters PL"],"additional_accession":[]},"is_claimable":false,"name":"Selumetinib in children with neurofibromatosis type 1 and asymptomatic inoperable plexiform neurofibroma at risk for developing tumor-related morbidity.","description":"Background
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.Methods
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 respo","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Nov","modification":"2026-07-14T16:59:22.643Z","creation":"2025-02-19T04:16:06.081Z"},"accession":"S-EPMC9629448","cross_references":{"pubmed":["35467749"],"doi":["10.1093/neuonc/noac109"]}}