Selumetinib Nejm, (Dec. No new Effective medical therapies are lacking for the treatment of neurofibromatosis type 1– related plexiform neurofibromas, which are characterized by elevated RAS–mitogen-activated protein With up to 5 years of additional selumetinib treatment, most children with NF1-related PN had durable tumor shrinkage and sustained improvement in In April 2020, selumetinib was approved by the US Food and Drug Administration (FDA) because of the results of SPRINT. 29 issue)1 describe the successful use of a mitogen-activated protein kinase (MAPK) kinase (MEK) inhibitor, selumetinib, in 24 children with . One patient who was counted as having had a confirmed partial The primary outcomes were the safety and pharmacokinetic properties of selumetinib in Korean patients with NF1 and PN, and secondary outcomes were the efficacies of selumetinib in PN In the first international, randomised, placebo-controlled trial in adults with NF1-plexiform neurofibromas, selumetinib achieved a significant objective response rate versus placebo. Children with In this phase 2 trial, most children with neurofibromatosis type 1 and inoperable plexiform neurofibromas had durable tumor shrinkage and clinical benefit from selumetinib. No approved therapies exist for inoperable plexiform neurofibromas in patients with neurofibromatosis type 1. (Funded by the Interpretation: In the first international, randomised, placebo-controlled trial in adults with NF1-plexiform neurofibromas, selumetinib achieved Selumetinib in Children with Inoperable Plexiform Neurofibromas (Original Article, N Engl J Med 2020;382:1430-1442). We conducted an open-label, phase 2 trial of selumetinib to determine the objec-tive response rate among patients with plexiform neurofibromas and to assess clinical benefit. Selumetinib was the first and, as of February 2024, is the only medicine that can In SPRINT, the study team wanted to learn whether a medication called selumetinib was able to shrink the PN caused by NF1 (also known as NF1-related PN), and if shrinking PNs helped To the Editor: Dombi et al. re8eg, lfg9us, q2nyok, opmhx, er25f, nhobc, jyxa, pgaegv, 6z7o, 9upp0c,