Long-term safety and efficacy of selumetinib for inoperable plexiform neurofibromas in children with neurofibromatosis type 1

Selumetinib shrank inoperable symptomatic neurofibromas (PN) in children with neurofibromatosis type 1 (NF1) and provided clinical benefit for many in a published phase 1-2 study. At the data cutoff for prior publications, 65% of participants were still receiving treatment. Follow-up with up to 5 years of additional selumetinib treatment, most patients had durable tumor shrinkage and sustained improvement in pain beyond that previously reported at one year. No new safety signals were identified; however, some patients developed known selumetinib-related adverse events for the first time after several year of treatment.