Long-Term Safety and Efficacy of Selumetinib in Children with Neurofibromatosis Type 1 on a Phase 1/2 Trial for Inoperable Plexiform Neurofibromas.

With up to 5 years of additional selumetinib treatment, most children with NF1-related PN had durable tumor shrinkage and sustained improvement in pain beyond that previously reported at one year. No new safety signals were identified, however ongoing monitoring for known selumetinib-related AEs is needed while treatment continues.