Selumetinib in Children With Inoperable Plexiform Neurofibromas

Phase 2 trial demonstrates that selumetinib, an oral MEK inhibitor, produces durable tumor shrinkage in 70% of children with neurofibromatosis type 1 and inoperable plexiform neurofibromas. Treatment resulted in clinically meaningful improvements in pain, quality of life, and functional outcomes with manageable toxicity.