The use of selumetinib as an effective non-surgical treatment option for pediatric orbital plexiform neurofibroma

Abstract

Orbital plexiform neurofibromas that occur in patients with neurofibromatsosis Type I (NF1) can cause clinically significant proptosis and facial disfigurement that may not always be amenable to surgical intervention. Selumetinib (Koselugo, AstraZeneca) is a mitogen-activated protein kinase (MAPK) kinase (MEK) inhibitor that was recently approved by the FDA for treatment of plexiform neurofibromas in children 2 years of age and older. We report a case of clinically significant orbital plexiform neurofibroma that responded favorably to treatment with selumetinib.