Visual Improvement in a Child With Periorbital Plexiform Neurofibroma Treated With Selumetinib: Case Report and Literature Review

This case report describes a 4-year-old girl with neurofibromatosis type 1 and an inoperable periorbital plexiform neurofibroma treated with selumetinib. The treatment resulted in significant tumor reduction, visual improvement, and lightening of café-au-lait macules over 8 months. The tumor volume decreased by approximately 70%, with marked resolution of proptosis and restoration of extraocular motility. Visual acuity improved from 20/100 to 20/40, and intraocular pressure stabilized without surgery. Mild adverse effects included transient elevations in creatine phosphokinase levels and mild hair loss. The case suggests selumetinib as a potential first-line treatment for similar pediatric cases, with future studies needed to assess long-term efficacy and safety.