Gross AM, Wolters PL, Dombi E, et al. - In this open-label, phase 2 trial of selumetinib, researchers determined the objective response rate among individuals with plexiform neurofibromas and evaluated clinical benefit. For this analysis, children with neurofibromatosis type 1 and symptomatic inoperable plexiform neurofibromas obtained oral selumetinib twice daily at a dose of 25 mg per square meter of body-surface area on a continuous dosing schedule (28-day cycles). In total, 50 children (median age, 10.2 years; range, 3.5 to 17.4) were recruited from August 2015 through August 2016. Disfigurement, motor dysfunction, and pain were the most frequent neurofibroma-related symptoms. The authors discovered that most children with neurofibromatosis type 1 and inoperable plexiform neurofibromas had durable tumor shrinkage and clinical advantage from selumetinib. Nausea, vomiting, or diarrhea; an asymptomatic increase in the creatine phosphokinase level; acneiform rash; and paronychia were the most frequent toxic effects.