Gross AM, Wolters PL, Dombi E, et al. - Researchers performed an open-label, phase 2 trial of selumetinib to determine the objective response rate among patients with plexiform neurofibromas and to evaluate clinical benefit. From August 2015 through August 2016, 50 children (median age, 10.2 years; range, 3.5 to 17.4) were recruited. Disfigurement, motor dysfunction, and pain were the most frequent neurofibroma-related symptoms. Nausea, vomiting, or diarrhea; an asymptomatic increase in the creatine phosphokinase level; acneiform rash; and paronychia were the most frequent toxic effects. Findings suggested that most children with neurofibromatosis type 1 and inoperable plexiform neurofibromas had durable tumor shrinkage and clinical advantage from selumetinib.