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Neurofibromatosis type 1 (NF1) is the most common inherited neurological disorder, affecting 1 in 3000 individuals. Among the many clinical manifestations, plexiform neurofibromas occur in half of patients and result in disfigurement, pain, and neurological dysfunction.
Selumetinib, an oral selective MEK inhibitor, is the first approved agent to target symptomatic plexiform neurofibromas in children with NF1. To assess its effectiveness in this setting, investigators conducted an industry-supported, open-label, phase II study of 50 patients (aged, 3–18 years) who received selumetinib (25 mg/m2) twice daily for up to 2 years.
Results were as follows:
70% of patients had a confirmed partial response (the primary objective); 56% had a response la…