Selumetinib use as targeted therapy for plexiform neurofibroma: a comprehensive review of the literature.

Neurofibromatosis type 1 is inherited in an autosomal dominant fashion. The approximate prevalence of neurofibromatosis type 1 is 1:2500 to 1:3500 regardless of ethnic group. Around 50% of neurofibromatosis type 1 patients develop plexiform neurofibroma, a lesion that has a tendency for malignant transformation. Understanding the pathophysiology of plexiform neurofibromatosis and the implication of neurofibromin signal transduction has led to the development and use of targeted therapy on this pathway. In this article, we comprehensively reviewed the recently published literature on the use of selumetinib to treat inoperable plexiform neurofibroma in neurofibromatosis type 1 patients.