Pharmacotherapy of Plexiform Neurofibromas in Patients with Neurofibromatosis Type 1: Possible Adverse Events and Their Management

Neurofibromatosis type 1 is a genetic disorder that often leads to plexiform neurofibromas, benign tumors affecting peripheral nerves. Previously, treatment in Russia was limited to symptomatic therapy and surgery, but the introduction of selumetinib has changed this approach. Clinical trials show that 65% of children treated with selumetinib experienced a partial response, with a 20% or more reduction in tumor volume, and 56% had a long-term response without surgery. Over 200 children in Russia have received selumetinib since its registration in 2021, with 104 patients treated at the Yu.E. Veltischev Research and Clinical Institute. Common adverse events include skin rash, dry skin, hair discoloration and loss, paronychia, and elevated creatine phosphokinase. The article discusses these adverse events, preventive measures, and follow-up recommendations.