Trametinib therapy for children with neurofibromatosis type 1 and life‐threatening plexiform neurofibroma or treatment‐refractory low‐grade glioma

Abstract

Trametinib is an effective therapy for advanced PNF and refractory PLGG in patients with NF-1 and is well tolerated in children. Further data and clinical trials are required to assess tolerance, efficacy and durability of response, and length of treatment required in such patients.