Sustained response to trametinib in an adult patient with neurofibromatosis type 1 and high-grade glioma.

We report the case of a 25-year-old female patient with neurofibromatosis type 1 who was diagnosed with a thalamic high-grade glioma (HGG). She was initially treated with a combination of radiotherapy and concurrent temozolomide (TMZ). Following 7 months of TMZ treatment, an MRI showed tumor progression. Empiric treatment with trametinib was started, which demonstrated significant regression of the lesion. She is currently alive with stable disease 4 years post-initial diagnosis.