Sustained response of three pediatric BRAF(V600E) mutated high-grade gliomas to combined BRAF and MEK inhibitor therapy

Outcomes for children with high-grade gliomas (HGG) remain dismal despite aggressive treatment strategies. The use of targeted therapy for BRAF(V600E) mutated malignancies including HGG is being explored as a potentially well tolerated and effective therapeutic option. The results of adult melanoma studies demonstrating that combination therapy with BRAF inhibitors and MEK inhibitors results in prolonged survival led us to employ this treatment strategy in children with BRAF(V600E) mutated HGG. In this case series, we describe three pediatric patients with HGG with confirmed BRAF(V600E) mutation who demonstrated responses to combination therapy with dabrafenib and trametinib.