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

Stephanie A. Toll, Hung N. Tran, Jennifer Cotter, Alexander R. Judkins, Benita Tamrazi, Jaclyn A. Biegel, Girish Dhall, Nathan J. Robison, Kaaren Waters, Palak Patel, Robert Cooper, Ashley S. Margol

Research output: Contribution to journalArticlepeer-review

27 Scopus citations

Abstract

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.

Original languageEnglish
Pages (from-to)551-557
Number of pages7
JournalOncotarget
Volume10
Issue number4
StatePublished - Jan 1 2019

Keywords

  • BRAF mutation
  • High-grade glioma
  • Pediatrics
  • Targeted therapy

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