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Sustained response of three pediatric BRAFV600E mutated high-grade gliomas to combined BRAF and MEK inhibitor therapy.

ABSTRACT: Outcomes for children with high-grade gliomas (HGG) remain dismal despite aggressive treatment strategies. The use of targeted therapy for BRAFV600E 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 BRAFV600E mutated HGG. In this case series, we describe three pediatric patients with HGG with confirmed BRAFV600E mutation who demonstrated responses to combination therapy with dabrafenib and trametinib.

SUBMITTER: Toll SA

PROVIDER: S-EPMC6355184 | biostudies-literature | 2019 Jan

REPOSITORIES: biostudies-literature

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Sustained response of three pediatric BRAFV600E mutated high-grade gliomas to combined BRAF and MEK inhibitor therapy.

Toll Stephanie A SA Tran Hung N HN Cotter Jennifer J Judkins Alexander R AR Tamrazi Benita B Biegel Jaclyn A JA Dhall Girish G Robison Nathan J NJ Waters Kaaren K Patel Palak P Cooper Robert R Margol Ashley S AS

Oncotarget 20190111 4

Outcomes for children with high-grade gliomas (HGG) remain dismal despite aggressive treatment strategies. The use of targeted therapy for BRAFV600E 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 BRAFV600E</ ...[more]

PMID: 30728904

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