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This article has an addendum. Addendum in: Oncotarget. 2023; 14:502-502.

Sustained response of three pediatric BRAFV600E 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 and Ashley S. Margol

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Oncotarget. 2019; 10:551-557. https://doi.org/10.18632/oncotarget.26560

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Stephanie A. Toll1,2,*, Hung N. Tran3,*, Jennifer Cotter2,4, Alexander R. Judkins2,4, Benita Tamrazi2,5, Jaclyn A. Biegel2,4, Girish Dhall1,2, Nathan J. Robison1,2, Kaaren Waters1, Palak Patel2, Robert Cooper3,* and Ashley S. Margol1,2,*

1Division of Hematology, Oncology and Blood and Marrow Transplantation, Children’s Center for Cancer and Blood Diseases, Children’s Hospital Los Angeles, Los Angeles, CA, USA

2University of Southern California Keck School of Medicine, Los Angeles, CA, USA

3Kaiser Permanente Southern California, Los Angeles, CA, USA

4Department of Pathology and Laboratory Medicine, Children’s Hospital Los Angeles, Los Angeles, CA, USA

5Department of Radiology and Imaging, Children’s Hospital Los Angeles, Los Angeles, CA, USA

*These authors contributed equally to this work

Correspondence to:

Stephanie A. Toll, email: [email protected]

Keywords: high-grade glioma; BRAF mutation; targeted therapy; pediatrics

Received: October 27, 2018     Accepted: December 27, 2018     Published: January 11, 2019


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.

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