Abstract

BRAFV600E inhibitor therapy, either alone or in combination with a MEK inhibitor, demonstrates positive responses in pediatric patients with gliomas harboring the BRAFV600E mutation. However, rare but life-threatening hypersensitivity reactions to BRAFV600E inhibitors may result in abrupt and permanent discontinuation of therapy. We report successful desensitization, tolerance, and continued response to an alternative BRAFV600E inhibitor plus the addition of a MEK inhibitor in a 7-year-old girl with bilateral optic pathway glioma who experienced a severe hypersensitivity reaction to vemurafenib. This strategy thereby avoided the systemic late effects of chemotherapy and radiation therapy.