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.