Rapid Response to Trametinib Combined With Chemotherapy for Infant BRAF-Fused Chiasmatic Glioma.
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Infants, less than 1 year, with chiasmatic gliomas (ICG) present a major therapeutic challenge due to large tumor size, decreased vision, rapid progression, and poor response to vincristine/carboplatin chemotherapy. The majority have a BRAF fusion, which may respond to downstream MEK inhibitors but response time is slow. There are no safety or efficacy data for MEK inhibitors in infants or in combination with chemotherapy. We report two infants with BRAF-fused ICG presenting at age 5 months with nystagmus and diencephalic syndrome. Significant tumor progression occurred after only 6 weeks on chemotherapy and showed dramatic response to the addition of trametinib.