Pierluigi Calò, Safiatou Diallo, Laetitia Lebrun, Nathalie Gilis, Marco Preziosi, Pierre Leblond
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Metastatic Low-Grade Glioma Successfully Treated in a Pediatric Patient With BRAF A598_T599insI Mutation
Background
Pediatric low-grade gliomas are common brain tumors often driven by MAPK pathway alterations, including rare BRAF mutations.
Case
This case report describes the first use of treatment combining dabrafenib and trametinib in a 10-year-old boy with pleomorphic xanthoastrocytoma harboring a BRAF A598_T599insI mutation. Surgery and chemotherapy failed, leading to metastatic progression; yet targeted therapy has achieved a sustained clinical and radiological response, lasting more than 2 years.
Conclusion
This case highlights the potential of RAF/MEK inhibitors in rare BRAF-mutated tumors and underscores the need for research to optimize treatment duration, manage side effects, and explore their role in non-canonical mutations.
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