Work

Metastatic Low‐Grade Glioma Successfully Treated in a Pediatric Patient With BRAF A598_T599insI Mutation

Year: 2025

Type: article

Abstract: 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.

Source: Cancer Reports

DOI: 10.1002/cnr2.70309

Authors: Pierluigi Calò, Safiatou Diallo, Laetitia Lebrun, Nathalie Gilis, Marco Preziosi, Pierre Leblond

Institutions: Université Libre de Bruxelles, Queen Fabiola Children's University Hospital, Erasmus Hospital, Institut d’Hématologie et d’Oncologie Pédiatrique

Language: English

Topic: Glioma Diagnosis and Treatment

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,280