小児低悪性度神経膠腫の現状：BRAFおよびMEK阻害に焦点を当てる

Pediatric low-grade gliomas (pLGGs) are the most common childhood central nervous system tumors and are frequently driven by alterations in the RAS/mitogen-activated protein kinase (RAS/MAPK) signaling pathway. Advances in molecular profiling have revealed key genetic drivers, including BRAF mutations, BRAF fusions, and other kinase gene rearrangements, enabling the development of genotype-guided targeted therapies. First-generation BRAF inhibitors and mitogen-activated protein kinase kinase (MEK) inhibitors have demonstrated significant clinical benefit in molecularly selected patient subgroups, prompting FDA approvals and a paradigm shift away from traditional chemotherapy. However, challenges such as resistance mechanisms, treatment durability, and long-term toxicities persist. This review summarizes the molecular landscape of pLGG, highlights current and emerging targeted therapies, and discusses unresolved issues including optimal treatment duration, toxicity management, and future directions for individualized care.