Chen Zhihua,Ji Xinting,Gu Shuo.Application of BRAF mutations and targeted therapy in pediatric brain gliomas: successes and challenges[J].Journal of Clinical Pediatric Surgery,,22():601-606.[doi:10.3760/cma.j.cn101785-202201049-001]
Application of BRAF mutations and targeted therapy in pediatric brain gliomas: successes and challenges
- Keywords:
- Proto-Oncogene Proteins B-raf; Mutation; Diffuse Intrinsic Pontine Glioma; Molecular Targeted Therapy; Surgical Procedures; Operative; Child
- Abstract:
- Glioma is the most common brain tumor in children and adolescents.Conventional treatments have failed to prolong its progression-free survival.For some refractory, recurrent and unresectable gliomas, 5-year survival rate was merely 10%.Studies have demonstrated that some gliomas harbor mutations in B-type rapidly accelerated fibrosarcoma (BRAF) gene, providing an opportunity for BRAF-targeted therapy and creating a research hotspot in pediatric gliomas.Several international multicenter clinical trials have indicated that monotherapy or combination therapy with BRAF and extracellular signal-regulated kinase (MEK) inhibitors could significantly extend progression-free survival.Their therapeutic effects have been widely recognized.Several BRAF inhibitors have been approved by the US Food and Drug Administration (FDA) for treating complex, refractory and recurrent gliomas.Selumetinib, as a potent inhibitor of MEK1/MEK2, could significantly delay the progression of neurofibromin 1 (NF1)-related PLGG.And it has been approved by FDA in 2020 for children with NF1-mutated neurofibromas.In May 2023, China National Medical Products Administration also approved the use of this drug for domestic market.Currently, multiple BRAF inhibitors are still going through clinical trials.This review introduced the general profiles of BRAF mutation in pediatric gliomas and summarized the clinical trials of BRAF-targeted therapy.It was intended to provide references for clinical applications and researches of pediatric neurogliomas in China.
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Memo
收稿日期:2023-02-28。
基金项目:海南省重点研发计划科技合作方向项目（ZDYF2020225）；海南省临床医学中心建设项目资助（琼卫医函〔2021〕75号）
通讯作者:顾硕,Email:gushuo007@163.com