Outcomes of target therapies for pediatric BRAF V600-mutant low-grade gliomas: A systematic review and meta-analysis.
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BACKGROUND: This systematic review and meta-analysis evaluated the efficacy, safety, and available functional outcomes of targeted therapies, including BRAF and MEK inhibitors, in pediatric patients with BRAF V600-mutant low-grade gliomas (pLGGs). The review also explored their impact on clinical management, recovery, and quality of life. METHODS: The review followed PRISMA 2020 guidelines. Systematic searches were conducted across PubMed, ScienceDirect, EBSCO-hosted databases, and CENTRAL. Eligible studies included randomized controlled trials (RCTs) and retrospective studies (RS) reporting on tumor overall response rates (ORRs), progression-free survival (PFS), adverse events (AEs), and functional outcomes in patients aged 1-17 years. Risk of bias was assessed using CASP; the certainty of evidence was evaluated using the GRADE framework. Meta-analyses used a random-effects model (STATA 18.5). Funnel plots, Egger's tests, and subgroup/sensitivity analyses explored bias and heterogeneity. RESULTS: Ten studies (16 treatment arms; 4 RCTs, 6 RS were included). Pooled ORR was 42.35% (95% CI, 29.41-55.29), and the median PFS was 23.03 months (95% CI, 17.18-28.88). Grade 3/4 AEs occurred in 30.56% (95% CI, 22.60-38.53), most commonly rash, fatigue, and fever. Subgroup analyses suggested higher efficacy and lower toxicity with BRAF inhibitors or combination therapy. Functional outcomes were inconsistently reported, though some evidence suggested improvements in vision and performance status. CONCLUSIONS: Targeted therapies are effective and well tolerated in pediatric BRAF V600-mutant LGG, offering a promising alternative to chemotherapy, particularly in relapsed or progressive disease. Emerging evidence suggests potential benefit in selected molecularly defined patients; however, further prospective studies are required to define their role in first-line treatment. Standardized outcome reporting and long-term follow-up are essential to optimize care.