Oxford Academic, Neuro-Oncology Pediatrics – BRAF pub: Growth recovery in patients with BRAF-altered pediatric low-grade glioma following treatment with tovorafenib

This publication reports a pooled retrospective analysis evaluating growth patterns in children with B-Raf proto-oncogene (BRAF)-altered relapsed or refractory pediatric low-grade glioma (pLGG) treated with the RAF inhibitor tovorafenib. Across 68 patients enrolled in the FIREFLY-1 study, Pediatric Neuro-Oncology Consortium 014 (PNOC014), and the Expanded Access Program (EAP), most patients experienced slowed linear growth during treatment; however, the analysis demonstrated that growth suppression was largely reversible after discontinuation of therapy. The majority of evaluable patients showed early post-treatment growth recovery and evidence of catch-up growth, particularly among younger children, with no indication of abnormal bone maturation or premature growth plate closure. Pharmacokinetic/pharmacodynamic (PK/PD) exposure-growth modeling further supported the reversibility of growth effects and suggested that younger baseline age may be associated with faster recovery trajectories. These findings highlight the importance of long-term growth monitoring and multidisciplinary management while supporting the continued clinical use of tovorafenib in pediatric patients with relapsed or refractory pediatric low-grade glioma.