Incidence of Bevacizumab-Associated Toxicity in Children With Low-Grade Glioma With and Without Neurofibromatosis Type 1.
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BACKGROUND: Low-grade gliomas (LGG), both sporadic and neurofibromatosis type 1 (NF1)-associated, are the most common pediatric brain tumors. When unresectable, progressive, or symptomatic, they require treatment. Bevacizumab (BVZ), a monoclonal antibody targeting vascular endothelial growth factor (VEGF), has shown efficacy for progressive LGG, especially optic pathway gliomas. Reported toxicities include hypertension (HTN) and proteinuria. Children with NF1 are predisposed to HTN and vasculopathy, but data on BVZ safety in this group are lacking. We compared the incidence of BVZ-associated toxicity in children with LGG with and without NF1. METHODS: Retrospective study of all BVZ-treated LGG patients (2010-2023) at a single center. RESULTS: Seventeen children with LGG were treated with BVZ: 8 NF1, 82% optic glioma, 18% other. All received BVZ as second- or third-line therapy combined with chemotherapy. In the non-NF1 group, 77% patients developed grade 1-2 HTN; none received antihypertensives. Six developed grade 1 proteinuria. In the NF1 group, 62.5% developed grade 1-2 HTN, including one with aortic coarctation who had worsening of previous HTN, 2 required antihypertensive therapy (p nonsignificant HTN non-NF1 vs. NF1). Only 1 NF1 patient developed proteinuria. No patients in either group stopped BVZ prematurely for toxicity. HTN was reversible after BVZ cessation in all except the coarctation of the aorta patient. CONCLUSIONS: HTN is more common than previously reported in both NF1 and non-NF1 patients treated with BVZ for LGG, but reversible. Blood pressure should be monitored and treated appropriately. BVZ appears to be a safe and efficacious therapeutic option for young patients with NF1-related optic glioma.