What question did this study set out to answer?

To highlight the clinical management of a pediatric patient with FOXR2-activated CNS neuroblastoma.

June 17, 2026Open Access

FOXR2-Mediated CNS Neuroblastoma in a Pediatric Patient Treated With Multimodal Therapy Including Proton Craniospinal Irradiation: A Case Report

Key Points

To highlight the clinical management of a pediatric patient with FOXR2-activated CNS neuroblastoma.
Reported on a four-year-old girl with multifocal FOXR2-altered CNS neuroblastoma.
Utilized subtotal resection, multi-agent chemotherapy per ACNS0334, autologous stem cell transplant, and proton craniospinal irradiation.
Focused on modern management strategies and toxicity reduction through proton therapy.
Successful treatment illustrated the utility of multimodal therapy in CNS neuroblastoma management.
Proton craniospinal irradiation aimed to reduce long-term treatment toxicity, particularly in young children.

Abstract

FOXR2-activated central nervous system (CNS) neuroblastoma is a rare embryonal tumor characterized by an aggressive nature, frequent leptomeningeal dissemination, and poor prognosis without intensive multimodal therapy. We report a four-year-old girl with multifocal FOXR2-altered CNS neuroblastoma treated with subtotal resection, multi-agent chemotherapy per ACNS0334, autologous stem cell transplant, and proton craniospinal irradiation (CSI) with focal boost. This case highlights modern management strategies and the rationale for proton therapy in reducing long-term treatment toxicity in very young children.

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