Central Nervous System Neuroblastoma, FOXR2-Activated: A Pooled Analysis of Published Clinical Outcomes

Central nervous system (CNS) neuroblastoma, FOXR2-activated, is a recently recognized entity in the WHO CNS5 classification, defined by activation of the FOXR2 transcription factor and unique histopathological features. This review synthesizes available literature and pooled clinical data, providing insight into demographics, treatment strategies, and survival outcomes.

A systematic search was performed in PubMed, Embase, and Scopus for cases reported till January 2025. Data extracted included demographics, clinical presentation, imaging, histopathology, treatment regimens, and survival. PRISMA methodology guided selection.

Ninety-four patients were identified across six studies. Median age was 4.2 years (range 0.8-15), with 83/94 (88%) supratentorial tumors. Gross total resection was achieved in 54/94 (57%), chemotherapy was administered in 85/94 (90%), and radiotherapy in 38/94 (40%). High-dose chemotherapy with autologous stem cell rescue was utilized in 22/94 (23%). Reported 5-year overall survival ranged from 70% to 100%, with a pooled estimate of approximately 82%.

CNS neuroblastoma, FOXR2-activated, demonstrates favorable survival outcomes in reported cohorts treated with multimodal therapy; however, these findings are derived from small retrospective series and require validation in prospective studies.