The Evolution of Pediatric Neuroblastoma Management

Background: Neuroblastoma, a common pediatric malignancy, exhibits diverse clinical behavior, necessitating evolving management strategies. This review examines historical milestones, current risk-adapted protocols, and emerging therapies, with insights for resource-limited settings like India.

Methods: A systematic search (1950–March 2025) across PubMed, EMBASE, and Cochrane Library identified 40 studies (15 trials, 20 cohorts, 5 reviews) on pediatric neuroblastoma management. Data on interventions, survival, and complications were extracted, with quality assessed using standardized tools. Narrative synthesis was conducted per PRISMA guidelines.

Results: Early surgical approaches yielded poor outcomes, but chemotherapy (1960s), MYCN amplification discovery (1985), and INRG risk stratification (2009) improved survival. Low-risk cases achieve >90% overall survival (OS) with surgery, intermediate-risk 94–96% OS with chemotherapy, and high-risk cases approach 50% OS with multimodal therapy, including tandem ASCT and immunotherapy. Emerging therapies (ALK inhibitors, MIBG, CAR T-cells) show promise but face access barriers in India.

Conclusion: Neuroblastoma management has advanced significantly, but challenges like relapse, late effects, and resource disparities persist, particularly in India. Precision medicine and localized strategies are critical for future progress.