Neuroblastoma, the most common solid tumor outside the brain in children under five, presents a clinical paradox: some tumors regress spontaneously, while others are highly aggressive and resistant to therapy. This variability has long challenged pediatric oncologists, but a new narrative review published in the World Journal of Pediatric Surgery offers a comprehensive framework to guide risk-adapted care. The review synthesizes current evidence on diagnosis, risk classification, surgery, chemotherapy, immunotherapy, and survivorship, providing a practical roadmap for clinicians.
The disease accounts for about 15% of pediatric cancer deaths, with five-year survival exceeding 90% for low- and intermediate-risk cases but falling below 60% for high-risk cases. The authors, from the Royal Hospital for Children in Glasgow and the University of Liverpool, emphasize that outcomes depend not only on tumor stage but also on age, histology, chromosomal changes, and molecular features such as MYCN amplification. These factors determine whether a child might benefit from observation alone or requires intensive multimodal therapy.
About 70% of neuroblastomas arise in the abdomen. Diagnosis typically involves urine catecholamine testing, MRI, MIBG scintigraphy, bone marrow assessment, biopsy, and genetic profiling. The International Neuroblastoma Risk Group Staging System (INRGSS) uses imaging-defined risk factors to classify disease before treatment, while molecular markers like MYCN amplification—present in 25% of tumors and 40-50% of high-risk cases—signal aggressive behavior.
Treatment strategies range from observation or surgery alone for low-risk patients to intensive regimens including chemotherapy, surgery, myeloablative therapy, stem cell rescue, radiotherapy, and immunotherapy with GD2-targeting monoclonal antibodies and retinoic acid for high-risk disease. For carefully selected infants, observation has yielded impressive results: a prospective study reported 10-year event-free survival of 94.7% and overall survival of 97.4%.
The review also addresses unresolved controversies, such as the role of CT versus MRI in surgical planning and the survival benefit of more extensive resection. The authors advocate for standardized surgical reporting to improve comparisons across trials. They stress that neuroblastoma cannot be managed with a single formula; the safest and most effective plan depends on viewing the child's age, tumor biology, anatomical risk, and likely treatment response as one connected picture.
Emerging therapies, including ALK-targeted agents and chimeric antigen receptor T-cell therapy, hold promise for more personalized treatment. However, the authors emphasize that survival is not the only endpoint. Long-term follow-up is essential to address fertility, hearing, endocrine health, cognition, emotional well-being, and secondary cancers in survivors.
This review serves as a valuable resource for surgeons, oncologists, radiologists, pathologists, and tumor boards. By integrating risk-based decision-making, it can support more consistent choices about when to observe, biopsy, operate, or intensify therapy, ultimately improving outcomes while minimizing unnecessary harm.
