A comprehensive review published in the World Journal of Pediatric Surgery offers a unified clinical framework for managing neuroblastoma, one of the most unpredictable childhood cancers. The review, authored by specialists from the Royal Hospital for Children in Glasgow and the University of Liverpool, synthesizes current evidence on diagnosis, risk classification, treatment, and long-term survivorship, providing a practical roadmap for pediatric oncologists and surgeons.
Neuroblastoma is the most common solid tumor outside the brain in children under five, accounting for roughly 15% of pediatric cancer deaths. Its behavior ranges widely: some tumors regress spontaneously, while others spread aggressively and resist intensive therapy. Five-year survival exceeds 90% for low- and intermediate-risk disease but falls below 60% for high-risk cases. This variability makes treatment decisions particularly challenging, requiring a delicate balance between eradicating the disease and minimizing toxicity and long-term side effects.
The review, published on January 6, 2026 (DOI:10.1136/wjps-2025-001127), emphasizes that outcomes depend on more than just tumor stage. Age, histology, chromosomal changes, and molecular features such as MYCN amplification play critical roles in determining prognosis and guiding therapy. About 70% of patients present with abdominal disease, and 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 (IDRFs) to classify disease before treatment, while molecular markers like MYCN amplification—present in roughly one-quarter 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 multimodal therapy for high-risk disease, including chemotherapy, surgery, myeloablative therapy with autologous stem cell rescue, radiotherapy, GD2-targeting monoclonal antibodies, and retinoic acid. For carefully selected infants, observation alone has shown excellent outcomes: a prospective study reported 10-year event-free survival of 94.7% and overall survival of 97.4%, supporting a conservative approach when strict criteria are met.
The review also highlights 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, particularly where the true extent of resection is uncertain. They stress that surgery should be viewed as one component of a coordinated treatment pathway, not an isolated technical goal.
Emerging therapies, including GD2-targeting antibodies, chimeric antigen receptor T-cell therapy, and targeting of ALK mutations, point toward more personalized treatment. However, the authors emphasize that survival is not the only endpoint. As more children survive neuroblastoma, long-term follow-up is essential to address fertility, hearing, endocrine health, cognition, emotional well-being, and secondary cancers. These priorities should shape clinical protocols and future trial design.
The review serves as a valuable resource for multidisciplinary tumor boards, helping clinicians make more consistent decisions about when to observe, biopsy, operate, or intensify therapy. By integrating biological, anatomical, and molecular factors, the framework aims to improve outcomes while minimizing unnecessary harm, particularly for low-risk patients who may be overtreated and high-risk patients who require aggressive but carefully coordinated care.
