Neuroblastoma, the most common extracranial solid tumor in children, presents a clinical paradox: some tumors regress spontaneously, while others are highly aggressive and resistant to therapy. A new narrative review published in the World Journal of Pediatric Surgery offers a unified framework for managing this disease, integrating risk classification, surgical principles, and multimodal treatment strategies. The review, authored by specialists from the Royal Hospital for Children in Glasgow and the University of Liverpool, underscores that outcomes hinge not only on tumor stage but also on age, histology, chromosomal aberrations, and molecular markers such as MYCN amplification.
The disease accounts for approximately 15% of pediatric cancer deaths, with five-year survival rates exceeding 90% for low- and intermediate-risk cases but dropping below 60% for high-risk disease. This disparity highlights the critical need for risk-adapted approaches. The review synthesizes current evidence on clinical presentation, imaging, pathology, and molecular biology, providing a practical roadmap for pediatric specialists. It emphasizes that treatment must balance intensity against potential toxicity and long-term quality of life.
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) utilizes imaging-defined risk factors (IDRFs) to classify disease before treatment. Molecular markers add another layer; MYCN amplification, present in roughly 25% of tumors and 40-50% of high-risk cases, signals aggressive behavior.
Treatment strategies range from observation alone in carefully selected infants to intensive multimodal therapy for high-risk disease. For low-risk patients, surgery or observation may suffice, sparing them unnecessary toxicity. High-risk patients require a combination of chemotherapy, surgery, myeloablative therapy with autologous stem cell rescue, radiotherapy, GD2-targeting monoclonal antibodies, and retinoic acid. The review cites a prospective study showing that in infants meeting strict observation criteria, 10-year event-free survival was 94.7% and overall survival 97.4%, supporting a watch-and-wait approach.
The authors address ongoing controversies, such as the role of extensive surgical resection. While CT may better delineate surgical anatomy in some high-risk abdominal tumors, the survival benefit of more aggressive resection remains debated. Standardized surgical reporting could improve future trials by more reliably distinguishing complete from incomplete resection.
“The central message is that neuroblastoma cannot be managed with a single formula,” the authors state. “The safest and most effective plan depends on seeing the child's age, tumor biology, anatomical risk, and likely treatment response as one connected picture.” They advocate for surgery as part of a coordinated treatment pathway, not an isolated technical goal.
The review also highlights emerging therapies, including GD2-targeting antibodies, chimeric antigen receptor T-cell therapy, and inhibitors of ALK mutations, pointing toward more personalized treatment. Additionally, the authors stress that survival is not the only endpoint; long-term follow-up for fertility, hearing, endocrine health, cognition, and secondary cancers is essential as more children survive.
This comprehensive framework serves as a valuable resource for surgeons, oncologists, radiologists, and pathologists, promoting consistent decisions about when to observe, biopsy, operate, or intensify therapy. Wider adoption of structured surgical reports could enhance international trial comparisons, and the discussion of molecular targets may shape future protocols.
