Neuroblastoma, the most common solid tumor outside the brain in children under five, presents a unique challenge due to its highly variable behavior. While some tumors regress spontaneously, others are aggressive and resistant to therapy. A new narrative review published in the World Journal of Pediatric Surgery on January 6, 2026, synthesizes current evidence to offer a practical, risk-guided approach to diagnosis, treatment, and survivorship care.
The review, authored by specialists from the Royal Hospital for Children in Glasgow and the University of Liverpool, emphasizes that outcomes depend not only on tumor stage but also on age, histology, chromosomal changes, and molecular features such as MYCN amplification. This risk classification is crucial because five-year survival exceeds 90% for low- and intermediate-risk disease but drops below 60% for high-risk cases. The authors stress that balancing treatment intensity with surgical risk, organ preservation, and long-term quality of life is paramount.
About 70% of neuroblastoma cases present as abdominal tumors. Diagnosis typically combines urine catecholamine testing, MRI, MIBG scintigraphy, bone marrow assessment, biopsy, and genetic profiling. The International Neuroblastoma Risk Group Staging System (INRGSS) uses imaging and image-defined risk factors to classify disease before treatment, guiding decisions from observation alone in low-risk infants to aggressive multimodal therapy in high-risk patients. Molecular markers like MYCN amplification, found in about one-quarter of tumors and 40-50% of high-risk cases, signal aggressive behavior and influence treatment intensity.
One of the key messages is that not all children require immediate intervention. For carefully selected infants, a prospective study reported 10-year event-free survival of 94.7% and overall survival of 97.4% with observation alone, supporting a watchful waiting approach when strict criteria are met. Conversely, high-risk disease requires coordinated chemotherapy, surgery, myeloablative therapy, stem cell rescue, radiotherapy, immunotherapy with GD2-targeting antibodies, and retinoic acid.
The review also addresses unresolved surgical controversies, such as the role of more extensive resection and the relative merits of CT versus MRI in surgical planning. Standardized surgical reporting is highlighted as a need to improve comparisons across international trials and better delineate the extent of resection. Emerging therapies, including GD2-targeting monoclonal antibodies, chimeric antigen receptor T-cell therapy, and agents targeting ALK mutations, are discussed as avenues toward more personalized treatment.
Beyond survival, the authors emphasize the importance of long-term follow-up for survivors, as they face risks of fertility issues, hearing loss, endocrine dysfunction, cognitive deficits, and secondary cancers. This holistic view is intended to guide both clinical protocols and future trial designs, ensuring that care extends beyond initial treatment.
This review serves as an updated reference for multidisciplinary teams, including surgeons, oncologists, radiologists, and pathologists, to make more consistent, risk-based decisions. By integrating biological, anatomical, and clinical factors, it aims to optimize outcomes while minimizing unnecessary treatment and long-term harm. For more details, the full article is available at https://doi.org/10.1136/wjps-2025-001127.
