Neuroblastoma, the most common solid tumor outside the brain in children under five, presents a formidable challenge due to its unpredictable 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 (DOI: 10.1136/wjps-2025-001127) provides a comprehensive framework for risk-guided care, integrating diagnosis, staging, and treatment to improve outcomes and reduce unnecessary interventions.
The review, authored by specialists from the Royal Hospital for Children in Glasgow and the University of Liverpool, highlights that neuroblastoma outcomes depend on more than just tumor stage. Age, histology, chromosomal changes, and molecular features such as MYCN amplification are critical factors. With five-year survival exceeding 90% for low- and intermediate-risk disease but falling below 60% for high-risk cases, the need for a tailored approach is clear.
Approximately 70% of patients present with abdominal tumors. 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 image-defined risk factors to classify disease before treatment, guiding decisions on whether to observe, operate, or intensify therapy.
Treatment strategies vary widely based on risk. For low-risk infants, observation alone may be appropriate, with a prospective study showing 10-year event-free survival of 94.7% and overall survival of 97.4% in carefully selected cases. In contrast, high-risk disease requires multimodal therapy including chemotherapy, surgery, myeloablative therapy with autologous stem cell rescue, radiotherapy, and immunotherapy with GD2-targeting monoclonal antibodies.
The authors emphasize that surgery is just one part of the treatment pathway, not an isolated goal. They call for standardized surgical reporting to improve comparisons across clinical trials, particularly regarding the extent of resection. For high-risk abdominal tumors, CT may better define surgical anatomy than MRI, but the survival benefit of more extensive resection remains debated.
The review also points to emerging targeted therapies, such as chimeric antigen receptor T-cell therapy and drugs targeting ALK mutations, as promising avenues for personalized treatment. However, the authors stress that survival is not the only endpoint. Long-term health issues, including fertility, hearing loss, endocrine dysfunction, cognitive impairment, and secondary cancers, require lifelong follow-up for survivors.
This risk-based framework is invaluable for pediatric surgeons, oncologists, radiologists, and pathologists. By viewing the child’s age, tumor biology, anatomical risk, and treatment response as a connected picture, clinicians can make more consistent decisions about when to observe, biopsy, operate, or intensify therapy. The review, published in the World Journal of Pediatric Surgery, an open-access journal indexed in PubMed and other major databases, offers a practical roadmap for multidisciplinary teams dealing with this complex disease.
