Neuroblastoma, the most common extracranial solid tumor in children, presents a formidable challenge due to its highly variable clinical behavior. While some tumors regress spontaneously, others are aggressive and resistant to intensive therapy. A new narrative review published in the World Journal of Pediatric Surgery offers a comprehensive framework that integrates diagnosis, risk classification, and multimodal treatment, aiming to refine clinical decision-making and improve outcomes.
The review, authored by specialists from the Royal Hospital for Children in Glasgow and the University of Liverpool, synthesizes current evidence on neuroblastoma. It underscores that prognosis depends not only on tumor stage but also on patient age, histology, chromosomal abnormalities, and molecular features such as MYCN amplification. Approximately 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) classifies disease based on imaging findings and image-defined risk factors, guiding pretreatment risk assessment. Molecular markers add critical prognostic information; MYCN amplification, present in about 25% of tumors and 40-50% of high-risk cases, signals 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, 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%. This supports a conservative approach when strict criteria are met, avoiding unnecessary treatment. However, unresolved questions remain, such as the optimal imaging modality for surgical planning and the survival benefit of more extensive resection. The authors advocate for standardized surgical reporting to improve comparison of outcomes across trials.
The review highlights emerging therapies, including GD2-targeting monoclonal antibodies, chimeric antigen receptor T-cell therapy, and inhibitors targeting ALK mutations. It also stresses the importance of long-term survivorship care, addressing fertility, hearing, endocrine health, cognition, emotional well-being, and secondary cancers. As survival rates improve, these considerations become integral to treatment planning and follow-up.
The authors emphasize that neuroblastoma cannot be managed with a single formula. The best treatment plan depends on integrating the child's age, tumor biology, anatomical risk, and likely response to therapy. For some infants, close observation is preferable to immediate intervention; for high-risk disease, coordinated multimodal care and careful surgical judgment are essential. Surgery should be viewed as part of the treatment pathway, not an isolated technical goal.
This risk-based framework provides a valuable reference for surgeons, oncologists, radiologists, pathologists, and multidisciplinary tumor boards. By clarifying when to observe, biopsy, operate, or intensify therapy, the review supports more consistent and personalized care. It also points toward future research directions, including the role of novel targeted therapies and the need for lifelong follow-up to ensure that survivors not only live longer but also enjoy a better quality of life.
