What is Neuroblastoma?
Neuroblastoma accounts for just over 7% of malignancies in patients less than 15 years of age and approximately 15% of all pediatric oncology deaths. It is the most common extracranial solid tumor in childhood and the most frequently diagnosed neoplasm during infancy. The tumor originates in cells of the sympaticoadrenal lineage of the neural crest, and can arise anywhere in the sympathetic nervous system. Most primary tumors (65%) occur within the abdomen with at least half of these arising in the adrenal medulla. Other common sites of disease include the neck, chest, and pelvis.
Presenting signs and symptoms are highly variable and dependent on site of primary tumor as well as the presence or absence of metastatic disease and/or paraneoplastic syndromes. The disease is remarkable for its broad spectrum of clinical behavior and biologic heterogeneity. Substantial improvement in outcome has been observed in subsets of patients with low- and intermediate-risk disease. In addition, recent studies have demonstrated improvement in outcome for children with a “high-risk” clinical phenotype with intensive-multi-modality treatment that includes consolidation with stem cell transplant followed by immunotherapy and retinoic acid. However, cure rates for high-risk patients remain at less than 50% emphasizing the need for need for better classifiers and treatments.
Improved understanding of normal neurodevelopment of the sympathicoadrenal system, the biology of neuroblastoma tumors, and the influence of the host on tumor pathogenesis and drug response will be important for the development of more effective, less toxic personalized treatment.