Advances in the Diagnosis and Treatment of Neuroblastoma

Learning Objectives After completing this course, the reader will be able to: Appreciate the heterogeneity of neuroblastoma and identify clinical and biological prognostic factors. Explain the determinants and the clinical significance of the neuroblastoma risk stratification system. Select appropriate treatment regimens for neuroblastoma patients that are tailored according to risk stratification. Access and take the CME test online and receive one hour of AMA PRA category 1 credit at CME.TheOncologist.com Neuroblastoma, a childhood neoplasm arising from neural crest cells, is characterized by a diversity of clinical behavior ranging from spontaneous remission to rapid tumor progression and death. To a large extent, outcome can be predicted by the stage of disease and the age at diagnosis. However, the molecular events responsible for the variability in response to treatment and the rate of tumor growth remain largely unknown. Over the past decade, transformation‐linked genetic changes have been identified in neuroblastoma tumors that have contributed to the understanding of tumor predisposition, metastasis, treatment responsiveness, and prognosis. The Children's Oncology Group recently developed a Neuroblastoma Risk Stratification System that is currently in use for treatment stratification purposes, based on clinical and biologic factors that are strongly predictive of outcome. This review discusses the current risk‐based treatment approaches for children with neuroblastoma and recent advances in biologic therapy.