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Advances in the Diagnosis and Treatment of Neuroblastoma

^a Department of Pediatrics and ^b Robert H. Lurie Comprehensive Cancer Center, Northwestern University, The Feinberg School of Medicine, Chicago, Illinois, USA; ^c AFLAC Cancer Center, ^d Children’s Healthcare of Atlanta, and ^e Emory University, Atlanta, Georgia, USA

Correspondence: Susan L. Cohn, M.D., Children’s Memorial Hospital, Division of Hematology/Oncology, Box #30, 2300 Children’s Plaza, Chicago, Illinois 60614, USA. Telephone: 773-880-4562; Fax: 773-880-3053; e-mail: scohn{at}northwestern.edu

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.

Key Words. Neuroblastoma • Tumor biology • Retinoids • Immunotherapy • Risk-based therapy

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