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Treatment and prognosis of neuroblastoma

Authors
Jason M Shohet, MD, PhD
Jed G Nuchtern, MD, FACS, FAAP
Section Editor
Julie R Park, MD
Deputy Editor
Sadhna R Vora, MD

INTRODUCTION

The term neuroblastoma is commonly used to refer to a spectrum of neuroblastic tumors (including neuroblastomas, ganglioneuroblastomas, and ganglioneuromas) that arise from primitive sympathetic ganglion cells, and like paraganglioma and pheochromocytomas, have the capacity to synthesize and secrete catecholamines (figure 1). (See "Clinical presentation and diagnosis of pheochromocytoma" and "Paragangliomas: Epidemiology, clinical presentation, diagnosis, and histology".)

Neuroblastomas, which account for 97 percent of all neuroblastic tumors, are clinically heterogeneous, varying in location, histopathologic appearance, and biologic characteristics [1]. They are most remarkable for their broad spectrum of clinical behavior, which can range from spontaneous regression, to maturation to a benign ganglioneuroma, or aggressive disease with metastatic dissemination leading to death [2]. Clinical diversity correlates closely with numerous clinical and biological factors, although its molecular basis remains largely unknown. For example, most infants with disseminated disease have a favorable outcome after treatment with chemotherapy and surgery, while the majority of children over the age of 18 months who have advanced neuroblastoma die from progressive disease despite intensive multimodality therapy.

The treatment and prognosis of neuroblastoma will be reviewed here. The epidemiology, clinical presentation, and diagnosis of neuroblastoma are presented separately, as is a discussion of neuroblastomas arising in the olfactory epithelium. (See "Epidemiology, pathogenesis, and pathology of neuroblastoma" and "Clinical presentation, diagnosis, and staging evaluation of neuroblastoma" and "Olfactory neuroblastoma (esthesioneuroblastoma)".)

PROGNOSTIC FACTORS

Neuroblastomas are diverse in their clinical behavior. Certain factors influence the biologic behavior of these tumors and are helpful in predicting outcome; some are patient-related (eg, age at the time of diagnosis), while the majority are tumor-related (disease stage, tumor histology, molecular and cytogenetic features).

Tumor stage — The extent of metastatic spread at presentation is the most important factor in determining outcome for patients with neuroblastoma [3-5]. Although regional spread to lymph nodes attached or adjacent to the primary tumor does not significantly affect outcome, distant metastatic disease (eg, bone marrow involvement) confers a much worse prognosis.

                              

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