Neuroblastoma is an embryonal cancer of the autonomic nervous system;  the most common cancer diagnosed during the first year of life. Although neuroblastoma accounts for disproportionately high morbidity and mortality among childhood cancers, it has one of the highest rates of spontaneous and complete regression. Tumours can arise anywhere along the sympathetic (autonomous) nervous system, with the majority occurring in the adrenal medulla. Primary tumours in the neck or upper chest can cause Horner’s syndrome (ptosis, miosis, and anhidrosis). Tumours along the spinal column can expend to cause cord compression, with resulting paralysis. Although many lower-stage neuroblastomas are encapsulated and can be surgically excised with little chance of complications, higher-stage tumours often infiltrate local organ structures, surround critical nerves and vessels such as the celiac axis, and are largely un-resect able at the time of diagnosis. Neuroblastomas typically metastasize to regional lymph nodes and to the bone marrow by means of the hematopoietic system. Tumour cells metastatic to marrow can infiltrate cortical bone. 

Neuroblastoma most commonly affects children age 5 or younger, though it may rarely occur in older children.

The clinical presentation is highly variable.

In most cases, neuroblastoma cells make enough catecholamines to be detected by blood or urine tests. If neuroblastoma cells are making catecholamines, the amount of Homovanillic Acid(HVA) and Vanillylmandelic Acid (VMA) in urine or blood will be higher than expected.

Neuroblastoma usually occurs sporadically, but familial cases are also observed.

Neuroblastoma have widely divergent outcomes, ranging from high chance of cure for the child with low risk disease to less than 50% for those with high risk disease. The identification of novel cell-mediated immunotherapy agents may lead to new therapeutic approaches.

Last modified
31 July 2020

ORPHA:635 Neuroblastoma