Neuroblastoma: paradigm for precision medicine

Pediatr Clin North Am. 2015 Feb;62(1):225-56. doi: 10.1016/j.pcl.2014.09.015.


Neuroblastoma (NB) is the third most common pediatric cancer. Although NB accounts for 7% of pediatric malignancies, it is responsible for more than 10% of childhood cancer-related mortality. Prognosis and treatment are determined by clinical and biological risk factors. Estimated 5-year survival rates for patients with non-high-risk and high-risk NB are more than 90% and less than 50%, respectively. Recent clinical trials have continued to reduce therapy for patients with non-high-risk NB, including the most favorable subsets who are often followed with observation approaches. In contrast, high-risk patients are treated aggressively with chemotherapy, radiation, surgery, and myeloablative and immunotherapies.

Keywords: ALK (anaplastic lymphoma kinase); Immunotherapy; MYCN; Myeloablative therapy (MAT); Neuroblastoma; Phox2B; Risk stratification; Segmental chromosome aberrations (SCA).

Publication types

  • Review

MeSH terms

  • Genetic Predisposition to Disease
  • Humans
  • Mutation
  • Neoplasm Staging
  • Neuroblastoma / genetics
  • Neuroblastoma / pathology*
  • Neuroblastoma / therapy
  • Prognosis
  • Risk Assessment
  • Risk Factors
  • Survival Rate