Children's Oncology Group's 2013 blueprint for research: neuroblastoma

Pediatr Blood Cancer. 2013 Jun;60(6):985-93. doi: 10.1002/pbc.24433. Epub 2012 Dec 19.


Estimated 5-year survival rates for patients with non-high-risk and high-risk neuroblastoma are 90% and 50%, respectively. Recent clinical trials have shown excellent outcomes with reduced therapy for non-high-risk disease. For patients with high-risk neuroblastoma treated with chemoradiotherapy, surgery, and stem cell transplantation, the addition of anti-disialoganglioside (GD2) immunotherapy plus cytokines improves survival. Upcoming trials will study the incorporation of targeted radionuclide therapy prior to myeloablative chemotherapy into high-risk treatment. Phase 2 trials will investigate druggable target(s) including mTOR inhibition and GD2-directed therapy in combination with chemotherapy for patients with recurrent neuroblastoma, and ALK inhibition for those with ALK-aberrant tumors.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / therapeutic use
  • Child
  • Clinical Trials as Topic*
  • Combined Modality Therapy
  • Humans
  • Immunotherapy
  • Neuroblastoma / therapy*
  • Research
  • Stem Cell Transplantation


  • Antineoplastic Agents