5. Neuroblastoma

  1. Ross Pinkerton MB, BCh, BaO, MD Executive Director, Division of Oncology2,
  2. Ananth Shankar MD, FRCPCH Consultant in Paediatric and Adolescent Oncology3 and
  3. Katherine K. Matthay BA, MD Mildred V. Strouss Professor of Translational Oncology, Director, Pediatric Hematology-Oncology4
  1. Katherine K. Matthay

Published Online: 8 MAY 2013

DOI: 10.1002/9781118625309.ch5

Evidence-Based Pediatric Oncology

Evidence-Based Pediatric Oncology

How to Cite

Matthay, K. K. (2013) Neuroblastoma, in Evidence-Based Pediatric Oncology (eds R. Pinkerton, A. Shankar and K. K. Matthay), John Wiley & Sons, Ltd, Oxford. doi: 10.1002/9781118625309.ch5

Editor Information

  1. 2

    Royal Children's Hospital, Children's Health Queensland, Brisbane, QLD, Australia

  2. 3

    University College London Hospitals NHS Foundation Trust London, UK

  3. 4

    Department of Pediatrics, UCSF School of Medicine and, UCSF Benioff Children's Hospital, San Francisco, CA, USA

Author Information

  1. UCSF School of Medicine, San Francisco, CA, USA

Publication History

  1. Published Online: 8 MAY 2013
  2. Published Print: 20 MAY 2013

ISBN Information

Print ISBN: 9780470659649

Online ISBN: 9781118625309



  • children;
  • minimal residual disease (MRD);
  • myeloablative therapy;
  • neuroblastoma;
  • randomized trials


Long-term survival for high-risk neuroblastoma improved from approximately 10% before 1989 to greater than 30% by 2002 with the use of more intensive combination therapy and myeloablative therapy. With the use of high-dose myeloablative chemotherapy, followed by therapy for minimal residual disease (MRD) with isotretinoin, the Children's Oncology Group (COG) was able to show with long-term follow-up of their earlier randomized trial that both modalities significantly improved survival for children with high-risk neuroblastoma, although due to the timing of the randomizations, the survival from diagnosis for the different groups could not be accurately determined. The success of the trials showing that myeloablative therapy and treatment of MRD improved outcome led to the recent COG trial showing that the addition of immunotherapy with anti-GD2 monoclonal antibody and cytokines to isotretinoin significantly improved event-free survival (EFS) for patients with good response after autologous stem cell transplant (ASCT).