Neuroblastoma pp 213-235 | Cite as

High-Risk Neuroblastoma and Current Protocols

  • Katherine K. MatthayEmail author
  • Dominique Valteau-CouanetEmail author


High-risk neuroblastoma comprises nearly half of all neuroblastoma and have a long-term survival of <50%. Patients >18 months with International Neuroblastoma Staging System (INSS) stage 4 disease and those with a tumor INSS >1 with MYCN amplification are considered as patients with a high-risk disease in both groups and represent the majority of the patients. The current standard North American and European approach to therapy of high-risk neuroblastoma involves four phases with an intensive induction therapy, a local control using surgery and radiation, a consolidation phase with single or tandem high-dose chemotherapy and autologous transplant, and differentiating and immunotherapy for minimal residual disease (MRD) in the maintenance phase. Future improvements will depend on more extensive tumor molecular profiling at diagnosis and relapse, improvement of the quality of response using immunotherapy or other targeted therapy such as tyrosine kinase inhibitors or 131I-metaiodobenzylguanidine (131I-MIBG), and further improvements of the MRD therapy with more targeted small molecules.


High-risk neuroblastoma Very-high-risk neuroblastoma High-dose chemotherapy Autologous stem cell rescue Bu-Mel (Busulfan–Melphalan) 131 MIBG CEM (Carboplatine–Etoposide–Melphalan) Immunotherapy Maintenance treatment 13-Cis-retinoic acid Anti-GD2 


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© Springer Nature Switzerland AG 2020

Authors and Affiliations

  1. 1.Pediatric Hematology-OncologyUniversity of California San Francisco School of Medicine and UCSF Benioff Children’s HospitalSan FranciscoUSA
  2. 2.Children and Adolescent Oncology DepartmentGustave RoussyVillejuif CedexFrance

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