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Brain Cancer Health Center

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Neuroblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment of High-Risk Neuroblastoma

The Children's Oncology Group (COG) high-risk group assignment criteria are described in Table 8.

Table 8. Children's Oncology Group (COG) Neuroblastoma High-Risk Group Assignment Schema

INSS Stage Age MYCNStatus INPC Classification DNA Ploidya
INPC = International Neuroblastoma Pathologic Classification; INSS = International Neuroblastoma Staging System.
a DNA Ploidy: DNA Index (DI) > 1 is favorable, DI = 1 is unfavorable; hypodiploid tumors (with DI < 1) will be treated as a tumor with a DI > 1 (DI < 1 [hypodiploid] to be considered favorable ploidy).
b INSS stage 2A/2B symptomatic patients with spinal cord compression, neurologic deficits, or other symptoms are treated with immediate chemotherapy for four cycles.
c INSS stage 3 or stage 4 patients with clinical symptoms as listed above receive immediate chemotherapy.
2A/2Bb ≥365 d-21 y Amplified Unfavorable -
3c <365 d Amplified Any Any
≥365 d-21 y Nonamplified Unfavorable -
≥365 d-21 y Amplified Any -
4c <365 d Amplified Any Any
≥548 d-21 y Any Any -
4S <365 d Amplified Any Any

Approximately 8% to 10% of infants with stage 4S disease will have MYCN-amplified tumors and are usually treated on high-risk protocols. The overall event-free survival (EFS) and overall survival (OS) for infants with stage 4 and 4S disease and MYCN-amplification were only 30% at 2 to 5 years posttreatment in a European study.[1]

For children with high-risk neuroblastoma, long-term survival with current treatments is about 54%.[2] Children with aggressively treated, high-risk neuroblastoma may develop late recurrences, some more than 5 years after completion of therapy.[3,4]

Treatment Options for High-Risk Neuroblastoma

Outcomes for patients with high-risk neuroblastoma remain poor despite recent improvements in survival in randomized trials.

A treatment option for high-risk neuroblastoma is the following:

  1. A regimen of chemotherapy, surgery, stem cell transplant (SCT), radiation therapy, and anti-GD2 antibody ch14.18 with interleukin-2/granulocyte-macrophage colony-stimulating factor (GM-CSF) and isotretinoin.

Chemotherapy, surgery, SCT, radiation therapy, and anti-GD2 antibody ch14.18, with interleukin-2/GM-CSF and isotretinoin

Treatment for patients with high-risk disease is generally divided into the following three phases:

  • Induction (includes chemotherapy and surgical resection).
  • Consolidation (hematopoietic stem cell rescue/transplantation [HSCT] and radiation therapy to the site of the primary tumor).
  • Maintenance (immunotherapy and retinoid).
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