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Brain Cancer Health Center

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Neuroblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment Option Overview for Neuroblastoma

Because most children with neuroblastoma in North America are treated according to the Children's Oncology Group (COG) risk-group assignment, the treatments described in this summary are based on the most recently published COG risk stratification system. Each child is assigned to a low-risk, intermediate-risk, or high-risk group (refer to Tables 6, 7, and 8 for more information) based on the following:[1,2,3,4,5,6]

  • International Neuroblastoma Staging System (INSS) stage.
  • Age.
  • International Neuroblastoma Pathologic Classification (INPC).
  • Ploidy.
  • Amplification of the MYCN oncogene within tumor tissue.[1,2,3,4,5,6]

Other biological factors that influence treatment selection include unbalanced 11q loss of heterozygosity and loss of heterozygosity for chromosome 1p.[7,8]

The treatment of neuroblastoma has evolved over the past 60 years. Generally, treatment is based on whether the tumor is low, intermediate, or high risk:

  • For low-risk tumors, the approach is either observation or resection. Five-year overall survival (OS) was 97% in a large COG study.[9]
  • For intermediate-risk tumors, chemotherapy is usually given before definitive resection, with the amount and duration based on clinical and tumor biological risk factors and response to therapy. The 3-year OS rate for intermediate-risk patients was about 96% in a large COG study,[10] and thus, the current trend is to decrease chemotherapy to diminish side effects.
  • For high-risk patients, treatment has intensified to include chemotherapy, surgery, radiation therapy, hematopoietic stem cell transplantation, differentiation therapy, and immunotherapy, resulting in survival rates of 40% to 50%.

Table 5. Treatment Options for Neuroblastoma

Stage (COG Risk-Group Assignment) Treatment Options
COG = Children's Oncology Group; GM-CSF = granulocyte-macrophage colony-stimulating factor; 131I-mIBG = iodine 131-metaiodobenzylguanidine; SCT = stem cell transplant.
Low-Risk Neuroblastoma Surgeryfollowed by observation.
Chemotherapy with or without surgery(for symptomatic disease or unresectable progressive disease after surgery).
Observation without biopsy(for perinatal neuroblastoma with small adrenal tumors).
Intermediate-Risk Neuroblastoma Chemotherapy with or without surgery.
Surgery and observation(in infants).
Radiation therapy (only for emergent therapy).
High-Risk Neuroblastoma A regimen of chemotherapy, surgery, SCT, radiation therapy, and anti-GD2 antibody ch14.18, with interleukin-2/GM-CSF and isotretinoin.
Stage 4S Neuroblastoma Observation with supportive care(for asymptomatic patients with favorable tumor biology).
Chemotherapy(for symptomatic patients, very young infants, or those with unfavorable biology).
Recurrent Neuroblastoma Locoregional recurrence in patients initially classified as low risk Surgery followed by observation or chemotherapy.
Chemotherapy that may be followed by surgery.
Metastatic recurrence in patients initially classified as low risk Observation(if metastatic disease is in a 4S pattern in an infant).
Locoregional recurrence in patients initially classified as intermediate risk Surgery (complete resection).
Surgery (incomplete resection) followed by chemotherapy.
Metastatic recurrence in patients initially classified as intermediate risk High-risk therapy.
Recurrence in patients initially classified as high risk Chemotherapy.
131 I-mIBG alone, in combination with other therapy, or followed by stem cell rescue.
Second autologous SCT after retrieval chemotherapy.
Recurrence in the central nervous system Surgery and radiation therapy.
Novel therapeutic approaches.
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