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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 COG risk group assignment. 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, and survival is greater than 98%.
  • For intermediate-risk tumors, chemotherapy is usually given before resection, with the amount and duration based on clinical and tumor biological risk factors. The survival rate for intermediate-risk patients in recent trials is close to 95%, 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, 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 NeuroblastomaSurgery.
Observation without biopsy.
Chemotherapy.
Intermediate-Risk NeuroblastomaSurgery and chemotherapy
Surgery and observation(for infants).
Radiation therapy.
High-Risk NeuroblastomaA regimen of chemotherapy, surgery, SCT, and radiation therapy, cis-retinoic acid, anti-GD2 antibody ch14.18, and interleukin-2/GM-CSF.
Stage 4S NeuroblastomaObservation with supportive care(for asymptomatic patients).
Chemotherapy(for symptomatic patients).
Recurrent NeuroblastomaLocoregional recurrence in patients initially classified as low riskSurgery followed by observation or chemotherapy.
Metastatic recurrence in patients initially classified as low riskObservation.
Surgery followed by chemotherapy.
Locoregional recurrence in patients initially classified as intermediate riskSurgery (complete resection).
Surgery (incomplete resection) followed by chemotherapy.
Metastatic recurrence in patients initially classified as intermediate riskHigh-dose chemotherapy.
Recurrence in patients initially classified as high riskSecond autologous SCT after retrieval chemotherapy.
Chemotherapy.
131 I-mIBG alone, in combination with other therapy, or followed by stem cell rescue.
Recurrence in the central nervous systemSurgery and radiation therapy.
Novel therapeutic approaches.
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