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.
- International Neuroblastoma Pathologic Classification (INPC).
- 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.
- 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, 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.|