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Treatment Option Overview for Neuroblastoma
Source: Neuroblastoma Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 28, 2025 · Captured 2026-09-09.
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Generally, treatment of neuroblastoma is based on whether the tumor is classified as non-high risk (low or intermediate risk) or high risk. Because 5-year survival rates are generally 90% or higher, the goal of treatment for non–high-risk disease is to cure the disease with minimal toxicity. Outcomes for patients with high-risk disease have improved over time with the use of increasingly intensive multimodal therapy, but they remain suboptimal.
Low risk. For patients with low-risk tumors, the approach is either observation or resection, with chemotherapy restricted to symptomatic patients with low-risk biology. The 5-year overall survival (OS) rate was 98% for the low-risk patients among more than 5,000 patients enrolled in the Children's Oncology Group (COG) ANBL00B1 (NCT00904241) biology study.[1]
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Intermediate risk. For patients with intermediate-risk tumors, chemotherapy is often given before definitive resection. Multiagent chemotherapy consisting of doxorubicin, cyclophosphamide, a platinum drug, and etoposide is used. The number of chemotherapy cycles is based on clinical and tumor biological risk factors and response to therapy.[2] The goal of chemotherapy is to deliver a sufficient duration of chemotherapy (with or without surgery) to achieve at least a partial response (at least 50% reduction of soft tissue masses) and resolution of metastatic disease.[2] In some studies, select patients have been observed without undergoing chemotherapy or attempted resection. The 5-year OS rate was about 95% for the intermediate-risk patients among more than 5,000 patients enrolled in the COG ANBL00B1 (NCT00904241) biology study.[1] In the COG ANBL0531 (NCT00499616) study, the duration and intensity of chemotherapy was decreased in several subsets of intermediate-risk children to further diminish side effects; no declines in outcomes were seen.[2,3]
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High risk. For high-risk patients, treatment has intensified to include chemotherapy, surgery, radiation therapy, myeloablative therapy and hematopoietic stem cell transplant (HSCT), isotretinoin, and immunotherapy, resulting in 5-year survival rates of 62%.[1] Statistically significant improvement in event-free survival (EFS) was observed in a randomized phase III COG study (ANBL0532 [NCT00567567]) with tandem cycles of myeloablative therapy and HSCT, compared with a single cycle of myeloablative therapy and HSCT. The 3-year EFS rate for patients who received tandem transplants was superior (P = .006) to the EFS rate for patients who received single transplants, although OS was not statistically different. In this study, a large proportion of patients were not randomized.[4][Level of evidence A1] For more information, see the Consolidation phase section.
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Table 6 summarizes the treatment options for patients with low-risk, intermediate-risk, and high-risk disease.
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| COG Risk-Group Assignment | Treatment Options | |
|---|---|---|
| COG = Children's Oncology Group; GM-CSF = granulocyte-macrophage colony-stimulating factor; HSCT = hematopoietic stem cell transplant. | ||
| Low-Risk Neuroblastoma | Surgery followed by observation. | |
| Observation with or without biopsy. | ||
| Chemotherapy with or without surgery (for symptomatic disease or unresectable progressive disease after surgery). | ||
| Radiation therapy (only for emergency therapy). | ||
| Intermediate-Risk Neuroblastoma | Chemotherapy with or without surgery. | |
| Surgery and observation (in infants). | ||
| Radiation therapy (for progressive disease, if needed). | ||
| High-Risk Neuroblastoma | A regimen of chemotherapy, surgery, tandem cycles of myeloablative therapy and HSCT, radiation therapy, and dinutuximab, with GM-CSF and isotretinoin. | |
| Stage 4S Neuroblastoma | Observation with supportive care (for asymptomatic patients with favorable tumor biology). | |
| Chemotherapy (for symptomatic patients, those with unfavorable tumor biology, and infants aged <3 months). | ||
| Surgery (rarely, for patients with hepatomegaly that compromises the kidney or other abdominal organs). | ||
| Radiation therapy (rarely, for patients with symptoms related to hepatomegaly from metastatic disease). | ||
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- Surgery followed by observation
- Observation with or without biopsy
- Chemotherapy with or without surgery
- Radiation therapy
- Chemotherapy with or without surgery
- Surgery and observation
- Radiation therapy
- A regimen of chemotherapy, surgery, tandem cycles of myeloablative therapy and HSCT, radiation therapy, and dinutuximab, with GM-CSF and isotretinoin
- Observation with supportive care
- Chemotherapy
- Surgery
- Radiation therapy
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