Table 1.
Phenotypic and Genetic Features of Neuroblastoma, Treatment, and Survival According to Prognostic Category
| Variable | Prognostic Category* | |||
|---|---|---|---|---|
| Low Risk | Intermediate Risk | High Risk | Tumor Stage 4S | |
| Pattern of disease | Localized tumor | Localized tumor with locoregional lymph-node extension; metastases to bone marrow and bone in infants | Metastases to bone marrow and bone (except in infants) | Metastases to liver and skin (with minimal bone marrow involvement) in infants |
| Tumor genomics | Whole-chromosome gains | Whole-chromosome gains | Segmental chromosomal aberrations | Whole-chromosome gains |
| Treatment | Surgery† | Moderate-intensity chemotherapy; surgery† | Dose-intensive chemotherapy, surgery, and external-beam radiotherapy to primary tumor and resistant metastatic sites; myeloablative chemotherapy with autologous hematopoietic stem-cell rescue; isotretinoin with anti-GD2 immunotherapy | Supportive care‡ |
| Survival rate (%) | >98 | 90 to 95 | 40 to 50 | >90 |
Patients are categorized into prognostic groups according to risk, as described by the Children’s Oncology Group, with the level of risk defining the likelihood of death from disease.7 Stage 4S disease is considered separately here because of the unique phenotype of favorable biologic features and relentless early progression but ultimately full and complete regression of disease.31,32
The goal of surgery is to safely debulk the tumor mass and avoid damage to surrounding normal structures while also obtaining sufficient material for molecular diagnostic studies. Some localized tumors may spontaneously regress without surgery.33
Low-dose chemotherapy, radiation therapy, or both are used in patients with life-threatening hepatic involvement, especially in infants under 2 months of age, who are at much higher risk for life-threatening complications from massive hepatomegaly.