About 12% of deaths from cancer among children <15 years old are from neuroblastoma and nearly half of all patients with neuroblastoma have a high-risk phenotype. Standard treatment for high-risk disease is myeloablative therapy with stem-cell rescue followed by treatment of minimal residual disease with isotretinoin but results are unsatisfactory. Now a multicentre US study (New England Journal of Medicine 2010;363:1324–34) has shown that immunotherapy may benefit children with high-risk neuroblastoma. A total of 226 patients with high risk neuroblastoma in remission after myeloablative therapy and stem-cell rescue were randomised to immunotherapy plus isotretinoin or isotretinoin alone. Immunotherapy consisted of the monoclonal antibody, ch14.18, against the disialoganglioside GD2 expressed by all neuroblastomas plus granulocyte-macrophage colony-stimulating factor and interleukin-2. The trial was stopped early because of better results in the immunotherapy group. Overall survival at 2 years was 86% (immunotherapy plus isotretinoin) versus 75% (isotretinoin alone) and 2-year event-free survival 66% versus 46%.

These researchers conclude that more routine use of this immunotherapy regimen for such patients may be beneficial.

In stark contrast to high-risk neuroblastoma, intermediate-risk neuroblastoma has a >80% rate of overall survival with moderately aggressive chemotherapy. A multicentre study (New England Journal of Medicine 2010;363:1313–23) has shown that very high survival rates can be achieved with substantially reduced duration and dosage …