Estimated 5-year survival rates for patients with non-high-risk and high-risk neuroblastoma are 90% and 50%, respectively. Recent clinical trials have shown excellent outcomes with reduced therapy for non-high-risk disease. For patients with high-risk neuroblastoma treated with chemoradiotherapy, surgery, and stem cell transplantation, the addition of anti-disialoganglioside (GD2) immunotherapy plus cytokines improves survival. Upcoming trials will study the incorporation of targeted radionuclide therapy prior to myeloablative chemotherapy into high-risk treatment. Phase 2 trials will investigate druggable target(s) including mTOR inhibition and GD2-directed therapy in combination with chemotherapy for patients with recurrent neuroblastoma, and ALK inhibition for those with ALK-aberrant tumors.
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