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Adding the anti-GD2 antibody ch14.18 with cytokines to isotretinoin after transplant improved two-year event-free survival in high-risk neuroblastoma from 46 to 66 percent, making immunotherapy a standard part of treatment.
Phase 3 trial of 226 children with high-risk neuroblastoma who had responded to induction and autologous transplant, randomised to isotretinoin alone or with five cycles of ch14.18 (dinutuximab) plus GM-CSF and interleukin-2.
Two-year event-free survival was 66 versus 46 percent and overall survival 86 versus 75 percent; pain, capillary leak and hypersensitivity were the main toxicities. Later follow-up confirmed the benefit.
Anti-GD2 immunotherapy after consolidation is standard for high-risk neuroblastoma worldwide; interleukin-2 was later dropped after the SIOPEN trial showed no added benefit.