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Two consecutive high-dose chemotherapy courses with stem cell rescue improved three-year event-free survival compared with a single transplant in high-risk neuroblastoma, and the gain was largest in children who went on to receive anti-GD2 immunotherapy.
Children's Oncology Group phase 3 trial of 652 children with high-risk neuroblastoma, of whom 355 were randomised after induction to single transplant (carboplatin-etoposide-melphalan) or tandem transplant (thiotepa-cyclophosphamide followed by carboplatin-etoposide-melphalan).
Three-year event-free survival was 61.6 versus 48.4 percent; overall survival did not differ significantly (74.1 versus 69.6 percent); among patients receiving immunotherapy, event-free survival was 73.7 versus 56.0 percent.
Tandem transplant is the standard consolidation in North American protocols for high-risk neuroblastoma; Europe uses single busulfan-melphalan.
Shares COG ANBL0532, High-risk neuroblastoma.