HR-NBL1/SIOPEN: busulfan-melphalan versus carboplatin-etoposide-melphalan high-dose chemotherapy for high-risk neuroblastoma
Busulfan and melphalan as the high-dose conditioning before stem cell rescue gave better event-free survival and less severe toxicity than the carboplatin-etoposide-melphalan regimen in high-risk neuroblastoma, becoming Europe's standard.
Overview
Phase 3 trial within the European HR-NBL1 study randomising 598 children with high-risk neuroblastoma after rapid COJEC induction to busulfan-melphalan or carboplatin-etoposide-melphalan (CEM) high-dose chemotherapy with autologous stem cell rescue.
Three-year event-free survival was 50 versus 38 percent (hazard ratio about 0.7), overall survival 60 versus 48 percent, and severe toxicity and toxic deaths were fewer with busulfan-melphalan.
- Three-year event-free survival 50 percent vs 38 percent.
- Severe acute toxicity 4 percent vs 10 percent.
Busulfan-melphalan is the standard single high-dose regimen for high-risk neuroblastoma in Europe and many other regions; North America uses tandem transplant.
- Randomisation occurred after induction, so early failures are excluded.
- Veno-occlusive disease is the characteristic busulfan toxicity.
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