# HR-NBL1/SIOPEN: busulfan-melphalan versus carboplatin-etoposide-melphalan high-dose chemotherapy for high-risk neuroblastoma

Source: https://onco.cc/key-papers/paper-hr-nbl1-busulfan-melphalan-ladenstein-lancet-oncol-2017/  
OnCo record `paper-hr-nbl1-busulfan-melphalan-ladenstein-lancet-oncol-2017` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Summary

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.

## Fields

- Kind: Key paper
- Last checked: 2026-09-17
- Journal: The Lancet Oncology
- Year: 2017
- DOI: 10.1016/S1470-2045(17)30070-0
- Authors: Ladenstein R, Pötschger U, Pearson ADJ, et al.
- Findings: Three-year event-free survival 50 percent vs 38 percent.; Severe acute toxicity 4 percent vs 10 percent.
- What it means: Busulfan-melphalan is the standard single high-dose regimen for high-risk neuroblastoma in Europe and many other regions; North America uses tandem transplant.
- Caveats: Randomisation occurred after induction, so early failures are excluded.; Veno-occlusive disease is the characteristic busulfan toxicity.

## Sources

- Lancet Oncol 2017: https://doi.org/10.1016/S1470-2045(17)30070-0
- PubMed: https://pubmed.ncbi.nlm.nih.gov/28259608/

## Connected records

- cancers: [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/)
- drugs: [Busulfan](https://onco.cc/drugs/busulfan/), [Carboplatin](https://onco.cc/drugs/carboplatin/), [Etoposide](https://onco.cc/drugs/etoposide/)
- trials: [SIOPEN HR-NBL1](https://onco.cc/trials/hr-nbl1/)
- journals: [The Lancet Oncology](https://onco.cc/journals/lancet-oncology/)

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