# SIOPEN HR-NBL1

Source: https://onco.cc/trials/hr-nbl1/  
OnCo record `hr-nbl1` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Europe's long-running high-risk neuroblastoma trial set busulfan-melphalan as the transplant regimen and showed that adding IL-2 to anti-GD2 therapy added toxicity but not benefit.

## Summary

R1: BuMel superior to CEM (3-year EFS 50% vs 38%; Lancet Oncol 2017). R2: dinutuximab beta + IL-2 vs dinutuximab beta alone: 3-year EFS 56% vs 60%, no benefit, more toxicity (Lancet Oncol 2018). R3/R4 explored induction regimens (rapid COJEC vs modified N7; 2025 report). ClinicalTrials.gov lists an estimated 3,300 participants; enrolment opened in 2002.

## Fields

- Kind: Trial
- Status: mixed
- Last checked: 2026-09-07
- Registry id: NCT01704716
- Phase: 3
- Setting: High-risk neuroblastoma (Europe): multiple randomisations, including busulfan-melphalan vs CEM conditioning and dinutuximab beta ± IL-2
- Sponsor: SIOPEN / Children's Cancer and Leukaemia Group
- Enrolled: 3300
- Result: BuMel > CEM (EFS 50% vs 38%); IL-2 adds no benefit to anti-GD2.
- Outcomes: Event-free survival at 3 years (R1 conditioning): Busulfan-melphalan 50% vs CEM 38%; Event-free survival at 3 years (R2 immunotherapy): Dinutuximab beta + IL-2 56% vs Dinutuximab beta 60%

## Sources

- ClinicalTrials.gov NCT01704716: https://clinicaltrials.gov/study/NCT01704716

## Connected records

- cancers: [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/)
- technologies: [Autologous stem cell transplant (high-dose therapy)](https://onco.cc/technologies/autologous-stem-cell-transplant/)
- drugs: [Busulfan](https://onco.cc/drugs/busulfan/), [Dinutuximab (ch14.18) / dinutuximab beta](https://onco.cc/drugs/dinutuximab/)
- companies: [Children's Cancer and Leukaemia Group](https://onco.cc/companies/cclg/)
- people: [Ruth Ladenstein](https://onco.cc/people/ruth-ladenstein/)
- pairings: [Caution: IL-2 added to anti-GD2 therapy](https://onco.cc/pairings/il2-anti-gd2-caution/)
- bottlenecks: [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/)
- key papers: [HR-NBL1/SIOPEN: busulfan-melphalan versus carboplatin-etoposide-melphalan high-dose chemotherapy for high-risk neuroblastoma](https://onco.cc/key-papers/paper-hr-nbl1-busulfan-melphalan-ladenstein-lancet-oncol-2017/)
- institutions: [SIOP Europe (European Society for Paediatric Oncology)](https://onco.cc/institutions/siop-europe/)
- roadmaps: [Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first](https://onco.cc/roadmaps/paediatric-oncology-roadmap/)

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