SIOPEN HR-NBL1
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.
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). Over 3,700 children enrolled since 2002.
- 50 vs 38 out of 100 free of a major event at 3 years with Busulfan-melphalan compared with CEM; 12 more per 100.
- Roughly one extra person helped for every 8 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.
- The p-value (0.0005) says a difference this large would rarely happen by chance; it does not say how large or how useful the difference is.
- 56 vs 60 out of 100 free of a major event at 3 years with Dinutuximab beta + IL-2 compared with Dinutuximab beta; 4 fewer per 100.
- On this measure the first group did worse, not better.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- These results apply to the people the trial enrolled: High-risk neuroblastoma (Europe): multiple randomisations, including busulfan-melphalan vs CEM conditioning and dinutuximab beta ± IL-2. People in a different situation may not see the same effect.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
Pages like this
not linked directly; found by shared links- TechnologyTandem autologous transplant
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Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
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Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
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Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
- PersonAlexandra "Alex" Scott
Shares Neuroblastoma (paediatric), Rare and paediatric cancers without markets.