OnCo
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COG ANBL0032

The trial that made anti-GD2 immunotherapy standard for children with high-risk neuroblastoma, improving survival by about 20 points.

2-year EFS 66% vs 46%; 2-year OS 86% vs 75% (NEJM 2010); long-term 5-year OS 73% vs 57% (Yu 2021). Randomisation stopped early for efficacy; approval 2015.

Setting
High-risk neuroblastoma after transplant: ch14.18 (dinutuximab) + GM-CSF + IL-2 + isotretinoin vs isotretinoin alone
Phase
Phase 3
Sponsor
Children's Oncology Group
Registry
Headline result
2-year EFS 66% vs 46%; OS 86% vs 75%.
Reported
2010
Enrolled
226
Replication
SIOPEN HR-NBL1 confirmed anti-GD2 benefit with dinutuximab beta (and showed IL-2 adds no efficacy).

Outcomes

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In plain words
What these results mean for people, not percentages
226 people took part
Event-free survival at 2 yearsprimarysurrogate endpoint
  • 66 vs 46 out of 100 free of a major event at 2 years with Immunotherapy + isotretinoin compared with Isotretinoin; 20 more per 100.
  • Roughly one extra person helped for every 5 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.
  • The p-value (0.01) says a difference this large would rarely happen by chance; it does not say how large or how useful the difference is.
  • 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.
Overall survival at 2 yearssurvival endpoint
  • 86 vs 75 out of 100 alive at 2 years with Immunotherapy + isotretinoin compared with Isotretinoin; 11 more per 100.
  • Roughly one extra person helped for every 9 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.
  • The p-value (0.02) says a difference this large would rarely happen by chance; it does not say how large or how useful the difference is.
  • Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.
Be careful
  • These results apply to the people the trial enrolled: High-risk neuroblastoma after transplant: ch14.18 (dinutuximab) + GM-CSF + IL-2 + isotretinoin vs isotretinoin alone. 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.

226 participants enrolled.

Event-free survival at 2 yearsprimary
· p = 0.01
Immunotherapy + isotretinoin66 of 100
n = 113
Isotretinoin46 of 100
n = 113
Source
Overall survival at 2 years
· p = 0.02
Immunotherapy + isotretinoin86 of 100
Isotretinoin75 of 100
EndpointArmnValueHR (95% CI)pSource
Event-free survival at 2 yearsprimaryImmunotherapy + isotretinoin11366%0.01link
Isotretinoin11346%
Overall survival at 2 yearsImmunotherapy + isotretinoin86%0.02
Isotretinoin75%
Replication
SIOPEN HR-NBL1 confirmed anti-GD2 benefit with dinutuximab beta (and showed IL-2 adds no efficacy).

Connected

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