trialsTrialPositive

COG ANBL0032

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

COG ANBL0032, trial NCT00026312 run by the Children's Oncology Group and published in the New England Journal of Medicine in 2010, made anti-GD2 immunotherapy standard for children with high-risk neuroblastoma, improving survival by about twenty points. It randomised 226 children after transplant to the anti-GD2 antibody ch14.18, now dinutuximab, with GM-CSF, interleukin-2 and isotretinoin, or isotretinoin alone, met its primary event-free survival endpoint at two years and improved overall survival, was stopped early for efficacy, and led to approval in 2015 with long-term follow-up confirming the gain. OnCo links it to paediatric neuroblastoma, GD2 as a target, dinutuximab, the Children's Oncology Group, Alice L. Yu, Julie R. Park and the bottleneck of paediatric cancers without markets. Whether anti-GD2 can move earlier into induction is the open question.

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%.

Started

2001-10-26

Reported

2010

Enrolled

226 randomisedClinicalTrials.gov lists 1,449 participants (actual) enrolled over the life of the study; the NEJM 2010 analysis randomised 226 eligible patients.

Replication

SIOPEN HR-NBL1 confirmed anti-GD2 benefit with dinutuximab beta (and showed IL-2 adds no efficacy).

Outcomes

In plain words

What these results mean for people, not percentages

226 randomised

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.

Caveats

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 randomised.

Event-free survival at 2 yearsprimary

· p = 0.01

Immunotherapy + isotretinoin66 of 100

n = 113

Isotretinoin46 of 100

n = 113

Download

Overall survival at 2 years

· p = 0.02

Immunotherapy + isotretinoin86 of 100

Isotretinoin75 of 100

Download

Endpoint	Arm	n	Value	HR (95% CI)	p	Source
Event-free survival at 2 yearsprimary	Immunotherapy + isotretinoin	113	66%	-	0.01	link
Event-free survival at 2 yearsprimary	Isotretinoin	113	46%	-	0.01	link
Overall survival at 2 years	Immunotherapy + isotretinoin	-	86%	-	0.02	link
Overall survival at 2 years	Isotretinoin	-	75%	-	0.02	link

Replication

SIOPEN HR-NBL1 confirmed anti-GD2 benefit with dinutuximab beta (and showed IL-2 adds no efficacy).

Connected

Similar pages

cancers

High-risk neuroblastoma Neuroblastoma (paediatric)

technologies

Monoclonal antibodies

targets

GD2 (disialoganglioside)

drugs

ApprovedMonoclonal antibody (anti-GD2, chimeric)

Dinutuximab (ch14.18) / dinutuximab beta · Unituxin / Qarziba (EU)

ApprovedOral retinoid (differentiating agent)

Isotretinoin · Accutane

companies

Children's Oncology Group (COG)

roadmaps

Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first

people

Alice L. Yu Julie R. Park

bottlenecks

Rare and paediatric cancers without markets