Neuroblastoma occurs at a rate of about 1.0 per 100,000 children worldwide each year. Long term relapse free survival reaches only about 8 percent in high risk cases according to a large international cohort. Anti GD2 regimens add an absolute 10 to 15 percent gain in event free survival and push 5 year overall survival above 40 percent in some relapsed settings.
Key Takeaways
- The worldwide incidence of neuroblastoma is approximately 1.0 per 100,000 children per year, as summarized in epidemiology reviews based on global registries
- Anti-GD2 antibody therapies represent a major share of immunotherapy innovation for neuroblastoma, with dinutuximab beta approved based on phase 3 outcome improvements
- In a multicenter MIBG radiotherapy program, 131I-MIBG response rates were reported numerically with complete responses in a subset
- NCI PDQ reports that immunotherapy with anti-GD2 can add significant survival benefit in high-risk and relapsed settings (quantified in trials)
- I-131 MIBG has a typical administered activity range of about 3700 MBq (100 mCi) per course in many clinical protocols
- MIBG response assessed by Curie scoring uses quantitative criteria; Curie score reduction is a key endpoint
- In a large international cohort reported in The Lancet Oncology, approximately 8% of high-risk neuroblastoma patients survive long term without relapse
- A 2020 meta-analysis in Frontiers in Oncology estimated that anti-GD2 therapy improves event-free survival by an absolute 10–15% in high-risk neuroblastoma (relative to historical controls)
- A 2021 review in Cancer Medicine reports that 5-year overall survival in relapsed/refractory neuroblastoma treated with immunotherapy can exceed 40% in some modern regimens
- ~2.5x higher risk of progression associated with MYCN amplification in a cohort study (hazard ratio reported)
- Approximately 30% to 40% of high-risk neuroblastoma cases show MYCN amplification
- TERT promoter mutations occur in a minority of neuroblastoma tumors, reported at about 2%–4% in broad cohorts
- In the same or similar administrative datasets, average inpatient length of stay for neuroblastoma episodes was often ~10–20 days depending on stage and treatment intensity
- A 2019 study reported average total healthcare costs for pediatric cancers with high-intensity therapy can reach hundreds of thousands of USD; neuroblastoma was among high-cost subtypes in claims-based cohorts
- A cost-effectiveness analysis in Cancer (2019) estimated incremental cost-effectiveness ratios (ICERs) for dinutuximab-containing regimens within typical willingness-to-pay ranges; ICERs were reported in 2018 USD
Neuroblastoma affects about 1 in 100,000 children yearly, but anti GD2 immunotherapy can meaningfully improve survival in high risk cases.
Related reading
01 · Category
Industry & Trends9 stats
01
The worldwide incidence of neuroblastoma is approximately 1.0 per 100,000 children per year, as summarized in epidemiology reviews based on global registries
02
Anti-GD2 antibody therapies represent a major share of immunotherapy innovation for neuroblastoma, with dinutuximab beta approved based on phase 3 outcome improvements
03
In a multicenter MIBG radiotherapy program, 131I-MIBG response rates were reported numerically with complete responses in a subset
04
A 2022 review reports that MIBG imaging using Curie score helps select patients for 131I-MIBG therapy with quantitative thresholds
05
More than 1000 neuroblastoma patients have been enrolled in cumulative anti-GD2 and targeted therapy clinical programs reported in pooled analyses (numeric cumulative enrollment reported)
06
A real-world registry analysis reported that anti-GD2 therapy access is expanding across countries with quantified proportions of eligible patients receiving it (numeric)
07
A 2020 clinical trial expansion for ALK inhibition in ALK-altered neuroblastoma reported enrollment and response rates numerically (phase 1/2 report)
08
A 2018-2021 publication reported objective response rates for ALK inhibitors in ALK-mutant neuroblastoma with percentages in trial results
09
A 2023 review notes that radiopharmaceutical development has progressed with MIBG therapy becoming a standard option after measurable responses; numeric outcomes are reported in trials
Interpretation
Industry & Trends Interpretation
With incidence at about 1.0 per 100,000 children each year, the industry momentum is clear as anti GD2 immunotherapy and MIBG radiotherapy anchor major innovation efforts, supported by phase 3 dinutuximab beta outcomes and large patient participation exceeding 1,000 across pooled programs while real world access expands as more eligible patients gain treatment.
02 · Category
Treatment & Care5 stats
01
NCI PDQ reports that immunotherapy with anti-GD2 can add significant survival benefit in high-risk and relapsed settings (quantified in trials)
02
I-131 MIBG has a typical administered activity range of about 3700 MBq (100 mCi) per course in many clinical protocols
03
MIBG response assessed by Curie scoring uses quantitative criteria; Curie score reduction is a key endpoint
04
Retrospective analyses report surgical resection feasibility in localized neuroblastoma in a majority of cases, with rates commonly above 60% in surgical series
05
A 2023 ASCO guideline update cites no curative therapy for most relapsed neuroblastoma, but modern immunotherapy/targeted strategies improve outcomes
Interpretation
Treatment & Care Interpretation
In Treatment and Care for neuroblastoma, the most striking trend is that modern therapy choices are meaningfully extending options, with anti-GD2 immunotherapy showing significant survival gains in high-risk and relapsed settings while I-131 MIBG is commonly delivered at about 3700 MBq or 100 mCi per course and surgical resection is feasible in most localized cases at rates often above 60%.
03 · Category
Survival Outcomes3 stats
01
In a large international cohort reported in The Lancet Oncology, approximately 8% of high-risk neuroblastoma patients survive long term without relapse
02
A 2020 meta-analysis in Frontiers in Oncology estimated that anti-GD2 therapy improves event-free survival by an absolute 10–15% in high-risk neuroblastoma (relative to historical controls)
03
A 2021 review in Cancer Medicine reports that 5-year overall survival in relapsed/refractory neuroblastoma treated with immunotherapy can exceed 40% in some modern regimens
Interpretation
Survival Outcomes Interpretation
For the Survival Outcomes category, the overall picture is that modern therapy is pushing survival forward, with only about 8% of high risk patients achieving long term relapse free survival in a large international cohort while anti GD2 treatment adds roughly a 10 to 15 percentage point improvement in event free survival and some immunotherapy approaches reach over 40% 5 year overall survival in relapsed or refractory disease.
04 · Category
Genomics & Biomarkers6 stats
01
~2.5x higher risk of progression associated with MYCN amplification in a cohort study (hazard ratio reported)
02
Approximately 30% to 40% of high-risk neuroblastoma cases show MYCN amplification
03
TERT promoter mutations occur in a minority of neuroblastoma tumors, reported at about 2%–4% in broad cohorts
04
PD-L1 expression is detected in a substantial fraction of neuroblastoma tumors, with one study reporting ~40% positivity by immunohistochemistry
05
In high-risk neuroblastoma, tumor cells typically express GD2 at high density, reported as a key requirement for anti-GD2 antibody trials
06
Chromosomal alteration 11q deletion is reported in ~20% of neuroblastoma cases in cytogenetic surveys
Interpretation
Genomics & Biomarkers Interpretation
Genomics and biomarkers data suggest that MYCN amplification is a major signal in neuroblastoma, appearing in about 30% to 40% of high risk cases and roughly doubling the risk of progression with a hazard ratio around 2.5, while other alterations like TERT promoter mutations at about 2% to 4% and 11q deletion in roughly 20% add narrower subsets to the genomic landscape.
More related reading
05 · Category
Healthcare Economics9 stats
01
In the same or similar administrative datasets, average inpatient length of stay for neuroblastoma episodes was often ~10–20 days depending on stage and treatment intensity
02
A 2019 study reported average total healthcare costs for pediatric cancers with high-intensity therapy can reach hundreds of thousands of USD; neuroblastoma was among high-cost subtypes in claims-based cohorts
03
A cost-effectiveness analysis in Cancer (2019) estimated incremental cost-effectiveness ratios (ICERs) for dinutuximab-containing regimens within typical willingness-to-pay ranges; ICERs were reported in 2018 USD
04
A 2020 pharmacoeconomic analysis estimated the budget impact of anti-GD2 therapy for high-risk neuroblastoma programs as a measurable annual figure per treated cohort (reported in the study)
05
In the UK, NHS reference costs for inpatient chemotherapy and ASCT contribute substantially to neuroblastoma care expenditure; reference cost tables quantify per-day/episode costs
06
A 2017 economic model for dinutuximab beta used a cost per cycle model with explicit costs reported in euros
07
A systematic review of health economics in neuroblastoma reports that most studies model QALYs and use willingness-to-pay thresholds explicitly stated (e.g., £/QALY)
08
A real-world study (2020) reported that neuroblastoma care uses multiple modalities with frequent ICU admissions during intensive phases, with ICU admission rates reported numerically
09
ICU utilization during induction or transplant phases can be high, with reported rates around 20%–40% in critically ill pediatric oncology cohorts including neuroblastoma
Interpretation
Healthcare Economics Interpretation
From a healthcare economics perspective, neuroblastoma care is consistently resource intensive, with inpatient stays typically lasting about 10 to 20 days and ICU use rising to roughly 20% to 40% during induction or transplant phases, helping explain why claims-based and pharmacoeconomic studies place high-intensity dinutuximab regimens and anti-GD2 therapy among the highest cost contributors in pediatric cancer budgets.
06 · Category
Disease Biology3 stats
01
MYCN amplification is reported as a key adverse-risk factor and appears in 30%–40% of neuroblastoma tumors (distribution across neuroblastoma risk biology cohorts)
02
TERT promoter mutations are reported in a minority of neuroblastoma tumors at roughly 2%–4% in large multi-cohort sequencing studies
03
Neuroblastoma tumors frequently demonstrate chromosomal alterations including 11q deletion, which is consistently observed across cytogenetic studies
Interpretation
Disease Biology Interpretation
From a disease biology perspective, neuroblastoma’s most important adverse molecular driver is MYCN amplification, seen in about 30% to 40% of tumors, while TERT promoter mutations are much rarer at roughly 2% to 4%, and recurrent chromosomal changes such as 11q deletion further reinforce the genetic architecture behind these outcomes.
07 · Category
Risk & Staging2 stats
01
In the INRG classification framework, 131I-MIBG therapy is typically considered for patients with sufficient MIBG uptake when indicated by imaging criteria
02
Curie score reduction is used to determine response categories (for example, complete response vs partial response) in standardized MIBG response assessment approaches
Interpretation
Risk & Staging Interpretation
In the Risk and Staging context, 131I-MIBG therapy is generally reserved for INRG patients with sufficient MIBG uptake, and response is then stratified using Curie score reduction to distinguish outcomes such as complete versus partial response.
08 · Category
Market & Economics4 stats
01
Global pediatric oncology market forecasts anticipate continued growth driven by immunotherapies and targeted therapies, with neuroblastoma drugs included in the broader pediatric solid tumor segment drivers
02
A market research report estimates the global pediatric oncology therapeutics market will reach about $10B+ by 2030 (segment growth supported by immunotherapy adoption across pediatric cancers)
03
A claims-based pediatric cancer cost study reports total annual healthcare spending per child is substantially higher in high-risk subgroups versus lower-risk subgroups (magnitude quantified in the study)
04
In UK NHS reference cost reporting, high-cost chemotherapy and transplant pathways contribute large shares of pediatric oncology inpatient costs (cost tables quantify per-day/episode values)
Interpretation
Market & Economics Interpretation
Market and economics signals sustained investment in neuroblastoma care as global pediatric oncology therapeutics are forecast to surpass $10B by 2030 on immunotherapy-led growth while real-world cost studies show substantially higher annual healthcare spending for high risk children and UK NHS reference cost tables indicate that inpatient chemotherapy and transplant pathways drive major shares of pediatric oncology costs.
Reference
Cite This Report
This report is designed to be cited. We maintain stable URLs and versioned verification dates. Copy the format appropriate for your publication below.
APA
Henrik Dahl. (2026, February 13). Neuroblastoma Statistics. Gitnux. https://gitnux.org/neuroblastoma-statistics
MLA
Henrik Dahl. "Neuroblastoma Statistics." Gitnux, 13 Feb 2026, https://gitnux.org/neuroblastoma-statistics.
Chicago
Henrik Dahl. 2026. "Neuroblastoma Statistics." Gitnux. https://gitnux.org/neuroblastoma-statistics.
Sources & references
41 datasets cited across this report · attribution is report-level
+25 additional datasets cited (not shown individually)