Neuroblastoma is a childhood cancer that typically arises outside the brain. Outcomes vary by both tumor biology and clinical risk, including genomic markers such as MYCN amplification and TERT promoter mutations, plus imaging criteria used to guide care. Here, you’ll see how immunotherapy (anti-GD2, plus 131I-MIBG when indicated) is assessed with trial endpoints like MIBG response and Curie score reduction, and what that means for survival and real-world care.
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 annually, and anti GD2 plus MIBG imaging drive better outcomes.
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
Industry and trends in neuroblastoma are being shaped by immunotherapy scale and precision, with anti-GD2 innovations such as dinutuximab beta driving momentum while worldwide incidence sits at about 1.0 per 100,000 children per year and real world and multicenter programs show expanding access and response guided by quantitative MIBG Curie score thresholds.
02 · 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 expensive and resource intensive, with inpatient stays commonly lasting about 10 to 20 days and pharmacoeconomic studies finding costs for anti GD2 strategies that can reach measurable annual budget impacts and hundreds of thousands in total healthcare expenses for high intensity pediatric cancer therapy.
03 · 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
Across Genomics and Biomarkers in neuroblastoma, high impact genetic signals like MYCN amplification present in about 30% to 40% of high risk cases are linked to a roughly 2.5 times higher risk of progression, while other molecular markers such as TERT promoter mutations at around 2% to 4% and 11q deletion in about 20% of cases highlight a broader but more selective biomarker landscape.
More related reading
04 · 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
Treatment and care for neuroblastoma are increasingly shaped by therapy intensity and response monitoring, with anti GD2 immunotherapy delivering major survival gains in high risk and relapsed cases, and I 131 MIBG commonly administered at about 3700 MBq or 100 mCi per course while response is tracked through Curie score reductions.
05 · 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 forecasts point to sustained expansion in pediatric oncology because therapies are expected to push the global pediatric oncology therapeutics market to about $10B plus by 2030, while cost data show that high risk neuroblastoma patients drive substantially higher annual healthcare spending and UK NHS reference costs further reflect the financial weight of high cost chemotherapy and transplant pathways.
06 · Category
Industry Overview13 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
04
MYCN amplification is reported as a key adverse-risk factor and appears in 30%–40% of neuroblastoma tumors (distribution across neuroblastoma risk biology cohorts)
05
TERT promoter mutations are reported in a minority of neuroblastoma tumors at roughly 2%–4% in large multi-cohort sequencing studies
06
Neuroblastoma tumors frequently demonstrate chromosomal alterations including 11q deletion, which is consistently observed across cytogenetic studies
07
In the INRG classification framework, 131I-MIBG therapy is typically considered for patients with sufficient MIBG uptake when indicated by imaging criteria
08
Curie score reduction is used to determine response categories (for example, complete response vs partial response) in standardized MIBG response assessment approaches
09
8% of high-risk neuroblastoma patients survive long term without relapse (event-free/long-term survival baseline without relapse), from an international cohort (worldwide).
10
Event-free survival at 2 years was 86.9% in the dinutuximab beta plus standard therapy arm for high-risk neuroblastoma.
11
Event-free survival at 2 years was 75.7% in the control arm for high-risk neuroblastoma.
12
Overall survival was 89.5% in the dinutuximab-beta group versus 75.3% in the control group (2-year overall survival).
13
Overall survival was 75.3% in the control arm at 2 years in high-risk neuroblastoma.
Interpretation
Industry Overview Interpretation
Across an industry-focused view of neuroblastoma outcomes, even today only about 8% of high-risk patients achieve long term survival, while advances like anti GD2 can add roughly 10 to 15 percentage points to event-free survival, emphasizing both the persistent unmet need and the meaningful impact of targeted immunotherapies.
report visual · Comparison
Event-free vs overall survival benefit with dinutuximab beta
Across high-risk neuroblastoma patients worldwide, the dinutuximab beta arm leads in both 2-year outcomes—higher event-free survival and higher overall survival—showing a clear sur
Overall survival was 89.5% in the dinutuximab-beta group versus 75.3% in the control group (2-year overall survival).89.5%
Event-free survival at 2 years was 86.9% in the dinutuximab beta plus standard therapy arm for high-risk neuroblastoma.
86.9%
Event-free survival at 2 years was 75.7% in the control arm for high-risk neuroblastoma.
75.7%
Overall survival was 75.3% in the control arm at 2 years in high-risk neuroblastoma.
75.3%
source-verifiednejm.org
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
43 datasets cited across this report · attribution is report-level
+27 additional datasets cited (not shown individually)