Definition and research guide
This page combines a MeSH definition, publication statistics and selected research abstracts. It is a starting point for reading the literature, not a medical textbook or a systematic evidence review.
Content available: 4 complete abstracts (729 words), 10 references. Full article text is not stored or reproduced here; follow the PMID, DOI or PMC links to read the original sources and their access terms.
Definition
A common neoplasm of early childhood arising from neural crest cells in the sympathetic nervous system, and characterized by diverse clinical behavior, ranging from spontaneous remission to rapid metastatic progression and death. This tumor is the most common intraabdominal malignancy of childhood, but it may also arise from thorax, neck, or rarely occur in the central nervous system. Histologic features include uniform round cells with hyperchromatic nuclei arranged in nests and separated by fibrovascular septa. Neuroblastomas may be associated with the opsoclonus-myoclonus syndrome. (From DeVita et al., Cancer: Principles and Practice of Oncology, 5th ed, pp2099-2101; Curr Opin Oncol 1998 Jan;10(1):43-51)
Definition: NLM Medical Subject Headings (MeSH) scope note (Public domain (U.S. National Library of Medicine); attribution requested). View the MeSH record.
Also known as: Neuroblastomas.
Where it fits in the MeSH classification
- Broader topics
- Neuroectodermal Tumors, Primitive, Peripheral
- Narrower topics
- Esthesioneuroblastoma, Olfactory; Ganglioneuroblastoma
Research landscape
PubMed indexes 34,205 records tagged with the MeSH heading “Neuroblastoma” (counts retrieved 2026-10-29). 5,417 of them (15.8%) were published from 2020 onward, and the busiest period so far is through 1999 with 14,031 records.
Reviews account for 2,388 records (7.0%) and meta-analyses for 79. Randomized controlled trials number 92 (0.3%) as a share of all records on the topic. Randomized trials make up only a small share of this literature, so much of what is known comes from laboratory, observational or descriptive work.
Show the data
| Period | Records |
|---|---|
| through 1999 | 14,031 |
| 2000-2009 | 7,340 |
| 2010-2019 | 7,417 |
| 2020 onward | 5,417 |
Publication-type chart data
| Type | Records | Share |
|---|---|---|
| Reviews | 2,388 | 7.0% |
| Meta-analyses | 79 | 0.2% |
| Randomized controlled trials | 92 | 0.3% |
Method: PubMed query "Neuroblastoma"[MeSH Terms] with publication-type and date filters, retrieved 2026-10-29.
Reading the evidence in cancer and oncology
Cancer research spans very different kinds of evidence. Laboratory studies in cell lines and animal models reveal mechanisms but often do not translate to patients. Early-phase trials mainly test safety and dosing, while phase 3 randomized trials and their meta-analyses are the usual basis for treatment conclusions. Outcomes also differ in meaning: tumour shrinkage, progression-free survival and overall survival are not interchangeable, and screening studies can be distorted by lead-time and overdiagnosis. When reading a result, check the cancer type and stage studied, the comparison treatment, and which outcome was measured.
Evidence from open-access studies
These open-access studies come from PubMed’s best-match results for this heading (papers whose main topic it is), ranked here by citation count, with one recent addition. Each abstract is reproduced in full under its recorded open licence so that you can read the authors’ own words. They illustrate the literature; they are not a systematic summary of it. Citation counts measure attention, not reliability or clinical benefit.
| Study / authors | Year | Source label / design | Europe PMC citations | Licence / source |
|---|---|---|---|---|
| Longitudinal single-cell multiomic atlas of high-risk neuroblastoma reveals chemotherapy-induced tumor microenvironment rewiring | 2025 | Research article Design not recorded in structured metadata | 47 | cc by PMID 40229600 · DOI · PMC12081299 |
| The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial | 2025 | Research article Design not recorded in structured metadata | 26 | cc by PMID 39952926 · DOI · PMC11828896 |
| Reprogramming neuroblastoma by diet-enhanced polyamine depletion | 2025 | Research article Design not recorded in structured metadata | 23 | cc by PMID 40993392 · DOI · PMC12527938 |
| Dual lipid modulation overcomes ferroptosis resistance in high-risk neuroblastoma | 2026 | Research article Design not recorded in structured metadata | 10 | cc by PMID 41299087 · DOI · PMC13156318 |
Longitudinal single-cell multiomic atlas of high-risk neuroblastoma reveals chemotherapy-induced tumor microenvironment rewiring [1]
Abstract reading summary — source excerpts
- Opening excerpt
- High-risk neuroblastoma, a leading cause of pediatric cancer mortality, exhibits substantial intratumoral heterogeneity, contributing to therapeutic resistance.
- Closing excerpt
- These findings collectively reveal intrinsic and extrinsic regulators of therapy response in high-risk neuroblastoma.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (151 words)
High-risk neuroblastoma, a leading cause of pediatric cancer mortality, exhibits substantial intratumoral heterogeneity, contributing to therapeutic resistance. To understand tumor microenvironment evolution during therapy, we longitudinally profiled 22 patients with high-risk neuroblastoma before and after induction chemotherapy using single-nucleus RNA and ATAC sequencing and whole-genome sequencing. This revealed profound shifts in tumor and immune cell subpopulations after therapy and identified enhancer-driven transcriptional regulators of neuroblastoma neoplastic states. Poor outcome correlated with proliferative and metabolically active neoplastic states, whereas more differentiated neuronal-like states predicted better prognosis. Proportions of mesenchymal neoplastic cells increased after therapy and a high proportion correlated with a poorer chemotherapy response. Macrophages significantly expanded towards pro-angiogenic, immunosuppressive and metabolic phenotypes. We identified paracrine signaling networks and validated the HB-EGF-ERBB4 axis between macrophage and neoplastic subsets, which promoted tumor growth through the induction of ERK signaling. These findings collectively reveal intrinsic and extrinsic regulators of therapy response in high-risk neuroblastoma.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 40229600 · DOI · PMC12081299
The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial [2]
Abstract reading summary — source excerpts
- Opening excerpt
- In this single-arm, non-randomized, phase 2 trial (NCT03363373), 74 patients with relapsed/refractory high-risk neuroblastoma and residual disease in bone/bone marrow (BM) received naxitamab on Days 1, 3, and 5 (3 mg/kg/day) with granulocyte-macrophage colony-stimulating factor (Days -4 to 5) every 4 weeks, until complete response (CR) or partial response (PR) followed by 5 additional cycles every 4 weeks.
- Closing excerpt
- Overall, naxitamab demonstrated clinically meaningful efficacy with manageable safety in patients with residual neuroblastoma in bone/BM.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (203 words)
In this single-arm, non-randomized, phase 2 trial (NCT03363373), 74 patients with relapsed/refractory high-risk neuroblastoma and residual disease in bone/bone marrow (BM) received naxitamab on Days 1, 3, and 5 (3 mg/kg/day) with granulocyte-macrophage colony-stimulating factor (Days -4 to 5) every 4 weeks, until complete response (CR) or partial response (PR) followed by 5 additional cycles every 4 weeks. Primary endpoint in the prespecified interim analysis was overall response (2017 International Neuroblastoma Response Criteria). Among 26 responders (CR + PR) in the efficacy population (N = 52), 58% had refractory disease, and 42% had relapsed disease. Overall response rate (ORR) was 50% (95% CI: 36-64%), and CR and PR were observed in 38% and 12%, respectively. With the 95% CI lower limit for ORR exceeding 20%, the primary endpoint of overall response was met. Patients with evaluable bone disease had a 58% (29/50) bone compartment response (CR, 40%; PR, 18%). BM compartment response was 74% (17/23; CR, 74%). One-year overall survival and progression-free survival (secondary endpoints) were 93% (95% CI: 80-98%) and 35% (95% CI: 16-54%), respectively. Naxitamab-related Grade 3 adverse events included hypotension (58%) and pain (54%). Overall, naxitamab demonstrated clinically meaningful efficacy with manageable safety in patients with residual neuroblastoma in bone/BM.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 39952926 · DOI · PMC11828896
Reprogramming neuroblastoma by diet-enhanced polyamine depletion [3]
Abstract reading summary — source excerpts
- Opening excerpt
- Neuroblastoma is a highly lethal childhood tumour derived from differentiation-arrested neural crest cells1,2.
- Closing excerpt
- These results suggest that the genes of specific cellular programmes have evolved hallmark codon usage preferences that enable coherent translational rewiring in response to metabolic stresses, and that this process can be targeted to activate differentiation of paediatric cancers.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (186 words)
Neuroblastoma is a highly lethal childhood tumour derived from differentiation-arrested neural crest cells1,2. Like all cancers, its growth is fuelled by metabolites obtained from either circulation or local biosynthesis3,4. Neuroblastomas depend on local polyamine biosynthesis, and the inhibitor difluoromethylornithine has shown clinical activity5. Here we show that such inhibition can be augmented by dietary restriction of upstream amino acid substrates, leading to disruption of oncogenic protein translation, tumour differentiation and profound survival gains in the Th-MYCN mouse model. Specifically, an arginine- and proline-free diet decreases the amount of the polyamine precursor ornithine and enhances tumour polyamine depletion by difluoromethylornithine. This polyamine depletion causes ribosome stalling, unexpectedly specifically at codons with adenosine in the third position. Such codons are selectively enriched in cell cycle genes and low in neuronal differentiation genes. Thus, impaired translation of these codons, induced by combined dietary and pharmacological intervention, favours a pro-differentiation proteome. These results suggest that the genes of specific cellular programmes have evolved hallmark codon usage preferences that enable coherent translational rewiring in response to metabolic stresses, and that this process can be targeted to activate differentiation of paediatric cancers.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 40993392 · DOI · PMC12527938
Dual lipid modulation overcomes ferroptosis resistance in high-risk neuroblastoma [4]
Abstract reading summary — source excerpts
- Opening excerpt
- Ferroptosis-an iron-dependent form of cell death triggered by phospholipid peroxidation-has emerged as a promising therapeutic avenue in cancer treatment.
- Closing excerpt
- Together, these findings establish MUFA synthesis blockade and PUFA enrichment as a tumor-targeted, ferroptosis-enhancing strategy, and offer a nanomedicine-based therapeutic platform for high-risk NB.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (189 words)
Ferroptosis-an iron-dependent form of cell death triggered by phospholipid peroxidation-has emerged as a promising therapeutic avenue in cancer treatment. Although neuroblastoma (NB) has been identified as a ferroptosis susceptible cancer, our studies reveal striking heterogeneity in ferroptosis sensitivity across high-risk NB models. Through a targeted metabolic compound screen, we identified stearoyl-CoA desaturase 1 (SCD1)-a key enzyme in monounsaturated fatty acid (MUFA) synthesis-as a robust ferroptosis-sensitizing target. Genetic and pharmacological inhibition of SCD1 restored ferroptosis sensitivity in resistant NB cells. Notably, high SCD1 expression correlates with poor patient prognosis. Co-treatment with arachidonic acid (AA), a polyunsaturated fatty acid (PUFA), further enhanced ferroptotic cell death via increased PUFA/MUFA ratio. Nevertheless, neither baseline lipidomic profiles nor transcriptomes of key ferroptosis regulators reliably predicted ferroptosis sensitivity. To overcome AA's poor solubility, we engineered AA-loaded lipid nanoparticles (AA-LNPs), which selectively accumulated in high-risk NB tumors and synergized with SCD1 inhibition. This dual-sensitization strategy, termed LipidSens, significantly suppressed tumor growth and induced ferroptosis in cell-derived xenograft mouse models without systemic toxicity. Together, these findings establish MUFA synthesis blockade and PUFA enrichment as a tumor-targeted, ferroptosis-enhancing strategy, and offer a nanomedicine-based therapeutic platform for high-risk NB.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 41299087 · DOI · PMC13156318
Key reviews and meta-analyses
The most cited review-type papers among PubMed’s best-match results for this heading, listed with bibliographic details only. Follow the links for the full text and access terms.
- Research article Esthesioneuroblastoma: a meta-analysis and review [5]
- Research article Advancing therapy for neuroblastoma [6]
- Research article Molecular targeting therapies for neuroblastoma: Progress and challenges [7]
- Research article The SH-SY5Y human neuroblastoma cell line, a relevant in vitro cell model for investigating neurotoxicology in human: Focus on organic pollutants [8]
- Research article Recent advances in the developmental origin of neuroblastoma: an overview [9]
- Research article Neuroblastoma [10]
References
- Yu W, Biyik-Sit R, Uzun Y, Chen CH, Thadi A, Sussman JH, et al.. Longitudinal single-cell multiomic atlas of high-risk neuroblastoma reveals chemotherapy-induced tumor microenvironment rewiring. . 2025. PMID 40229600 · DOI · PMC12081299
- Mora J, Chan GCF, Morgenstern DA, Amoroso L, Nysom K, Faber J, et al.. The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial. . 2025. PMID 39952926 · DOI · PMC11828896
- Cherkaoui S, Turn CS, Yuan Y, Lu W, Yang L, McBride MJ, et al.. Reprogramming neuroblastoma by diet-enhanced polyamine depletion. . 2025. PMID 40993392 · DOI · PMC12527938
- Koeken I, Walravens M, Fernández-Acosta R, Van Hoyweghen R, Vintea I, Kong Y, et al.. Dual lipid modulation overcomes ferroptosis resistance in high-risk neuroblastoma. . 2026. PMID 41299087 · DOI · PMC13156318
- Dulguerov P, Allal AS, Calcaterra TC. Esthesioneuroblastoma: a meta-analysis and review. Lancet Oncol. 2001. PMID 11902539 · DOI
- Qiu B, Matthay KK. Advancing therapy for neuroblastoma. Nat Rev Clin Oncol. 2022. PMID 35614230 · DOI
- Zafar A, Wang W, Liu G, Wang X, Xian W, McKeon F, et al.. Molecular targeting therapies for neuroblastoma: Progress and challenges. Med Res Rev. 2021. PMID 33155698 · DOI · PMC7906923
- Lopez-Suarez L, Awabdh SA, Coumoul X, Chauvet C. The SH-SY5Y human neuroblastoma cell line, a relevant in vitro cell model for investigating neurotoxicology in human: Focus on organic pollutants. Neurotoxicology. 2022. PMID 35914637 · DOI
- Ponzoni M, Bachetti T, Corrias MV, Brignole C, Pastorino F, Calarco E, et al.. Recent advances in the developmental origin of neuroblastoma: an overview. J Exp Clin Cancer Res. 2022. PMID 35277192 · DOI · PMC8915499
- Chung C, Boterberg T, Lucas J, Panoff J, Valteau-Couanet D, Hero B, et al.. Neuroblastoma. Pediatr Blood Cancer. 2021. PMID 33818884 · DOI · PMC8785544
About this article
- Sources
- NLM Medical Subject Headings (definition, synonyms); NCBI PubMed (counts, identifiers); Europe PMC (open-access abstracts and citation counts).
- Verification
- Every reference was resolved against PubMed on 2026-10-31; title, year and DOI matched.
- Review state
- automated source verified; no human expert review has been recorded yet.
- Version
- 1, last updated 2026-10-29.
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Evidence strength
EvidenceHelix's computed view of the study designs behind this topic: how common meta-analyses, randomized trials and reviews are, how recent the literature is, and a plain-language reading. It describes the design mix of the literature. It is not a quality appraisal of any study, and it is not medical advice.