Trial reportNeuro-oncology2025
Phase 3 randomized trial of high-dose methotrexate for young children with high-risk embryonal brain tumors: A report from the Children's Oncology Group.
Trial report in Neuro-oncology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
11 citing papers in PubMed.
- Strategic roadmap for delivery of clinical trials in rare childhood central nervous system (CNS) tumours: a multi-stakeholder consensus.British journal of cancer · 2026Article
- Pediatric medulloblastoma.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Review
- Therapy and biomarker dependent progression-free survival in infant sonic hedgehog medulloblastoma: a multi-national retrospective cohort study.EClinicalMedicine · 2026Article
- Developing a core outcome set for the NeuroPoint Alliance Quality Outcomes Database Pediatric Tumor Surgery Registry: a modified Delphi study.Journal of neuro-oncology · 2026Article
- Radiation-sparing management of WNT medulloblastoma of early childhood with high dose chemotherapy: In response to Gupta et al.Journal of neuro-oncology · 2026Article
- Machine learning-based identification of extracellular matrix-related prognostic subtypes in SHH-activated medulloblastoma.Discover oncology · 2026Article
- Medulloblastoma: Current Standard of Care and Future Treatment Opportunities.Paediatric drugs · 2026Review
- The tumor microenvironment of medulloblastoma: from emerging biological insights to novel therapeutic targeting.Frontiers in oncology · 2026Review
- Response to: Bayesian -reappraisal of ACNS0332 and ACNS0334 strengthens -subgroup treatment effects in high-risk pediatric group 3 medulloblastoma.Neuro-oncology · 2025Article
- Bayesian reappraisal of ACNS0332 and ACNS0334 strengthens subgroup treatment effects in high-risk pediatric Group 3 medulloblastoma.Neuro-oncology · 2025Article
- Old Tools in a New Era: The Continued Relevance of Chemotherapy in Pediatric Neuro-Oncology.Current oncology (Toronto, Ont.) · 2025Review
Corrections and comments
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Authors and funding
18 authors.
Funding
Abstract
backgroundEmbryonal brain tumors are the leading cause of cancer death in young children.
methodsACNS0334 was a phase 3 randomized study evaluating high-dose methotrexate in young children < 36 months old with newly diagnosed high-risk embryonal brain tumors. Treatment included 3 cycles of induction chemotherapy with or without methotrexate followed by 3 cycles of high-dose consolidation chemotherapy with hematopoietic stem-cell infusion. Primary endpoint was complete response (CR) at the end of therapy. Secondary endpoints included a comparison of event-free survival (EFS) between arms and to historical controls. Molecular characterization was conducted retrospectively. Tests of significance were one-sided.
resultsOf 77 eligible patients, 59 with detectable disease were evaluated for response and 28 (47.5%) achieved CR, 15/30 (50%) treated with methotrexate compared to 13/29 (45%) without methotrexate (P = 0.35). For MB, CR was 12/19 (63%) with methotrexate compared to 6/20 (30%) without methotrexate (P = 0.039). Considering molecular diagnosis, all SHH MB (n = 11) were survivors. Five-year EFS was 70% (90% CI: 39.6-87.2) for 10 Group 3 MB with methotrexate versus 33.3% (90% CI: 15.0-52.9) for 15 without (P = 0.037). In other embryonal tumors, CR was 3/11 (27%) with methotrexate compared to 7/9 (78%) without (P = 0.99). No benefit with methotrexate was observed for Embryonal Tumor with Multilayered Rosettes (n = 14, EFS 20.0% [90% CI: 1.8-52.5] with methotrexate versus 33.3% [90% CI: 10.8-58.1] without, P = 0.58), or pineoblastoma (n = 9, EFS 16.7% [90% CI: 1.6-46.1] with methotrexate versus 0% without, P = 0.52).
conclusionsThe addition of methotrexate to intensive chemotherapy improved CR and EFS for young children with high-risk Group 3 MB, but not other diagnoses.
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