ArticleNeuro-oncology2020
Bridging the treatment gap in infant medulloblastoma: molecularly informed outcomes of a globally feasible regimen.
Article in Neuro-oncology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.
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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
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 21 citations in OpenAlex.
- Neurodevelopmental assessments used to measure preschoolers' cognitive development in Latin America: a systematic review.Journal of pediatric psychology · 2024Pooled it
- Therapeutic radiation drives leptomeningeal dissemination of medulloblastoma through an innate immune process.Developmental cell · 2025Article
- Therapeutic approaches for targeting the pediatric brain tumor microenvironment.Drug delivery and translational research · 2025Review
- Medulloblastoma: biology and immunotherapy.Frontiers in immunology · 2025Review
- A single-center experience of central nervous system tumors in children under three years old.Frontiers in pediatrics · 2024Article
- The importance of basic and translational research in caring for children with malignant solid tumors in Latin America.Revista panamericana de salud publica = Pan American journal of public health · 2024Review
- Evolution of Systemic Therapy in Medulloblastoma Including Irradiation-Sparing Approaches.Diagnostics (Basel, Switzerland) · 2023Review
- Outcomes of a radiation sparing approach in medulloblastoma by subgroup in young children: an institutional review.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2023Article
- Spotlighting cellular therapies to advance the treatment of medulloblastoma.Neuro-oncology · 2023Article
- Cellular immunotherapy for medulloblastoma.Neuro-oncology · 2023Review
- Brain and Spinal Cord Tumors of Embryonic Origin.Advances in experimental medicine and biology · 2023Article
- Article
- Subgroup and subtype-specific outcomes in adult medulloblastoma.Acta neuropathologica · 2021Article
- Craniospinal irradiation as part of re-irradiation for children with recurrent medulloblastoma.Journal of neuro-oncology · 2021Article
Corrections and comments
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Authors and funding
13 authors at 4 institutions in 3 countries.
Funding
Abstract
backgroundInfant medulloblastoma represents an enormous challenge in neuro-oncology, due to their simultaneous high-risk of recurrence and high risk of severe neurodevelopmental sequelae with craniospinal irradiation. Currently infant medulloblastoma are treated with intensified protocols, either comprising intraventricular methotrexate or autologous transplant, both of which carry significant morbidity and are not feasible in the majority of the world. We sought to evaluate the molecular predictors of outcome in a cohort of infants homogeneously treated with induction chemotherapy, focal radiation and maintenance chemotherapy.
methodsIn a retrospective analysis, 29 young children treated with a craniospinal irradiation sparing strategy from Hospital Garrahan in Buenos Aires were profiled using Illumina HumanMethylationEPIC arrays, and correlated with survival.
resultsTwenty-nine children (range, 0.3-4.6 y) were identified, comprising 17 sonic hedgehog (SHH), 10 Group 3/4, and 2 non-medulloblastomas. Progression-free survival (PFS) across the entire cohort was 0.704 (95% CI: 0.551-0.899). Analysis by t-distributed stochastic neighbor embedding revealed 3 predominant groups, SHHβ, SHHγ, and Group 3. Survival by subtype was highly prognostic with SHHγ having an excellent 5-year PFS of 100% (95% CI: 0.633-1) and SHHβ having a PFS of 0.56 (95% CI: 0.42-1). Group 3 had a PFS of 0.50 (95% CI: 0.25-1). Assessment of neurocognitive outcome was performed in 11 patients; the majority of survivors fell within the low average to mild intellectual disability, with a median IQ of 73.5.
conclusionsWe report a globally feasible and effective strategy avoiding craniospinal radiation in the treatment of infant medulloblastoma, including a robust molecular correlation along with neurocognitive outcomes.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.