Trial reportJournal of neuro-oncology2019
Infusion of 5-Azacytidine (5-AZA) into the fourth ventricle or resection cavity in children with recurrent posterior Fossa Ependymoma: a pilot clinical trial.
Trial report in Journal of neuro-oncology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 2 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 2 syntheses or guidelines pooled it, 27 citations in OpenAlex.
- Intrathecal drug delivery in the era of nanomedicine.Advanced drug delivery reviews · 2020Pooled it
- Ventricular Cerebrospinal Fluid Sampling in Pediatric Diffuse Midline Glioma Patients: Institutional Experience and Review of the Literature.Frontiers in pediatrics · 2020Pooled it
- Targeting aberrant DNA methylation across subtypes of pediatric high-grade glioma inhibits tumor growth and RUNX3 represents a targetable methylated tumor suppressor.Cell communication and signaling : CCS · 2026Article
- Ependymoma: Advances in Systems Treatment Strategies.Current neurology and neuroscience reports · 2025Review
- Individualized therapeutic approaches for relapsed and refractory pediatric ependymomas: a single institution experience.Journal of neuro-oncology · 2025Article
- Review
- Advances in the Repurposing and Blood-Brain Barrier Penetrance of Drugs in Pediatric Brain Tumors.Cancers · 2025Review
- H3K27-Altered Diffuse Midline Glioma of the Brainstem: From Molecular Mechanisms to Targeted Interventions.Cells · 2024Review
- Direct Administration of Chemotherapy and Other Agents into the Fourth Ventricle to Treat Recurrent Malignant Brain Tumors in Children.Advances and technical standards in neurosurgery · 2024Review
- Chemotherapy in pediatric brain tumor and the challenge of the blood-brain barrier.Cancer medicine · 2023Review
- Carbon Dots in Treatment of Pediatric Brain Tumors: Past, Present, and Future Directions.International journal of molecular sciences · 2023Review
- Leptomeningeal dissemination in pediatric brain tumors.Neoplasia (New York, N.Y.) · 2023Article
- Advances on Epigenetic Drugs for Pediatric Brain Tumors.Current neuropharmacology · 2023Review
- Review
- Polycomb repressive complex 2 in the driver's seat of childhood and young adult brain tumours.Trends in cell biology · 2021Review
- Identification of key methylation differentially expressed genes in posterior fossa ependymoma based on epigenomic and transcriptome analysis.Journal of translational medicine · 2021Article
- High-dose MTX110 (soluble panobinostat) safely administered into the fourth ventricle in a nonhuman primate model.Journal of neurosurgery. Pediatrics · 2020Article
- Current and Emerging Methods of Management of Ependymoma.Current oncology reports · 2019Review
- Plasma and cerebrospinal fluid pharmacokinetics of the DNA methyltransferase inhibitor, 5-azacytidine, alone and with inulin, in nonhuman primate models.Neuro-oncology advancesArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundDNA methylation inhibitors are logical therapeutic candidates for ependymomas originating in the posterior fossa of the brain. Our objective was to test the safety of infusing 5-Azacytidine (5-AZA), a DNA methylation inhibitor, directly into cerebrospinal fluid (CSF) spaces of the fourth ventricle or tumor resection cavity in children with recurrent ependymoma originating in the posterior fossa. MATERIALS AND
methodsIn patients with recurrent ependymoma whose disease originated in the posterior fossa, a maximal safe subtotal tumor resection was performed. At the conclusion of the tumor resection, a catheter was surgically placed into the fourth ventricle or tumor resection cavity and attached to a ventricular access device. CSF flow from the posterior fossa to the sacrum was confirmed by CINE phase contrast magnetic resonance imaging (MRI) postoperatively. 12 consecutive weekly 10 milligram (mg) infusions of 5-Azacytidine (AZA) were planned. Disease response was monitored with MRI scans and CSF cytology.
resultsSix patients were enrolled. One patient was withdrawn prior to planned 5-AZA infusions due to surgical complications after tumor resection. The remaining five patients received 8, 12, 12, 12, and 12 infusions, respectively. There were no serious adverse events or new neurological deficits attributed to 5-AZA infusions. All five patients with ependymoma who received 5-AZA infusions had progressive disease. Two of the five patients, however, were noted to have decrease in the size of at least one intraventricular lesion.
conclusion5-AZA can be infused into the fourth ventricle or posterior fossa tumor resection cavity without causing neurological toxicity. Future studies with higher doses and/or increased dosing frequency are warranted.
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