ArticleNeuro-oncology2015
Phase I study of 5-fluorouracil in children and young adults with recurrent ependymoma.
Article in Neuro-oncology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 1 of them a synthesis that pooled it.
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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, 1 synthesis or guideline pooled it, 34 citations in OpenAlex.
- Clinical Pharmacogenetics Implementation Consortium (CPIC) Guideline for Dihydropyrimidine Dehydrogenase Genotype and Fluoropyrimidine Dosing: 2017 Update.Clinical pharmacology and therapeutics · 2018Guideline
- A brief history of ependymoma.Neuro-oncology · 2026Review
- Developing an advanced risk stratification model for pediatric intracranial ependymoma based on the prospective trial E-HIT2000 and subsequent registries.Neuro-oncology · 2026Article
- Advances in molecular prognostication and treatments in ependymoma.Journal of neuro-oncology · 2025Review
- Development and Application of Physiologically-Based Pharmacokinetic Model to Predict Systemic and Organ Exposure of Colorectal Cancer Drugs.Pharmaceutics · 2025Article
- Optimizing outcomes in intracranial ependymoma: a contemporary review.Frontiers in oncology · 2025Review
- Development of Chromosome 1q+ Specific Treatment for Highest Risk Pediatric Posterior Fossa Ependymoma.Clinical cancer research : an official journal of the American Association for Cancer Research · 2024Article
- Handling Incomplete or Late-Onset Toxicities in Early-Phase Dose-Finding Clinical Trials: Current Practice and Future Prospects.JCO precision oncology · 2024Review
- Anticancer therapy-induced adverse drug reactions in children and preventive and control measures.Frontiers in pharmacology · 2024Review
- Review
- Utilizing preclinical models to develop targeted therapies for rare central nervous system cancers.Neuro-oncology · 2021Review
- Review
- YAP1/TAZ drives ependymoma-like tumour formation in mice.Nature communications · 2020Article
- Intracranial ependymomas: molecular insights and translation to treatment.Brain pathology (Zurich, Switzerland) · 2020Review
- Central nervous system ependymoma: clinical implications of the new molecular classification, treatment guidelines and controversial issues.Clinical & translational oncology : official publication of the Federation of Spanish Oncology Societies and of the National Cancer Institute of Mexico · 2019Review
- 5-Fluorouracil treatment induces characteristic T>G mutations in human cancer.Nature communications · 2019Article
- Identification of FDA-Approved Oncology Drugs with Selective Potency in High-Risk Childhood Ependymoma.Molecular cancer therapeutics · 2018Article
- The current consensus on the clinical management of intracranial ependymoma and its distinct molecular variants.Acta neuropathologica · 2017Article
- Translational Pharmacokinetic-Pharmacodynamic Modeling and Simulation: Optimizing 5-Fluorouracil Dosing in Children With Pediatric Ependymoma.CPT: pharmacometrics & systems pharmacology · 2016Article
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
Abstract
backgroundWe report a phase I study to examine the pharmacokinetics, safety, and recommended dosage of weekly intravenous bolus 5-fluorouracil (5-FU) in children and young adults with recurrent ependymoma.
methodsPatients 22 years of age or less with recurrent ependymoma were treated with bolus dosage 5-FU weekly for 4 weeks followed by a 2-week rest period, defining one cycle. Patients could continue on therapy for 16 cycles. The starting 5-FU dosage was 500 mg/m(2). Dose-limiting toxicity was determined after one cycle. Patients were initially enrolled according to a rolling-6 design; subsequent dose re-escalation phase was based on a 3 + 3 design.
resultsWe treated patients at 400 (n = 6), 500 (n = 15), and 650 (n = 5) mg/m(2), with de-escalation due to toxicity. Twenty-three of twenty-six patients enrolled were evaluable. Five patients experienced grade 4 neutropenia (n = 2: 650 mg/m(2); n = 3: 500 mg/m(2)). One patient experienced grade 3 diarrhea. At 500 mg/m(2), the median 5-FU maximal concentration, AUC0-∞, and alpha half-life were 825 µM, 205 µM × h, and 9.9 min, respectively. Interim analysis revealed an association between hematologic toxicity and prior number of chemotherapeutic regimens (P = .03). The study was amended to re-escalate the dosage in a less heavily pretreated cohort of patients.
conclusionsThese phase I clinical data provide initial pharmacokinetic parameters to describe i.v. bolus 5-FU disposition in children with recurrent ependymoma. Tumor exposures effective in preclinical testing can be achieved with tolerable bolus dosages in patients. Bolus 5-FU is well tolerated and possesses antitumor activity.
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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.