ArticleEpilepsia open2022
An examination of the efficacy and safety of fenfluramine in adults, children, and adolescents with Dravet syndrome in a real-world practice setting: A report from the Fenfluramine European Early Access Program.
Article in Epilepsia open, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 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
21 citing papers in PubMed, 2 syntheses or guidelines pooled it, 39 citations in OpenAlex.
- [Advances and guidance in the treatment of drug-resistant epilepsy: a review by the Andalusian Epilepsy Society of the new drugs cenobamate, fenfluramine and cannabidiol].Revista de neurologia · 2024Guideline
- Dravet syndrome: A systematic literature review of the illness burden.Epilepsia open · 2023Pooled it
- Cocreation of Digital Outcome Measures for Dravet Syndrome: Multistage Co-Design Feasibility Study.Journal of participatory medicine · 2026Article
- Observational
- Efficacy and safety of fenfluramine in Dravet syndrome: The impact of patient clinical characteristics.Epilepsia open · 2026Article
- DECREASE study: A European pool-analysis of patients with significant reductions in concomitant antiseizure medications in cenobamate Early Access Programs.Epilepsia open · 2026Article
- Ameliorating Seizures in Dravet Syndrome: A Review of Newly Approved and Investigational Drugs, RNA and Gene-Based Therapies.CNS drugs · 2026Review
- Safety of Drugs Used in Difficult-to-Treat Epileptic Syndromes: A Disproportionality Analysis Using the Eudravigilance Database.Pharmaceuticals (Basel, Switzerland) · 2025Article
- Monogenic Epilepsies in Adult Epilepsy Clinics and Gene-Driven Approaches to Treatment.Current neurology and neuroscience reports · 2025Review
- Expert-Agreed Practical Recommendations on the Use of Fenfluramine in Developmental and Epileptic Encephalopathies Based on Clinical Experience and Literature Review.Neurology and therapy · 2025Review
- Fenfluramine treatment for Dravet syndrome: Long term real-world analysis demonstrates safety and reduced health care burden.Epilepsia · 2025Article
- Recent Advances in Pharmacologic Treatments of Drug-Resistant Epilepsy: Breakthrough in Sight.CNS drugs · 2024Review
- Fenfluramine treatment in pediatric patients with Dravet syndrome reduces seizure burden and overall healthcare costs: A retrospective and observational real-world study.Epilepsia open · 2024Observational
- Practical considerations for the use of fenfluramine to manage patients with Dravet syndrome or Lennox-Gastaut syndrome in clinical practice.Epilepsia open · 2024Review
- Developmental and epileptic encephalopathies.Nature reviews. Disease primers · 2024Review
- New epilepsy therapies in development.Nature reviews. Drug discovery · 2024Review
- Review
- Black-Box Warnings of Antiseizure Medications: What is Inside the Box?Pharmaceutical medicine · 2023Review
- Pharmacological diversity amongst approved and emerging antiseizure medications for the treatment of developmental and epileptic encephalopathies.Therapeutic advances in neurological disorders · 2023Review
- Article
Corrections and comments
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Authors and funding
12 authors at 11 institutions in 6 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo examine the efficacy and safety of fenfluramine in patients with Dravet syndrome (DS) in three age groups: <6, 6-17, and ≥18 years old, treated in a real-world setting.
methodsPatients with DS were treated with fenfluramine in the European Union Early Access Program (EAP). Following a 28-day baseline period to establish the pretreatment monthly convulsive seizure frequency (MCSF), fenfluramine was started at a dose chosen by the treating physician and gradually titrated based on efficacy and tolerability up to a maximum of 0.7 mg/kg/day. Seizure incidence was recorded in a written diary, and adverse events (AEs) were reported at each patient visit. Cardiovascular safety was assessed by transthoracic echocardiography before treatment started and at least every 6 months thereafter.
resultsA total of 149 patients have enrolled in the EAP and 63 were <6 years old, 62 were 6-17 years old, and 24 were ≥18 years old. After 3 months of treatment 62%, 53%, and 50% of patients demonstrated ≥75% reduction in MCSF in the <6, 6-17, and ≥18-year-old groups, respectively. This pattern of response was sustained through 12 months of treatment with 55%, 46%, and 80% of the <6, 6-17, and ≥18-year-old groups, respectively, experiencing a ≥75% reduction in MCSF. Most common AEs were loss of appetite (21%) and somnolence (16%). No valvular heart disease or pulmonary artery hypertension was observed. SIGNIFICANCE: The magnitude, consistency, and durability of the response to add-on fenfluramine is consistent across age groups in patients with Dravet syndrome.
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Registered trials
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 Socratic graph.