ArticleEpilepsia2021
Effect of fenfluramine on convulsive seizures in CDKL5 deficiency disorder.
Article in Epilepsia, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
28 citing papers in PubMed, 2 syntheses or guidelines pooled it, 37 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
- Optimal dose of fenfluramine in adjuvant treatment of drug-resistant epilepsy: evidence from randomized controlled trials.Frontiers in neurology · 2024Pooled it
- Effect of Fenfluramine on convulsive seizures and rescue medication usage: A case series of adults within the Lennox-Gastaut syndrome spectrum.Epilepsy & behavior reports · 2026Article
- Fenfluramine in Rett syndrome: A multidimensional clinical study.Epilepsia open · 2026Article
- Fenfluramine for seizures in Dravet syndrome and Lennox-Gastaut syndrome: Mechanisms, clinical evidence, safety, and practical integration.Epilepsy & behavior reports · 2026Review
- Efficacy and safety of fenfluramine in Dravet syndrome: The impact of patient clinical characteristics.Epilepsia open · 2026Article
- Severe Cutaneous Adverse Reactions Associated With Newer-Generation Antiseizure Medications: A Real-World Pharmacovigilance Study Based on FAERS and JADER.CNS neuroscience & therapeutics · 2026Article
- Fenfluramine: an effective treatment for developmental epileptic encephalopathies beyond Dravet and Lennox-Gastaut Syndromes.Journal of neurology · 2025Article
- Precision Therapeutics in Lennox-Gastaut Syndrome: Targeting Molecular Pathophysiology in a Developmental and Epileptic Encephalopathy.Children (Basel, Switzerland) · 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
- Women With Genetic Epilepsies.Neurology. Genetics · 2025Review
- X-Linked Epilepsies: A Narrative Review.International journal of molecular sciences · 2024Review
- Current state of the epilepsy drug and device pipeline.Epilepsia · 2024Review
- Current Overview of CDKL-5 Deficiency Disorder Treatment.Pediatric reports · 2024Article
- Drug repurposing in Rett and Rett-like syndromes: a promising yet underrated opportunity?Frontiers in medicine · 2024Article
- Fenfluramine: a plethora of mechanisms?Frontiers in pharmacology · 2023Review
- Reintroducing Fenfluramine as a Treatment for Seizures: Current Knowledge, Recommendations and Gaps in Understanding.Neuropsychiatric disease and treatment · 2023Review
- Fenfluramine: A Review of Pharmacology, Clinical Efficacy, and Safety in Epilepsy.Children (Basel, Switzerland) · 2022Review
- In vitro evaluation of fenfluramine and norfenfluramine as victims of drug interactions.Pharmacology research & perspectives · 2022Review
- In vitro evaluation suggests fenfluramine and norfenfluramine are unlikely to act as perpetrators of drug interactions.Pharmacology research & perspectives · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
CDKL5 deficiency disorder (CDD) is an X-linked pharmacoresistant neurogenetic disorder characterized by global developmental delays and uncontrolled seizures. Fenfluramine (FFA), an antiseizure medication (ASM) indicated for treating convulsive seizures in Dravet syndrome, was assessed in six patients (five female; 83%) with CDD whose seizures had failed 5-12 ASMs or therapies. Median age at enrollment was 6.5 years (range: 2-26 years). Mean FFA treatment duration was 5.3 months (range: 2-9 months) at 0.4 mg/kg/day (n = 2) or 0.7 mg/kg/day (n = 4; maximum: 26 mg/day). One patient had valproate added for myoclonic seizures. The ASM regimens of all other patients were stable. Among five patients with tonic-clonic seizures, FFA treatment resulted in a median 90% reduction in frequency (range: 86%-100%). Tonic seizure frequency was reduced by 50%-60% in two patients with this seizure type. One patient experienced fewer myoclonic seizures; one patient first developed myoclonic seizures on FFA, which were controlled with valproate. Adverse events were reported in two patients. The patient with added valproate experienced lethargy; one patient had decreased appetite and flatus. No patient developed valvular heart disease or pulmonary arterial hypertension. Our preliminary results suggest that FFA may be a promising ASM for CDD. Randomized clinical trials are warranted.
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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 OpenQuestion graph.