Fenfluramine as antiseizure medication for epilepsy.


Journal

Developmental medicine and child neurology
ISSN: 1469-8749
Titre abrégé: Dev Med Child Neurol
Pays: England
ID NLM: 0006761

Informations de publication

Date de publication:
08 2021
Historique:
accepted: 05 01 2021
pubmed: 11 2 2021
medline: 12 10 2021
entrez: 10 2 2021
Statut: ppublish

Résumé

Fenfluramine hydrochloride has classically been described as acting pharmacologically through a serotonergic mechanism. Therefore, it was initially used as an anorectic drug, given that impaired serotonin homeostasis may be associated with increased food intake. Although positive results were documented, cardiovascular concerns resulted in its temporary withdrawal. Nevertheless, a novel role in patients with epilepsy was later suggested by isolated clinical observations. The wide application of genetic testing allowed the classification (predominantly as Dravet syndrome) of patients in whom benefit was seen, while with the development of zebrafish models, its antiepileptic properties were confirmed at a molecular level. Data from randomized clinical trials have shown a beneficial effect of fenfluramine, as an adjunct therapy, on seizure control for children with Dravet syndrome, though there is still uncertainty about the impact on neurodevelopment in these patients. No signs of heart valve disease have been documented to date. Long-term and appropriately designed clinical studies will verify whether fenfluramine is a therapeutic agent of high importance, living up to the promise shown so far. What this paper adds Fenfluramine is a very promising repurposed therapy specifically for seizures in Dravet syndrome. The long-term effect of fenfluramine on neurodevelopmental prognosis requires further investigation.

Identifiants

pubmed: 33565102
doi: 10.1111/dmcn.14822
doi:

Substances chimiques

Anticonvulsants 0
Fenfluramine 2DS058H2CF

Types de publication

Journal Article Research Support, Non-U.S. Gov't Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

899-907

Subventions

Organisme : Department of Health
Pays : United Kingdom

Informations de copyright

© 2021 The Authors. Developmental Medicine & Child Neurology published by John Wiley & Sons Ltd on behalf of Mac Keith Press.

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Auteurs

Maria Gogou (M)

Department of Neurology, Great Ormond Street Hospital for Children NHS Trust, London, UK.

J Helen Cross (JH)

Department of Neurology, Great Ormond Street Hospital for Children NHS Trust, London, UK.
Developmental Neurosciences, University College London NIHR BRC Great Ormond Street Institute of Child Health, London, UK.

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