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Successful use of fenfluramine as an add‐on treatment for Dravet syndrome
Open accessAbstract
Summary: Purpose: Despite the development of new antiepileptic drugs, Dravet syndrome frequently remains therapy resistant and is a catastrophic epilepsy syndrome. Fenfluramine is an amphetamine‐like drug that has been used in the past as a part of antiobesity treatments. Because of the possible cardiac adverse effects (valve thickening, pulmonary hypertension) associated with use of fenfluramine, it was withdrawn from the market in 2001. In Belgium, a Royal Decree permitted examination of the potential anticonvulsive effects of fenfluramine in a clinical trial consisting of a small group of patients diagnosed with Dravet syndrome. Methods: Herein, we report 12 patients, 7 female and 5 male, with a genetically proven (11 of 12) diagnosis of Dravet syndrome who received fenfluramine as add‐on therapy. Key Findings: Their ages at their last evaluation ranged from 3–35 years. The mean dosage of fenfluramine was 0.34 (0.12–0.90) mg/kg/day. Exposure duration to fenfluramine ranged from 1–19 years. Seven of the patients who were still receiving the fenfluramine treatment at the time of the last visit had been seizure‐free for at least 1 year. In total, patients had been seizure‐free for a mean of 6 (1–19) years. In seven patients, the fenfluramine treatment was interrupted once during the follow‐up; seizures reappeared in three of the seizure‐free patients. Subsequent reintroduction of fenfluramine controlled the seizures in these three patients again. Only two patients exhibited a mild thickening of one or two cardiac valves without clinical significance. Significance: Compared with a recent long‐term follow‐up series in which a maximum of 16% of patients with Dravet syndrome were seizure‐free, our result of 70% of patients with Dravet syndrome remaining seizure‐free is noteworthy. Given the limitations of this observational study, a larger prospective study should be undertaken to confirm these promising results.
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“Identification of the efficacy of fenfluramine in Dravet syndrome followed the observation that it was particularly beneficial in children with drug-resistant, self-induced photosensitive epilepsy 195 , resulting in seizure freedom in 7 of 12 patients with Dravet syndrome in one study 196 .”
From Developmental and epileptic encephalopathies (2024) · cites this paper as [196]
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Cites
Proposal for Revised Clinical and Electroencephalographic Classification of Epileptic Seizures
(1981) · Epilepsia · doi:10.1111/j.1528-1157.1981.tb06159.x · Cited by 3,433
De Novo Mutations in the Sodium-Channel Gene SCN1A Cause Severe Myoclonic Epilepsy of Infancy
Claes, L., Del-Favero, J., Ceulemans, B., Lagae, L., Van Broeckhoven, C., & De Jonghe, P. (2001) · The American Journal of Human Genetics · doi:10.1086/320609 · Cited by 1,292
Self-citationEpileptic Syndromes in Infancy, Childhood and Adolescence
Stephenson, J. B. P. (2004) · European Journal of Paediatric Neurology · doi:10.1016/j.ejpn.2004.03.001 · Cited by 685
The core Dravet syndrome phenotype
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The spectrum of SCN1A-related infantile epileptic encephalopathies
Harkin, L. A., McMahon, J. M., Iona, X., Dibbens, L. M., Pelekanos, J. T., Zuberi, S. M., Sadleir, L. G., Andermann, E., Gill, D. S., Farrell, K., et al. (2007) · Brain · doi:10.1093/brain/awm002 · Cited by 530
The genetics of Dravet syndrome
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Dravet syndrome: The long‐term outcome
Genton, P., Velizarova, R., & Dravet, C. (2011) · Epilepsia · doi:10.1111/j.1528-1167.2011.03001.x · Cited by 266
LES EPILEPSIES GRAVES DE L'ENFANT.
Dravet, C. (1978) · Cited by 240
A long‐term follow‐up study of Dravet syndrome up to adulthood
Akiyama, M., Kobayashi, K., Yoshinaga, H., & Ohtsuka, Y. (2010) · Epilepsia · doi:10.1111/j.1528-1167.2009.02466.x · Cited by 192
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Symonds, J. D., Zuberi, S. M., Stewart, K., McLellan, A., O‘Regan, M., MacLeod, S., Jollands, A., Joss, S., Kirkpatrick, M., Brunklaus, A., et al. (2019) · Brain · doi:10.1093/brain/awz195 · Cited by 459
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Self-citationThe Pharmacoresistant Epilepsy: An Overview on Existent and New Emerging Therapies
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Nabbout, R., Mistry, A., Zuberi, S., Villeneuve, N., Gil-Nagel, A., Sanchez-Carpintero, R., Stephani, U., Laux, L., Wirrell, E., Knupp, K., et al. (2020) · JAMA Neurology · doi:10.1001/jamaneurol.2019.4113 · Cited by 251
Metadata
- Authors
- Ceulemans, B., Boel, M., Leyssens, K., Van Rossem, C., Neels, P., Jorens, P. G., & Lagae, L.
- Affiliations
- University of Antwerp (BE)
- KU Leuven (BE)
- Corresponding author
- Ceulemans, Berten
- Venue
- Epilepsia
- Publisher
- Wiley
- Published
- 2012-05-03
- Volume
- 53
- Issue
- 7
- Pages
- 1131-1139
- ISSN
- 0013-9580 (ISSN-L)
- 1528-1167
- OpenAlex
- W1926532831
- Cited by
- 210
- References
- 51
- Citations by year
- 2026: 7 · 2025: 10 · 2024: 18 · 2023: 13 · 2022: 20 · 2021: 28 · 2020: 27 · 2019: 23 · 2018: 18 · 2017: 13 · 2016: 15 · 2015: 8 · 2014: 5 · 2013: 3 · 2012: 1
- FWCI
- 3.88
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Epilepsia (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Clinical Neurology › Epilepsy research and treatment
- Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Pharmacological Effects and Toxicity Studies
- Health Sciences › Medicine › Physiology (medical) › Diet and metabolism studies
- Keywords
- Dravet syndrome, fenfluramine, adjunctive therapy, seizure freedom, drug repurposing, amphetamine-type stimulants, pulmonary hypertension, drug withdrawal, seizure recurrence, long-term follow-up
- MeSH terms
- Adolescent, Adult, Anticonvulsants, Child, Preschool, Electroencephalography, Epilepsies, Myoclonic, Female, Fenfluramine, Follow-Up Studies, Humans, Male, Selective Serotonin Reuptake Inhibitors, Young Adult
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