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Title
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Successful use of fenfluramine as an add-on treatment for Dravet syndrome
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Author
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Abstract
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| 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possible cardiac adverse effects (valve thickening, pulmonary hypertension) associated with use of fenfluramine, it was withdrawn from the market in 2001.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 335 years. The mean dosage of fenfluramine was 0.34 (0.120.90) mg/kg/day. Exposure duration to fenfluramine ranged from 119 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 (119) 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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Language
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English
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Source (journal)
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Epilepsia. - Boston, Mass.
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Publication
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Boston, Mass.
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2012
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ISSN
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0013-9580
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DOI
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10.1111/J.1528-1167.2012.03495.X
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Volume/pages
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53
:7
(2012)
, p. 1131-1139
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ISI
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000305944600008
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Full text (Publisher's DOI)
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Full text (publisher's version - intranet only)
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