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Successful use of Fenfluramine as add-on treatment in Dravet syndrome: A three years prospective follow-up
Building: Bourbon Cataratas Convention Centre, Foz do Iguaçu
Room: Cataratas II
Date: 2014-05-05 03:30 PM – 03:45 PM
Last modified: 2014-02-09
Abstract
Purpose:
Evaluation of efficacy of fenfluramine as an add-on anti-epileptic drug in Dravet syndrome.
Methods:
Dravet syndrome remains, notwithstanding the introduction of newer anti-epileptic drugs, a therapy resistant and catastrophic epilepsy syndrome.
Fenfluramine is an amphetamine-like drug often used in the past as part of anti-obesity treatment. Because of possible cardiac side-effects (valve thickening, pulmonary hypertension), it has been withdrawn from the market in the US and Europe. In Belgium, it was allowed to study its potential anti-convulsive effect in a small group of well-defined patients with Dravet syndrome.
Results:
Twelve patients, seven girls and five boys, with a genetically proven (11/12) diagnosis of Dravet syndrome received add-on therapy with fenfluramine.
The retrospective data were published in 2012 (Ceulemans et al, Epilepsia 2012 Jul;53(7):1131-9). We now present the results of 3 years prospective follow-up of ten patients of this group, including two new patients Children were carefully monitored for reduction of seizure frequency and cardiac side effects. Mean age ranged from 3 - 35 years. Dosage of fenfluramine was 0,34 (0,12-0,90) mg/kg/d. All patients had at least a >75% of reduction of seizures in the study period of 3 years. Eight out of twelve were seizure free for at least one year. Three were seizure free for the complete trial period.
In four patients a mild thickening of 1 or 2 cardiac valves, without clinical significance, was seen. The mitral valve seems to be the most frequently damaged valve. Only one patient had valve thickening on each ultrasound cardiac examination. Loss of appetite (4 patients) seems not a major problem.
Conclusions:
These new prospective data confirm the promising results from the long term retrospective study of fenfluramine as add on anti epileptic medication in Dravet syndrome. Eight out of twelve patients (66%) remained seizure free for at least for one year. The mitral valve seems to be the most vulnerable for valve thickening by this drug. Realising that this observational study has limitations, a larger prospective study is under way, Now the drug has recently be granted as orphan drug designation in Europe and the US.
Evaluation of efficacy of fenfluramine as an add-on anti-epileptic drug in Dravet syndrome.
Methods:
Dravet syndrome remains, notwithstanding the introduction of newer anti-epileptic drugs, a therapy resistant and catastrophic epilepsy syndrome.
Fenfluramine is an amphetamine-like drug often used in the past as part of anti-obesity treatment. Because of possible cardiac side-effects (valve thickening, pulmonary hypertension), it has been withdrawn from the market in the US and Europe. In Belgium, it was allowed to study its potential anti-convulsive effect in a small group of well-defined patients with Dravet syndrome.
Results:
Twelve patients, seven girls and five boys, with a genetically proven (11/12) diagnosis of Dravet syndrome received add-on therapy with fenfluramine.
The retrospective data were published in 2012 (Ceulemans et al, Epilepsia 2012 Jul;53(7):1131-9). We now present the results of 3 years prospective follow-up of ten patients of this group, including two new patients Children were carefully monitored for reduction of seizure frequency and cardiac side effects. Mean age ranged from 3 - 35 years. Dosage of fenfluramine was 0,34 (0,12-0,90) mg/kg/d. All patients had at least a >75% of reduction of seizures in the study period of 3 years. Eight out of twelve were seizure free for at least one year. Three were seizure free for the complete trial period.
In four patients a mild thickening of 1 or 2 cardiac valves, without clinical significance, was seen. The mitral valve seems to be the most frequently damaged valve. Only one patient had valve thickening on each ultrasound cardiac examination. Loss of appetite (4 patients) seems not a major problem.
Conclusions:
These new prospective data confirm the promising results from the long term retrospective study of fenfluramine as add on anti epileptic medication in Dravet syndrome. Eight out of twelve patients (66%) remained seizure free for at least for one year. The mitral valve seems to be the most vulnerable for valve thickening by this drug. Realising that this observational study has limitations, a larger prospective study is under way, Now the drug has recently be granted as orphan drug designation in Europe and the US.
Keywords
Dravet syndrome; fenfluramine
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