What is the evidence for fenfluramine in Dravet syndrome?

Five-year extended follow-up status of 10 patients with Dravet syndrome treated with fenfluramine

Ceulemans B, Schoonjans AS, Marchau F, Paelinck BP, Lagae L.

Epilepsia 2016; 57:e129-34.

Abstract

Dravet syndrome (DS) is a rare and therapy-resistant epilepsy syndrome. A retrospective analysis of add-on fenfluramine treatment in 12 patients with DS was published in 2012 and provided evidence of a meaningful long-term response. Herein we present the results of a subsequent 5-year prospective observation of this original cohort.

Ten patients with a mean current age of 24 years were followed prospectively from 2010 until 2014. The mean current dose of fenfluramine was 0.27 mg/kg/day, with a mean treatment duration of 16.1 years. Seizure frequency was derived from a seizure diary. Cardiac examinations and assessments of clinical effectiveness and adverse events were performed at least annually. Three patients were seizure-free for the entire 5 years, and an additional four patients experienced seizure-free intervals of at least 2 years.

Fenfluramine was generally well-tolerated. Two patients had mild (stable) valve thickening on the last echocardiography that was deemed clinically insignificant. No patient had any clinical or echocardiographic signs of pulmonary hypertension.

These findings support the long-term control of convulsive seizures by low-dose fenfluramine while being well tolerated in this cohort of patients with DS. After up to 27 years of treatment, no patient has developed any clinical signs or symptoms of cardiac valvulopathy or pulmonary hypertension.

 

You may also check out:

Low-dose fenfluramine significantly reduces seizure frequency in Dravet syndrome: a prospective study of a new cohort of patients

Schoonjans A, Paelinck BP, Marchau F, et al. Eur J Neurol 2016; doi: 10.1111/ene.13195. (Epub ahead of print).

Both references are now included in the neurochecklist:

Dravet syndrome: differentials and management

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Abstract link

By DEA - http://www.justice.gov/dea/programs/forensicsci/microgram/mg0609/mg0609.pdf, Public Domain, Link
By DEA – http://www.justice.gov/dea/programs/forensicsci/microgram/mg0609/mg0609.pdf, Public Domain, Link
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