Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2017 Feb;24(2):309-314.
doi: 10.1111/ene.13195. Epub 2016 Oct 28.

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

A Schoonjans  1 , B P Paelinck  2 , F Marchau  3 , B Gunning  4 , A Gammaitoni  5 , B S Galer  5 , L Lagae  6 , B Ceulemans  1
Affiliations

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

A Schoonjans et al. Eur J Neurol. 2017 Feb.

Abstract

Background and purpose: Dravet syndrome (DS) is a severe, drug-resistant epilepsy. Fenfluramine has been reported to have a long-term clinically meaningful anticonvulsive effect in patients with DS.

Methods: This prospective, open-label study assessed the safety and effectiveness of low-dose fenfluramine in a new cohort of patients with DS. Following a 3-month baseline period, fenfluramine was added to each patient's current antiepileptic drug regimen at a dose of 0.25-1.0 mg/kg/day (max. 20 mg/day). The incidence of major motor seizures (tonic, clonic, tonic-clonic, atonic and myoclonic seizures lasting >30 s) in both the baseline and treatment periods was assessed via a seizure diary. Periodic echocardiographic examinations during the treatment period were used to assess cardiovascular safety.

Results: Nine patients (aged 1.2-29.8 years) enrolled in the study and were treated with fenfluramine for a median duration of 1.5 (range, 0.3-5.1) years. Median frequency of major motor seizures was 15.0/month in the baseline period. All patients demonstrated a reduction in seizure frequency during the treatment period with a median reduction of 75% (range, 28-100%). Seven patients (78%) experienced a ≥50% reduction in major motor seizure frequency. The most common adverse events were somnolence (n = 5) and anorexia (n = 4). No evidence of cardiac valvulopathy or pulmonary hypertension was observed.

Conclusions: The effectiveness and safety of low-dose fenfluramine as an add-on therapy for DS in this new prospective cohort supports previous findings.

Keywords: Dravet syndrome; clinical trial; epileptic encephalopathy; orphan drug; refractory epilepsy; serotonin; severe myoclonic epilepsy in infancy.

PubMed Disclaimer

Figures

Figure 1
Figure 1
The effect of add‐on fenfluramine on the frequency of major motor seizures in patients with Dravet syndrome. *Significant differences in Wilcoxon non‐parametric test (P < 0.05) compared with baseline period.
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Dravet C. The core Dravet syndrome phenotype. Epilepsia 2011; 52(Suppl. 2): 3–9. - PubMed
    1. Bayat A, Hjalgrim H, Moller RS. The incidence of SCN1A‐related Dravet syndrome in Denmark is 1:22,000: a population‐based study from 2004 to 2009. Epilepsia 2015; 56: e36–e39. - PubMed
    1. Bender AC, Morse RP, Scott RC, Holmes GL, Lenck‐Santini PP. SCN1A mutations in Dravet syndrome: impact of interneuron dysfunction on neural networks and cognitive outcome. Epilepsy Behav 2012; 23: 177–186. - PMC - PubMed
    1. Brunklaus A, Ellis R, Reavey E, Forbes GH, Zuberi SM. Prognostic, clinical and demographic features in SCN1A mutation‐positive Dravet syndrome. Brain 2012; 135(Pt 8): 2329–2336. - PubMed
    1. Shi XY, Tomonoh Y, Wang WZ, et al Efficacy of antiepileptic drugs for the treatment of Dravet syndrome with different genotypes. Brain Dev 2016; 38: 40–46. - PubMed