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. 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.

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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

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