Skip to main page content
Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2016 Jul;57(7):e129-34.
doi: 10.1111/epi.13407. Epub 2016 May 20.

Five-year Extended Follow-Up Status of 10 Patients With Dravet Syndrome Treated With Fenfluramine

Affiliations
Free article

Five-year Extended Follow-Up Status of 10 Patients With Dravet Syndrome Treated With Fenfluramine

Berten Ceulemans et al. Epilepsia. .
Free article

Erratum in

  • Erratum.
    Epilepsia. 2017 Mar;58(3):509-510. doi: 10.1111/epi.13714. Epub 2017 Feb 13. Epilepsia. 2017. PMID: 28262943 No abstract available.

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.

Keywords: Dravet syndrome; FFA; Fenfluramine; Orphan drugs; SCN1A.

Similar articles

See all similar articles

Cited by 14 articles

See all "Cited by" articles

Publication types

LinkOut - more resources

Feedback