Guerrini, R. et al. (2022) An examination of the efficacy and safety of fenfluramine in adults, children, and adolescents with Dravet syndrome in a real-world practice setting: a report from the fenfluramine European Early Access Program. Epilepsia Open, 7(4), pp. 578-587. (doi: 10.1002/epi4.12624) (PMID:35801621) (PMCID:PMC9712464)
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Abstract
Objective: To examine the efficacy and safety of fenfluramine in patients with Dravet syndrome (DS) in three age groups: <6, 6 to 17, and ≥18 years old, treated in a real-world setting. Methods: Patients with DS were treated with fenfluramine in the European Union Early Access Program (EAP). Following a 28-day baseline period to establish the pretreatment monthly convulsive seizure frequency (MCSF), fenfluramine was started at a dose chosen by the treating physician and gradually titrated based on efficacy and tolerability up to a maximum of 0.7 mg/kg/day. Seizure incidence was recorded in a written diary, and adverse events (AEs) were reported at each patient visit. Cardiovascular safety was assessed by transthoracic echocardiography before treatment started and at least every 6 months thereafter. Results: A total of 149 patients have enrolled in the EAP and 63 were <6 years old, 62 were 6 to 17 years old, and 24 were ≥18 years old. After 3 months of treatment 62%, 53%, and 50% of patients demonstrated ≥75% reduction in MCSF in the <6, 6-17, and ≥18-year-old groups, respectively. This pattern of response was sustained through 12 months of treatment with 55%, 46%, and 80% of the <6, 6-17, and ≥18-year-old groups, respectively, experiencing a ≥75% reduction in MCSF. Most common AEs were loss of appetite (21%) and somnolence (16%). No valvular heart disease or pulmonary artery hypertension was observed. Significance: The magnitude, consistency, and durability of the response to add-on fenfluramine is consistent across age groups in patients with Dravet Syndrome.
Item Type: | Articles |
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Additional Information: | The study was funded by Zogenix. |
Status: | Published |
Refereed: | Yes |
Glasgow Author(s) Enlighten ID: | Zuberi, Dr Sameer |
Authors: | Guerrini, R., Specchio, N., Aledo‐Serrano, Á., Pringsheim, M., Darra, F., Mayer, T., Gil‐Nagel, A., Polster, T., Zuberi, S. M., Lothe, A., Gammaitoni, A., and Strzelczyk, A. |
College/School: | College of Medical Veterinary and Life Sciences > School of Medicine, Dentistry & Nursing |
Journal Name: | Epilepsia Open |
Publisher: | Wiley |
ISSN: | 2470-9239 |
ISSN (Online): | 2470-9239 |
Published Online: | 08 July 2022 |
Copyright Holders: | Copyright © 2022 The Authors |
First Published: | First published in Epilepsia Open 7(4): 578-587 |
Publisher Policy: | Reproduced under a Creative Commons License |
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