Efficacy and safety of Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: A real-world study.
Adolescent
Adult
Anorexia
/ chemically induced
Anticonvulsants
/ administration & dosage
Child
Child, Preschool
Epilepsies, Myoclonic
/ diagnostic imaging
Female
Fenfluramine
/ administration & dosage
Follow-Up Studies
Humans
Male
Prospective Studies
Seizures
/ diagnostic imaging
Selective Serotonin Reuptake Inhibitors
/ administration & dosage
Treatment Outcome
Young Adult
Dravet syndrome
SCN1A
childhood epilepsy
convulsive seizures
fenfluramine
Journal
Epilepsia
ISSN: 1528-1167
Titre abrégé: Epilepsia
Pays: United States
ID NLM: 2983306R
Informations de publication
Date de publication:
11 2020
11 2020
Historique:
received:
04
05
2020
revised:
18
08
2020
accepted:
19
08
2020
pubmed:
19
9
2020
medline:
4
2
2021
entrez:
18
9
2020
Statut:
ppublish
Résumé
Dravet syndrome (DS) is a drug-resistant, infantile onset epilepsy syndrome with multiple seizure types and developmental delay. In recently published randomized controlled trials, fenfluramine (FFA) proved to be safe and effective in DS. DS patients were treated with FFA in the Zogenix Early Access Program at four Italian pediatric epilepsy centers. FFA was administered as add-on, twice daily at an initial dose of 0.2 mg/kg/d up to 0.7 mg/kg/d. Seizures were recorded in a diary. Adverse events and cardiac safety (with Doppler echocardiography) were investigated every 3 to 6 months. Fifty-two patients were enrolled, with a median age of 8.6 years (interquartile range [IQR] = 4.1-13.9). Forty-five (86.5%) patients completed the efficacy analysis. The median follow-up was 9.0 months (IQR = 3.2-9.5). At last follow-up visit, there was a 77.4% median reduction in convulsive seizures. Thirty-two patients (71.1%) had a ≥50% reduction of convulsive seizures, 24 (53.3%) had a ≥75% reduction, and five (11.1%) were seizure-free. The most common adverse event was decreased appetite (n = 7, 13.4%). No echocardiographic signs of cardiac valvulopathy or pulmonary hypertension were observed. There was no correlation between type of genetic variants and response to FFA. In this real-world study, FFA provided a clinically meaningful reduction in convulsive seizure frequency in the majority of patients with DS and was well tolerated.
Substances chimiques
Anticonvulsants
0
Serotonin Uptake Inhibitors
0
Fenfluramine
2DS058H2CF
Types de publication
Journal Article
Multicenter Study
Pragmatic Clinical Trial
Langues
eng
Sous-ensembles de citation
IM
Pagination
2405-2414Informations de copyright
© 2020 International League Against Epilepsy.
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