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Efficacy and safety of Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: A real‐world study
Author(s) -
Specchio Nicola,
Pietrafusa Nicola,
Doccini Viola,
Trivisano Marina,
Darra Francesca,
Ragona Francesca,
Cossu Alberto,
Spolverato Silvia,
Battaglia Domenica,
Quintiliani Michela,
Luigia Gambardella Maria,
Rosati Anna,
Mei Davide,
Granata Tiziana,
Dalla Bernardina Bernardo,
Vigevano Federico,
Guerrini Renzo
Publication year - 2020
Publication title -
epilepsia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.687
H-Index - 191
eISSN - 1528-1167
pISSN - 0013-9580
DOI - 10.1111/epi.16690
Subject(s) - dravet syndrome , fenfluramine , medicine , interquartile range , adverse effect , epilepsy , pediatrics , epilepsy syndromes , anesthesia , lennox–gastaut syndrome , receptor , psychiatry , serotonin
Abstract Objective 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. Methods 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. Results 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. Significance 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.