Authors :
XIANA RODRIGUEZ OSORIO, MD PhD – Hospital Clínico Universitario of Santiago de Compostela.
Presenting Author: Álvaro Juiz Fernández, MD – Hospital Clínico Universitario of Santiago de Compostela.
Alejandro Fernandez-Cabrera, MD – Hospital Universitario Lucus Agusti
Ángel Pérez Álvarez, MD – Hospital Central of Asturias, Oviedo
Marta Marin Gracia, MD – University Clinic of Navarra
Francisco Javier López González, MD PhD – Hospital Clínico Universitario of Santiago de Compostela.
Rationale:
Fenfluramine (FFA), marketed in Spain in 2022, is indicated as adjunctive treatment for epileptic seizures in patients with Dravet syndrome and Lennox-Gastaut syndrome from 2 years of age. Our aim is to describe real-world experience with FFA use in pediatric and adult patients across several centers in northern Spain.
Methods:
A retrospective observational study including patients treated with FFA with at least 1 year of follow-up. Demographic and clinical characteristics, treatment response and tolerability, and retention rate were evaluated.
Results:
A total of 24 patients (15 male; 62.5%) were included, with a mean age of 29.67 ± 17.52 years, all diagnosed with Lennox-Gastaut syndrome. At 12 months, a mean reduction of 35.72% in total seizure frequency was observed. Overall, 54% (13/24) were responders, and among them, 76% (10/13) achieved a ≥ 80% reduction. Sixteen patients had drop seizures at baseline. After 12 months of treatment, 5/16 became seizure-free, 4/16 achieved a ≥ 80% reduction, and 3/16 showed a 50–79% reduction. The mean FFA dose at 12 months was 20.85 ± 6.43 mg/day.
A total of 34 adverse events (AEs) were reported: 23 mild, 10 moderate, and 1 severe. The most frequent were somnolence (N=10) and diarrhea (N=9). Five patients discontinued treatment: 2 due to AEs, 2 due to lack of efficacy, and 1 due to both. The 1-year retention rate was 79.2%.
Conclusions:
Real-world experience supports the use of FFA as an effective and well-tolerated therapeutic option.
Funding: No funding was received in support of this abstract.