Outcomes of pediatric and adult patients with fenfluramine in a real-life setting
Abstract number :
2.344
Submission category :
7. Anti-seizure Medications / 7C. Cohort Studies
Year :
2025
Submission ID :
863
Source :
www.aesnet.org
Presentation date :
12/7/2025 12:00:00 AM
Published date :
Authors :
Presenting Author: Vicente Villanueva Haba, MD, Phd – Refractory Epilepsy Unit, Neurology Service, Hospital Universitario y Politécnico La Fe, Valencia, Spain
Elena González-Alguacil, MD – Hospital Niño Jesus (Madrid)
Patricia Smeyers, MD, PhD – Hospital Universitario LA Fe, Valencia
Victor Soto-Insuga, PhD – Hospital Niño Jesus (Madrid)
Lucas Dario Iacampo, MD – Hospital Universitario Canarias
David Conejo, MD – Hospital Universitario Burgos
Belen Ortuño, MD – Hospital Universitario Sant Joan de Deu
Yolanda Lopez, MD – Hospital Regional Malaga
Alejandro Fernandez-Cabrera, MD – Hospital Universitario Lucus Agusti
Paula Martinez-Agredano, MD – Hospital Universitario Cordoba
Jose Carlos Estevez, MD – Hospital Universitario Cordoba
Alba Sierra, MD – Hospital Universitario Sant Pau
Eulalia Turón-Viñas, MD – Hospital de la Santa Creu i Sant Pau, IIBSP Research Institute, Barcelona, Spain.
Belen Baena, MD – Hospital Universitario Gregorio Marañon
Blanca Mercedes-Alvarez, MD – Hospital Universitario Gregorio Marañon
Maria de Toledo, MD – Hospital Universitario La Princesa
Rocio Trincado, MD – Hospital Universitario Niño Jesus, Madird
Debora Sayas, Nurse – Hospital Universitario La Fe
Fatima Romero, MD – Spanish Epilepsu Society
Jose Maria Serratosa, MD, PhD – Hospital Universitario Fundacion Jimenez Diaz, Madrid
Rationale: Antiseizure medication (ASM) real-world data provide useful insights about the use of ASM in clinical practice. Fenfluramina (FFL) is a new medication approved as an orphan drug in patients with Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS). This study analyzes FFL use in a series of patients with LGS, DS and other encephalopathies
Methods: Multicenter, retrospective, observational study. Inclusion criteria were: 1) Patients treated with FFL in clinical practice started at least 12 months before database closure 2) Patients or legal representatives agreed to participate. The source of data was patient clinical records and analyzed time-points included baseline, 3 months, 6 months, 1 year. Dosage, concomitant ASM, effectiveness (including total and disabling seizures as drop or convulsive seizures) and safety were evaluated.
Results: In this interim analysis a total of 44 patients were included (24 patients LGS, 14 patients DS and 6 patients other encephalopathies) being 48.8% > 14 year-old. Baseline median number of total and most disabling seizures per month was 33.3 and 18.0 respectively. The median number of prior and concomitant ASM was 8 and 3 respectively. Most frequent baseline ASM were Valproate (61.4%). The mean dosage of FFL at 3, 6 and 12 months was 18.9, 22.5 and 22.4 mg/day in patients > 14 year-old and 0.45, 0.51 and 0.55 mg/Kg/day in patients ≤ 14 years-old. Retention rate at 3 months, 6 months and 12 months was 97.7%, 81.8% and 70.5% respectively. Effectiveness regarding total seizures/most disabling seizures at 3 months, 6 months and 12 months showed 54.7%/67.5%, 55.6%/65.7.5% and 58.5%/80.7% of ≥50% responder. Seizure freedom at the last visit was achieved by 7.3% of patients. Along the follow-up 50.8% patients reported adverse events (11.3% lead to discontinuation), being somnolence (36.4%) the most frequent. No cardiac problems were reported in this series.
Conclusions: Outcomes in a real-life children and adult patient population treated with FFL showed promising efficacy with a good tolerability profile. Further information is needed to consolidate results in clinical practice
Funding: The study was funded with a UCB IIS grant
Anti-seizure Medications