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
Abstract Objective To examine the efficacy and safety of fenfluramine in patients with Dravet syndrome (DS) in three age groups: <6, 6‐17, 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)....
| Main Authors: | , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Wiley
2022-12-01
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| Series: | Epilepsia Open |
| Subjects: | |
| Online Access: | https://doi.org/10.1002/epi4.12624 |
| _version_ | 1811321750428844032 |
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| author | Renzo Guerrini Nicola Specchio Ángel Aledo‐Serrano Milka Pringsheim Francesca Darra Thomas Mayer Antonio Gil‐Nagel Tilman Polster Sameer M. Zuberi Amélie Lothe Arnold Gammaitoni Adam Strzelczyk |
| author_facet | Renzo Guerrini Nicola Specchio Ángel Aledo‐Serrano Milka Pringsheim Francesca Darra Thomas Mayer Antonio Gil‐Nagel Tilman Polster Sameer M. Zuberi Amélie Lothe Arnold Gammaitoni Adam Strzelczyk |
| author_sort | Renzo Guerrini |
| collection | DOAJ |
| description | Abstract Objective To examine the efficacy and safety of fenfluramine in patients with Dravet syndrome (DS) in three age groups: <6, 6‐17, 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 6‐17 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. |
| first_indexed | 2024-04-13T13:23:07Z |
| format | Article |
| id | doaj.art-b5e7d1e7768f4ec49153337296d89035 |
| institution | Directory Open Access Journal |
| issn | 2470-9239 |
| language | English |
| last_indexed | 2024-04-13T13:23:07Z |
| publishDate | 2022-12-01 |
| publisher | Wiley |
| record_format | Article |
| series | Epilepsia Open |
| spelling | doaj.art-b5e7d1e7768f4ec49153337296d890352022-12-22T02:45:16ZengWileyEpilepsia Open2470-92392022-12-017457858710.1002/epi4.12624An 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 ProgramRenzo Guerrini0Nicola Specchio1Ángel Aledo‐Serrano2Milka Pringsheim3Francesca Darra4Thomas Mayer5Antonio Gil‐Nagel6Tilman Polster7Sameer M. Zuberi8Amélie Lothe9Arnold Gammaitoni10Adam Strzelczyk11Children's Hospital A Meyer University of Florence, Full Member of European Reference Network on Rare and Complex Epilepsies EpiCARE Florence ItalyRare and Complex Epilepsy Unit, Department of Neuroscience Bambino Gesù Children's Hospital IRCCS, Full Member of European Reference Network on Rare and Complex Epilepsies EpiCARE Rome ItalyEpilepsy Program, Department of Neurology Hospital Ruber Internacional Madrid SpainClinic for Neuropediatrics and Neurorehabilitation, Epilepsy Center for Children and Adolescents Schön Klinik Vogtareuth GermanyChild Neuropsychiatry, Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics University of Verona, Full Member of European Reference Network on Rare and Complex Epilepsies EpiCARE Verona ItalyEpilepsy Center Kleinwachau Dresden‐Radeberg GermanyEpilepsy Program, Department of Neurology Hospital Ruber Internacional Madrid SpainKrankenhaus Mara – Bethel Epilepsy Centre, Department of Epileptology, Medical School Bielefeld University Bielefeld GermanyPaediatric Neurosciences Research Group, Royal Hospital for Children & Institute of Health & Wellbeing University of Glasgow Glasgow UKZogenix International Maidenhead UKZogenix, Inc. Emeryville California USAEpilepsy Center Frankfurt Rhine‐Main and Department of Neurology Goethe‐University Frankfurt Frankfurt am Main GermanyAbstract Objective To examine the efficacy and safety of fenfluramine in patients with Dravet syndrome (DS) in three age groups: <6, 6‐17, 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 6‐17 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.https://doi.org/10.1002/epi4.12624clinical practiceDravet syndromefenfluraminerefractory epilepsyseizures |
| spellingShingle | Renzo Guerrini Nicola Specchio Ángel Aledo‐Serrano Milka Pringsheim Francesca Darra Thomas Mayer Antonio Gil‐Nagel Tilman Polster Sameer M. Zuberi Amélie Lothe Arnold Gammaitoni Adam Strzelczyk 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 clinical practice Dravet syndrome fenfluramine refractory epilepsy seizures |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| topic | clinical practice Dravet syndrome fenfluramine refractory epilepsy seizures |
| url | https://doi.org/10.1002/epi4.12624 |
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