{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,3]],"date-time":"2026-10-03T02:18:43Z","timestamp":1790993923695,"version":"4.1.0"},"reference-count":20,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2022,8,8]],"date-time":"2022-08-08T00:00:00Z","timestamp":1659916800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100012739","name":"Zogenix","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100012739","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Epilepsia Open"],"published-print":{"date-parts":[[2022,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To examine the efficacy and safety of fenfluramine in patients with Dravet syndrome (DS) in three age groups: &lt;6, 6\u201017, and \u226518\u2009years old, treated in a real\u2010world setting.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Patients with DS were treated with fenfluramine in the European Union Early Access Program (EAP). Following a 28\u2010day 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\u00a0mg\/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\u2009months thereafter.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 149 patients have enrolled in the EAP and 63 were &lt;6\u2009years old, 62 were 6\u201017\u2009years old, and 24 were \u226518\u2009years old. After 3\u2009months of treatment 62%, 53%, and 50% of patients demonstrated \u226575% reduction in MCSF in the &lt;6, 6\u201017, and \u226518\u2010year\u2010old groups, respectively. This pattern of response was sustained through 12\u00a0months of treatment with 55%, 46%, and 80% of the &lt;6, 6\u201017, and \u226518\u2010year\u2010old groups, respectively, experiencing a \u226575% reduction in MCSF. Most common AEs were loss of appetite (21%) and somnolence (16%). No valvular heart disease or pulmonary artery hypertension was observed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Significance<\/jats:title><jats:p>The magnitude, consistency, and durability of the response to add\u2010on fenfluramine is consistent across age groups in patients with Dravet syndrome.<\/jats:p><\/jats:sec>","DOI":"10.1002\/epi4.12624","type":"journal-article","created":{"date-parts":[[2022,7,8]],"date-time":"2022-07-08T10:19:47Z","timestamp":1657275587000},"page":"578-587","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":43,"title":["An examination of the efficacy and safety of fenfluramine in adults, children, and adolescents with Dravet syndrome in a <scp>real\u2010world<\/scp> practice setting: A report from the Fenfluramine European Early Access Program"],"prefix":"10.1002","volume":"7","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7272-7079","authenticated-orcid":false,"given":"Renzo","family":"Guerrini","sequence":"first","affiliation":[{"name":"Children's Hospital A Meyer University of Florence, Full Member of European Reference Network on Rare and Complex Epilepsies EpiCARE  Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8120-0287","authenticated-orcid":false,"given":"Nicola","family":"Specchio","sequence":"additional","affiliation":[{"name":"Rare and Complex Epilepsy Unit, Department of Neuroscience Bambino Ges\u00f9 Children's Hospital IRCCS, Full Member of European Reference Network on Rare and Complex Epilepsies EpiCARE  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4889-3365","authenticated-orcid":false,"given":"\u00c1ngel","family":"Aledo\u2010Serrano","sequence":"additional","affiliation":[{"name":"Epilepsy Program, Department of Neurology Hospital Ruber Internacional  Madrid Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Milka","family":"Pringsheim","sequence":"additional","affiliation":[{"name":"Clinic for Neuropediatrics and Neurorehabilitation, Epilepsy Center for Children and Adolescents Sch\u00f6n Klinik  Vogtareuth Germany"},{"name":"Department for Congenital Heart Disease and Pediatric Cardiology German Heart Center  Munich Germany"},{"name":"Research Institute for Rehabilitation, Transition and Palliation Paracelsus Medical University  Salzburg Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Darra","sequence":"additional","affiliation":[{"name":"Child 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 Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Mayer","sequence":"additional","affiliation":[{"name":"Epilepsy Center Kleinwachau  Dresden\u2010Radeberg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Gil\u2010Nagel","sequence":"additional","affiliation":[{"name":"Epilepsy Program, Department of Neurology Hospital Ruber Internacional  Madrid Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9531-7312","authenticated-orcid":false,"given":"Tilman","family":"Polster","sequence":"additional","affiliation":[{"name":"Krankenhaus Mara \u2013 Bethel Epilepsy Centre, Department of Epileptology, Medical School Bielefeld University  Bielefeld Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sameer M.","family":"Zuberi","sequence":"additional","affiliation":[{"name":"Paediatric Neurosciences Research Group, Royal Hospital for Children &amp; Institute of Health &amp; Wellbeing University of Glasgow  Glasgow UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Am\u00e9lie","family":"Lothe","sequence":"additional","affiliation":[{"name":"Zogenix International  Maidenhead UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4775-2862","authenticated-orcid":false,"given":"Arnold","family":"Gammaitoni","sequence":"additional","affiliation":[{"name":"Zogenix, Inc.  Emeryville California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6288-9915","authenticated-orcid":false,"given":"Adam","family":"Strzelczyk","sequence":"additional","affiliation":[{"name":"Epilepsy Center Frankfurt Rhine\u2010Main and Department of Neurology Goethe\u2010University Frankfurt  Frankfurt am Main Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,8,8]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1528-1167.2011.02994.x"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.eplepsyres.2016.10.006"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.13591"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.yebeh.2016.09.007"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2015-1807"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(19)32500-0"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.yebeh.2014.12.028"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.yebeh.2019.06.021"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.yebeh.2020.106989"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2019.4113"},{"key":"e_1_2_10_12_1","unstructured":"SchefferIE DevinskyO PerryMS WhelessJ ThieleEA WirrellE et al.Efficacy and tolerability of adjunctive FINTEPLA (fenfluramine HCl) in an open\u2010label extension study of Dravet syndrome patients treated for up to 3\u2009years [poster]. 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