{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T02:47:13Z","timestamp":1786934833698,"version":"build-2736575974"},"reference-count":31,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2021,8,10]],"date-time":"2021-08-10T00:00:00Z","timestamp":1628553600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Epilepsia"],"published-print":{"date-parts":[[2021,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>Dravet syndrome (DS) is a rare but severe drug\u2010resistant epilepsy. Before the approval of fenfluramine (FFA) for the treatment of seizures in DS, patients in Germany could receive treatment under a compassionate use program (CUP).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We conducted a multicenter, retrospective, observational study to describe the efficacy, tolerability, and retention of FFA within the CUP. Patients received add\u2010on therapy with oral FFA gradually titrated to a target dose between .13 and .7\u00a0mg\/kg\/day.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Overall, 78 patients with DS (median age = 8.0 years, range = 2.1\u201346.0; 53% female, median concomitant antiseizure medications [ASMs] = 3) were treated with FFA for a median duration of 255.5\u00a0days (range = 31\u2013572). Responder rates (a \u226550% reduction;<jats:italic>n<\/jats:italic>\u00a0=\u00a078) and seizure\u2010freedom rates at 3\u00a0months were 68% and 14% for total seizures, respectively, and 67% and 23% for generalized tonic\u2013clonic seizures. Responder rates were consistent at 6 and 12\u00a0months (<jats:italic>n<\/jats:italic>\u00a0=\u00a066 and<jats:italic>n<\/jats:italic>\u00a0=\u00a043, respectively). Median seizure days per month significantly decreased from 10.0 (range = .5\u201330) to 3.0 (range = 0\u201330) in the 3\u2010month period before and after FFA treatment (<jats:italic>p<\/jats:italic>\u00a0&lt;\u00a0.001). Significantly fewer patients reported at least one episode of status epilepticus (28% vs. 14% patients before and after FFA initiation,<jats:italic>p<\/jats:italic>\u00a0=\u00a0.005). During FFA treatment, 35 (45%) patients were able to discontinue a concomitant ASM. At the last follow\u2010up date, 66 (85%) patients remained on treatment with FFA. The most common adverse events were somnolence (36%), decreased appetite (22%), and ataxia (8%). Forty\u2010eight (62%) patients were reported as having a meaningful global clinical improvement.<\/jats:p><\/jats:sec><jats:sec><jats:title>Significance<\/jats:title><jats:p>In a large cohort of patients, FFA demonstrated efficacy across a range of outcomes including clinically significant reductions in convulsive seizures, and was well tolerated, providing valuable information for real\u2010world practice.<\/jats:p><\/jats:sec>","DOI":"10.1111\/epi.17034","type":"journal-article","created":{"date-parts":[[2021,8,11]],"date-time":"2021-08-11T02:20:08Z","timestamp":1628648408000},"page":"2518-2527","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":41,"title":["Efficacy, tolerability, and retention of fenfluramine for the treatment of seizures in patients with Dravet syndrome: Compassionate use program in Germany"],"prefix":"10.1111","volume":"62","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6288-9915","authenticated-orcid":false,"given":"Adam","family":"Strzelczyk","sequence":"first","affiliation":[{"name":"Epilepsy Center Frankfurt Rhine\u2010Main, Center of Neurology and Neurosurgery Goethe University Frankfurt Frankfurt am Main Germany"},{"name":"Center for Personalized Translational Epilepsy Research Goethe University Frankfurt Frankfurt am Main Germany"}],"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 Clinic Vogtareuth Vogtareuth Germany"}],"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":"Tilman","family":"Polster","sequence":"additional","affiliation":[{"name":"Department of Epileptology Bethel Epilepsy Center Mara Hospital Bielefeld University Bielefeld Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1601-5384","authenticated-orcid":false,"given":"Kerstin A.","family":"Klotz","sequence":"additional","affiliation":[{"name":"Department of Neuropediatrics and Muscle Disorders Center for Pediatrics Medical Center Faculty of Medicine University of Freiburg Freiburg Germany"},{"name":"Berta Ottenstein Program Faculty of Medicine University of Freiburg Freiburg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hiltrud","family":"Muhle","sequence":"additional","affiliation":[{"name":"Department of Neuropediatrics Christian\u2010Albrecht University of Kiel and University Hospital Schleswig\u2010Holstein Kiel Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Alber","sequence":"additional","affiliation":[{"name":"Department of Neuropediatrics University of T\u00fcbingen T\u00fcbingen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6031-8537","authenticated-orcid":false,"given":"Regina","family":"Trollmann","sequence":"additional","affiliation":[{"name":"Department of Neuropediatrics Friedrich\u2010Alexander University Erlangen Erlangen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hartwig","family":"Spors","sequence":"additional","affiliation":[{"name":"Department of Neuropediatrics Justus Liebig University Giessen Giessen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gerhard","family":"Kluger","sequence":"additional","affiliation":[{"name":"Clinic for Neuropediatrics and Neurorehabilitation Epilepsy Center for Children and Adolescents Sch\u00f6n Clinic Vogtareuth Vogtareuth Germany"},{"name":"Research Institute, Rehabilitation, Transition, and Palliation Paracelsus Medical University Salzburg Salzburg Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gerhard","family":"Kurlemann","sequence":"additional","affiliation":[{"name":"St. Bonifatius Hospital Lingen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1545-7364","authenticated-orcid":false,"given":"Susanne","family":"Schubert\u2010Bast","sequence":"additional","affiliation":[{"name":"Epilepsy Center Frankfurt Rhine\u2010Main, Center of Neurology and Neurosurgery Goethe University Frankfurt Frankfurt am Main Germany"},{"name":"Center for Personalized Translational Epilepsy Research Goethe University Frankfurt Frankfurt am Main Germany"},{"name":"Department of Neuropediatrics Goethe University Frankfurt Frankfurt am Main Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2021,8,10]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1080\/14737175.2020.1801423"},{"key":"e_1_2_9_3_1","first-page":"10","article-title":"Fatal status epilepticus in Dravet syndrome","volume":"23","author":"De Liso P","year":"2020","journal-title":"Brain 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