{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,4]],"date-time":"2026-08-04T03:21:08Z","timestamp":1785813668098,"version":"3.56.0"},"reference-count":33,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2020,9,18]],"date-time":"2020-09-18T00:00:00Z","timestamp":1600387200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Epilepsia"],"published-print":{"date-parts":[[2020,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>Dravet syndrome (DS) is a drug\u2010resistant, infantile onset epilepsy syndrome with multiple seizure types and developmental delay. In recently published randomized controlled trials, fenfluramine (FFA) proved to be safe and effective in DS.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>DS patients were treated with FFA in the Zogenix Early Access Program at four Italian pediatric epilepsy centers. FFA was administered as add\u2010on, twice daily at an initial dose of 0.2\u00a0mg\/kg\/d up to 0.7\u00a0mg\/kg\/d. Seizures were recorded in a diary. Adverse events and cardiac safety (with Doppler echocardiography) were investigated every 3 to 6\u00a0months.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Fifty\u2010two patients were enrolled, with a median age of 8.6\u00a0years (interquartile range [IQR] = 4.1\u201013.9). Forty\u2010five (86.5%) patients completed the efficacy analysis. The median follow\u2010up was 9.0\u00a0months (IQR = 3.2\u20109.5). At last follow\u2010up visit, there was a 77.4% median reduction in convulsive seizures. Thirty\u2010two patients (71.1%) had a \u226550% reduction of convulsive seizures, 24 (53.3%) had a \u226575% reduction, and five (11.1%) were seizure\u2010free. The most common adverse event was decreased appetite (n\u00a0=\u00a07, 13.4%). No echocardiographic signs of cardiac valvulopathy or pulmonary hypertension were observed. There was no correlation between type of genetic variants and response to FFA.<\/jats:p><\/jats:sec><jats:sec><jats:title>Significance<\/jats:title><jats:p>In this real\u2010world study, FFA provided a clinically meaningful reduction in convulsive seizure frequency in the majority of patients with DS and was well tolerated.<\/jats:p><\/jats:sec>","DOI":"10.1111\/epi.16690","type":"journal-article","created":{"date-parts":[[2020,9,18]],"date-time":"2020-09-18T13:10:49Z","timestamp":1600434649000},"page":"2405-2414","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":54,"title":["Efficacy and safety of Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: A real\u2010world study"],"prefix":"10.1111","volume":"61","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8120-0287","authenticated-orcid":false,"given":"Nicola","family":"Specchio","sequence":"first","affiliation":[{"name":"Rare and Complex Epilepsy Unit Department of Neuroscience Bambino Ges\u00f9 Children\u2019s Hospital IRCCS Full Member of European Reference Network EpiCARE Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2446-796X","authenticated-orcid":false,"given":"Nicola","family":"Pietrafusa","sequence":"additional","affiliation":[{"name":"Rare and Complex Epilepsy Unit Department of Neuroscience Bambino Ges\u00f9 Children\u2019s Hospital IRCCS Full Member of European Reference Network EpiCARE Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Viola","family":"Doccini","sequence":"additional","affiliation":[{"name":"Neuroscience Department A. Meyer Children's Hospital\u2013University of Florence Full Member of European Reference Network EpiCARE Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9841-8581","authenticated-orcid":false,"given":"Marina","family":"Trivisano","sequence":"additional","affiliation":[{"name":"Rare and Complex Epilepsy Unit Department of Neuroscience Bambino Ges\u00f9 Children\u2019s Hospital IRCCS Full Member of European Reference Network EpiCARE Rome Italy"}],"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 Verona Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Ragona","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neuroscience IRCCS Foundation Carlo Besta Neurological Institute Full Member of the European Reference Network EpiCare Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6945-6151","authenticated-orcid":false,"given":"Alberto","family":"Cossu","sequence":"additional","affiliation":[{"name":"Child Neuropsychiatry Department of Surgical Sciences Dentistry, Gynecology, and Pediatrics University of Verona Verona Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Silvia","family":"Spolverato","sequence":"additional","affiliation":[{"name":"Child Neuropsychiatry Department of Surgical Sciences Dentistry, Gynecology, and Pediatrics University of Verona Verona Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Domenica","family":"Battaglia","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Department of Woman and Child Health and Public Health Child Health Area A. Gemelli University Polyclinic Foundation IRCCS Catholic University of the Sacred Heart Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michela","family":"Quintiliani","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Department of Woman and Child Health and Public Health Child Health Area A. Gemelli University Polyclinic Foundation IRCCS Catholic University of the Sacred Heart Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"Luigia Gambardella","sequence":"additional","affiliation":[{"name":"Pediatric Neurology Department of Woman and Child Health and Public Health Child Health Area A. Gemelli University Polyclinic Foundation IRCCS Catholic University of the Sacred Heart Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8754-7214","authenticated-orcid":false,"given":"Anna","family":"Rosati","sequence":"additional","affiliation":[{"name":"Neuroscience Department A. Meyer Children's Hospital\u2013University of Florence Full Member of European Reference Network EpiCARE Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6790-6251","authenticated-orcid":false,"given":"Davide","family":"Mei","sequence":"additional","affiliation":[{"name":"Neuroscience Department A. 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