{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,11]],"date-time":"2026-09-11T04:19:13Z","timestamp":1789100353516,"version":"build-2803163510"},"reference-count":20,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2023,12,21]],"date-time":"2023-12-21T00:00:00Z","timestamp":1703116800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Epilepsia"],"published-print":{"date-parts":[[2024,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Dravet syndrome (DS) is a rare developmental and epileptic encephalopathy. Infants with DS are especially vulnerable to the detrimental effects of prolonged and frequent seizures on development. Fenfluramine (FFA) is approved for the treatment of DS in patients aged 2\u2009years and older. This study aims to evaluate the safety and efficacy of FFA in patients with DS younger than 2\u2009years. We analyzed safety, tolerability, seizure, and neuropsychological outcome in a real\u2010world setting. Developmental profile was investigated using Griffiths Mental Development Scales (GMDS). Five patients received FFA at a mean age of 14.9\u2009months (9.6\u201318.6). Median follow\u2010up was 13\u2009months (interquartile range [IQR]\u2009=\u200912.9\u201324.4). All patients showed good tolerance to FFA. No significant variation of body mass index or echocardiographic issue was observed. Monthly median convulsive seizure frequency (MCSF) was 1.71 (IQR\u2009=\u20091.56\u20133.27) at the 6\u2010month baseline period and .92 (IQR\u2009=\u2009.43\u20131.28) at last follow\u2010up, with a median 54.43 (IQR\u2009=\u200940.91\u201360.83) percentage reduction in MCSF. Two of five patients had a performance improvement on GMDS subscales. Overall, the use of FFA below the age of 2 years in our small sample of patients was safe and represents a promising opportunity for seizure control and for protection of the neurodevelopmental outcome.<\/jats:p>","DOI":"10.1111\/epi.17854","type":"journal-article","created":{"date-parts":[[2023,12,9]],"date-time":"2023-12-09T06:59:05Z","timestamp":1702105145000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":16,"title":["Fenfluramine below the age of 2 years in Dravet syndrome: What about safety and efficacy?"],"prefix":"10.1111","volume":"65","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2446-796X","authenticated-orcid":false,"given":"Nicola","family":"Pietrafusa","sequence":"first","affiliation":[{"name":"Neurology, Epilepsy, and Movement Disorders Unit, Full Member of European Reference Network EpiCARE, Bambino Ges\u00f9 Children's Hospital IRCCS  Rome 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":"Neurology, Epilepsy, and Movement Disorders Unit, Full Member of European Reference Network EpiCARE, Bambino Ges\u00f9 Children's Hospital IRCCS  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7884-7354","authenticated-orcid":false,"given":"Susanna","family":"Casellato","sequence":"additional","affiliation":[{"name":"Diagnosis and Treatment of Developmental Epilepsy, Child Neuropsychiatry Unit Women and Children Department, AOU Sassari  Sassari Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9302-5412","authenticated-orcid":false,"given":"Cinzia","family":"Correale","sequence":"additional","affiliation":[{"name":"Neurology, Epilepsy, and Movement Disorders Unit, Full Member of European Reference Network EpiCARE, Bambino Ges\u00f9 Children's Hospital IRCCS  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0738-1240","authenticated-orcid":false,"given":"Simona","family":"Cappelletti","sequence":"additional","affiliation":[{"name":"Neurology, Epilepsy, and Movement Disorders Unit, Full Member of European Reference Network EpiCARE, Bambino Ges\u00f9 Children's Hospital IRCCS  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7836-0194","authenticated-orcid":false,"given":"Paola","family":"De\u00a0Liso","sequence":"additional","affiliation":[{"name":"Neurology, Epilepsy, and Movement Disorders Unit, Full Member of European Reference Network EpiCARE, Bambino Ges\u00f9 Children's Hospital IRCCS  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-3328-8235","authenticated-orcid":false,"given":"Ilaria","family":"Onida","sequence":"additional","affiliation":[{"name":"Diagnosis and Treatment of Developmental Epilepsy, Child Neuropsychiatry Unit Women and Children Department, AOU Sassari  Sassari Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0389-1035","authenticated-orcid":false,"given":"Stefano","family":"Sotgiu","sequence":"additional","affiliation":[{"name":"Diagnosis and Treatment of Developmental Epilepsy, Child Neuropsychiatry Unit Women and Children Department, AOU Sassari  Sassari Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0008-3925-1292","authenticated-orcid":false,"given":"Ambra","family":"Butera","sequence":"additional","affiliation":[{"name":"Neurology and Child Psychiatry Unit, Gaetano Barresi Department of Human Pathology of Adulthood and Development University of Messina  Messina 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":"Neurology, Epilepsy, and Movement Disorders Unit, Full Member of European Reference Network EpiCARE, Bambino Ges\u00f9 Children's Hospital IRCCS  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7513-0051","authenticated-orcid":false,"given":"Federico","family":"Vigevano","sequence":"additional","affiliation":[{"name":"Neurological Sciences and Rehabilitation Medicine Scientific Area, Bambino Ges\u00f9 Children's Hospital IRCCS  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,12,21]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.17239"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1080\/14740338.2021.1890025"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.17274"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.16844"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.yebeh.2016.06.006"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1080\/09297049.2014.967670"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.16474"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ebr.2021.100461"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.seizure.2021.10.024"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.yebeh.2022.108994"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.16456"},{"key":"e_1_2_10_13_1","doi-asserted-by":"crossref","unstructured":"GriffithsR.The Griffiths Mental Development Scales from birth to 2\u2009years. Manual. The 1996 revision.1996. Association for Research in Infant and Child Development Test Agency Henley.","DOI":"10.1037\/t03301-000"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.16690"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.17034"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/epi4.12624"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.13591"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1080\/09297049.2015.1054272"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1080\/09297049.2017.1365829"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.eplepsyres.2022.106976"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/epi4.12281"}],"container-title":["Epilepsia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/epi.17854","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,7,5]],"date-time":"2024-07-05T15:36:37Z","timestamp":1720193797000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/epi.17854"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12,21]]},"references-count":20,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2024,2]]}},"alternative-id":["10.1111\/epi.17854"],"URL":"https:\/\/doi.org\/10.1111\/epi.17854","archive":["Portico"],"relation":{},"ISSN":["0013-9580","1528-1167"],"issn-type":[{"value":"0013-9580","type":"print"},{"value":"1528-1167","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,12,21]]},"assertion":[{"value":"2023-11-03","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-12-07","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-12-21","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}