{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,16]],"date-time":"2026-09-16T19:20:13Z","timestamp":1789586413821,"version":"build-2803163510"},"reference-count":16,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2025,2,9]],"date-time":"2025-02-09T00:00:00Z","timestamp":1739059200000},"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":["Develop Med Child Neuro"],"published-print":{"date-parts":[[2025,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Aim<\/jats:title><jats:p>To conduct a postmarketing surveillance study of patients with Dravet syndrome in Japan to investigate the safety and effectiveness of long\u2010term, real\u2010world, clinical use of stiripentol (STP).<\/jats:p><\/jats:sec><jats:sec><jats:title>Method<\/jats:title><jats:p>This prospective study was conducted over 156\u2009weeks in all patients with Dravet syndrome who started STP treatment from its launch in Japan in November 2012 until August 2017. Adverse drug reactions (ADRs) were investigated by degree of seriousness. Effectiveness was determined based on a comprehensive assessment by the physician in charge as well as on the percentage change in the number of seizures from the pretreatment period.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In total, 520 patients (266 males, 254 females; mean age [SD] 10\u2009years 6\u2009months [9\u2009years 10\u2009months]; age range 0\u201350\u2009years) were included in the safety analysis set, and 515 patients in the effectiveness analysis set. ADRs occurred in 69.2%, including somnolence, decreased appetite, dizziness, in order of frequency. Twelve deaths occurred, the rate of which was not higher than the reported rates. No new safety concerns were identified. The rate of overall improvement (marked or moderate) after 156\u2009weeks or at treatment discontinuation was 37.7%. Decreases in the number of all seizure types over the long term were confirmed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>In real\u2010world clinical settings, long\u2010term STP treatment can be safe and effective in patients with Dravet syndrome.<\/jats:p><\/jats:sec>","DOI":"10.1111\/dmcn.16252","type":"journal-article","created":{"date-parts":[[2025,2,10]],"date-time":"2025-02-10T21:54:59Z","timestamp":1739224499000},"page":"1034-1041","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Safety and effectiveness of stiripentol in patients with Dravet syndrome: A prospective, 3\u2010year, postmarketing surveillance study"],"prefix":"10.1111","volume":"67","author":[{"ORCID":"https:\/\/orcid.org\/0009-0001-8875-2244","authenticated-orcid":false,"given":"Yuki","family":"Kitamura","sequence":"first","affiliation":[{"name":"Safety Vigilance &amp; Management Department, Reliability &amp; Quality Assurance Division Meiji Seika Pharma Co., Ltd.  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hiroaki","family":"Ohyabu","sequence":"additional","affiliation":[{"name":"Safety Vigilance &amp; Management Department, Reliability &amp; Quality Assurance Division Meiji Seika Pharma Co., Ltd.  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tatsuo","family":"Miura","sequence":"additional","affiliation":[{"name":"Safety Vigilance &amp; Management Department, Reliability &amp; Quality Assurance Division Meiji Seika Pharma Co., Ltd.  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Naomi","family":"Takei\u2010Masuda","sequence":"additional","affiliation":[{"name":"Safety Vigilance &amp; Management Department, Reliability &amp; Quality Assurance Division Meiji Seika Pharma Co., Ltd.  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daisuke","family":"Matsui","sequence":"additional","affiliation":[{"name":"Safety Vigilance &amp; Management Department, Reliability &amp; Quality Assurance Division Meiji Seika Pharma Co., Ltd.  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yushi","family":"Inoue","sequence":"additional","affiliation":[{"name":"Department of Clinical Research, National Epilepsy Center NHO Shizuoka Institute of Epilepsy and Neurological Disorders  Shizuoka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoko","family":"Ohtsuka","sequence":"additional","affiliation":[{"name":"Department of Child Neurology Asahigawaso Rehabilitation and Medical Center  Okayama Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,2,9]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1528\u20101167.2006.00688.x"},{"key":"e_1_2_10_3_1","unstructured":"Japan Intractable Diseases Information Center.Dravet syndrome (designated intractable disease 140) https:\/\/www.nanbyou.or.jp\/entry\/4745[accessed 2 July 2024]."},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1528\u20101167.2011.03008.x"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.15638"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.eplepsyres.2020.106535"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.eplepsyres.2014.02.008"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.eplepsyres.2015.03.012"},{"key":"e_1_2_10_9_1","first-page":"125","volume-title":"Epileptic Syndromes in Infancy, Childhood and Adolescence","author":"Dravet C","year":"2012"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.13670"},{"key":"e_1_2_10_11_1","volume-title":"Labour and Welfare","author":"Ministry of Health","year":"2000"},{"key":"e_1_2_10_12_1","unstructured":"Instructions for Package Inserts of Prescription Drugs Revised December 2023. 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