{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T21:49:30Z","timestamp":1792705770783,"version":"4.5.0"},"reference-count":30,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2023,5,17]],"date-time":"2023-05-17T00:00:00Z","timestamp":1684281600000},"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":[[2023,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Aim<\/jats:title><jats:p>To evaluate the safety and efficacy of stiripentol initiated before 2\u2009years of age in patients with Dravet syndrome.<\/jats:p><\/jats:sec><jats:sec><jats:title>Method<\/jats:title><jats:p>This was a 30\u2010year, real\u2010world retrospective study. We extracted the data of the 131 patients (59 females, 72 males) who initiated stiripentol before 2\u2009years of age between 1991 and 2021 from the four longitudinal databases of Dravet syndrome available in France.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Stiripentol was added to valproate and clobazam (93%) at 13\u2009months and a median dose of 50\u2009mg\/kg\/day. With short\u2010term therapy (&lt;6\u2009months on stiripentol, median 4\u2009months, median age 16\u2009months), the frequency of tonic\u2013clonic seizures (TCS) lasting longer than 5\u2009minutes decreased (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.01) and status epilepticus (&gt;30\u2009minutes) disappeared in 55% of patients. With long\u2010term therapy (last visit on stiripentol\u2009&lt;7\u2009years of age, median stiripentol 28\u2009months, median age 41\u2009months), the frequency of long\u2010lasting TCS continued to decline (<jats:italic>p<\/jats:italic>\u2009=\u20090.03). Emergency hospitalizations dropped from 91% to 43% and 12% with short\u2010 and long\u2010term therapies respectively (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001). Three patients died, all from sudden unexpected death in epilepsy. Three patients discontinued stiripentol for adverse events; 55% reported at least one adverse event, mostly loss of appetite\/weight (21%) and somnolence (11%). Stiripentol was used earlier, at lower doses, and was better tolerated by patients in the newest database than in the oldest (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.01).<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>Initiating stiripentol in infants with Dravet syndrome is safe and beneficial, significantly reducing long\u2010lasting seizures including status epilepticus, hospitalizations, and mortality in the critical first years of life.<\/jats:p><\/jats:sec>","DOI":"10.1111\/dmcn.15638","type":"journal-article","created":{"date-parts":[[2023,5,18]],"date-time":"2023-05-18T04:54:26Z","timestamp":1684385666000},"page":"1607-1616","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":29,"title":["Initiating stiripentol before 2\u2009years of age in patients with Dravet syndrome is safe and beneficial against status 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