{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,15]],"date-time":"2026-07-15T01:52:27Z","timestamp":1784080347993,"version":"3.55.0"},"reference-count":25,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2019,10,20]],"date-time":"2019-10-20T00:00:00Z","timestamp":1571529600000},"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":[[2019,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To assess the long\u2010term efficacy and tolerability of stiripentol (STP) as an adjunctive treatment in different forms of refractory epilepsies.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The medical records of all individuals consecutively treated with STP as add\u2010on therapy for refractory epilepsies, irrespective of their being focal, generalized, or both, and followed at Meyer Children's Hospital between January 2007 and May 2018, were reviewed. The drug scheme administration consisted of a starting dose of STP of 10\u201015\u00a0mg\/kg\/d with increments every week, up to a maximum of 50\u00a0mg\/kg\/d, based on both age and weight. Etiology of epilepsy was codified as structural, genetic, infectious, immune, metabolic, and unknown. Responders were defined as patients who achieved a seizure frequency reduction of \u226550%. Retention rate was defined as the probability of continuing STP without additional therapy. Tolerability was assessed by reporting adverse events.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 132 individuals aged from 5\u00a0months to 43\u00a0years received add\u2010on STP, including 30 patients with Dravet syndrome (DS). The median follow\u2010up was 14.8\u00a0months (range = 4\u00a0months\u201018\u00a0years, interquartile range = 25.72). Twenty\u2010nine individuals (22%) received more than two antiepileptic drugs. Benzodiazepines, mainly clobazam, were the most commonly used add\u2010on drugs. Sixty\u2010six patients (50%) were responders, and 13 of them (9.8%) were seizure\u2010free. Responder rate was higher in the genetic etiology group (57%), especially in DS (18\/30; 60%), and in patients with refractory focal onset epilepsy without bilateral tonic\u2010clonic seizures (5\/15; 33%). The median relapse\u2010free survival was 27\u00a0months in the 66 responders. The median time to STP failure was 24.6\u00a0months in all 132 individuals.<\/jats:p><\/jats:sec><jats:sec><jats:title>Significance<\/jats:title><jats:p>This study confirms the long\u2010term efficacy of add\u2010on STP treatment in patients with different types of refractory epilepsies, including focal onset epilepsy without bilateral tonic\u2010clonic seizures. Further confirmations based on prospectively designed studies are required to confirm STP efficacy in focal epilepsy.<\/jats:p><\/jats:sec>","DOI":"10.1111\/epi.16363","type":"journal-article","created":{"date-parts":[[2019,10,20]],"date-time":"2019-10-20T14:07:58Z","timestamp":1571580478000},"page":"2255-2262","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":19,"title":["Long\u2010term efficacy of add\u2010on stiripentol treatment in children, adolescents, and young adults with refractory epilepsies: A single center prospective observational study"],"prefix":"10.1111","volume":"60","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8754-7214","authenticated-orcid":false,"given":"Anna","family":"Rosati","sequence":"first","affiliation":[{"name":"Neuroscience Center of Excellence Meyer Children's Hospital University of Florence Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Boncristiano","sequence":"additional","affiliation":[{"name":"Neuroscience Center of Excellence Meyer Children's Hospital University of Florence Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Viola","family":"Doccini","sequence":"additional","affiliation":[{"name":"Neuroscience Center of Excellence Meyer Children's Hospital University of Florence Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Pugi","sequence":"additional","affiliation":[{"name":"Clinical Trial Office Meyer Children's Hospital Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8920-9078","authenticated-orcid":false,"given":"Tiziana","family":"Pisano","sequence":"additional","affiliation":[{"name":"Neuroscience Center of Excellence Meyer Children's Hospital University of Florence Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo","family":"Lenge","sequence":"additional","affiliation":[{"name":"Clinical Trial Office Meyer Children's Hospital Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salvatore","family":"De Masi","sequence":"additional","affiliation":[{"name":"Clinical Trial Office Meyer Children's Hospital Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7272-7079","authenticated-orcid":false,"given":"Renzo","family":"Guerrini","sequence":"additional","affiliation":[{"name":"Neuroscience Center of Excellence Meyer Children's Hospital University of Florence Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2019,10,20]]},"reference":[{"key":"e_1_2_7_2_1","unstructured":"https:\/\/www.ema.europa.eu\/documents\/product-information\/diacomit-epar-product-information_en.pdf. Accessed December 4 2018."},{"key":"e_1_2_7_3_1","unstructured":"https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/nda\/2018\/206709Orig1s000 207223Orig1s000TOC.cfm. Accessed December 4 2018."},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1528-1167.2011.03008.x"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/jnr.23688"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0009-9236(97)90044-8"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1528-1157.1999.tb02048.x"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(00)03157-3"},{"issue":"8","key":"e_1_2_7_9_1","first-page":"155","article-title":"Stiripentol in severe myoclonic epilepsy in infancy (SMEI): a placebo\u2010controlled Italian trial","volume":"43","author":"Guerrini R","year":"2002","journal-title":"Epilepsia"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0929-693X(02)00090-8"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1528-1167.2009.02179.x"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.12303"},{"key":"e_1_2_7_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.eplepsyres.2014.02.008"},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.eplepsyres.2015.03.012"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.eplepsyres.2016.05.012"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.3988\/jcn.2018.14.1.22"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.13704"},{"key":"e_1_2_7_18_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.14536"},{"key":"e_1_2_7_19_1","doi-asserted-by":"publisher","DOI":"10.1177\/0883073818811538"},{"key":"e_1_2_7_20_1","doi-asserted-by":"publisher","DOI":"10.1177\/08830738060210062101"},{"key":"e_1_2_7_21_1","doi-asserted-by":"crossref","unstructured":"BrigoF StortiM.Antiepileptic drugs for the treatment of severe myoclonic epilepsy in infancy. Cochrane Database Syst Rev. 2013;(11):CD010483.","DOI":"10.1002\/14651858.CD010483.pub2"},{"key":"e_1_2_7_22_1","doi-asserted-by":"crossref","unstructured":"BrigoF StortiM.Stiripentol for focal refractory epilepsy. Cochrane Database Syst Rev. 2014;(1):CD009887.","DOI":"10.1002\/14651858.CD009887.pub2"},{"key":"e_1_2_7_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.13670"},{"key":"e_1_2_7_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/epi.13709"},{"key":"e_1_2_7_25_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1528-1167.2009.02397.x"},{"key":"e_1_2_7_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.yebeh.2010.04.001"}],"container-title":["Epilepsia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/epi.16363","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/epi.16363","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/epi.16363","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,8]],"date-time":"2023-09-08T13:45:42Z","timestamp":1694180742000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/epi.16363"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,10,20]]},"references-count":25,"journal-issue":{"issue":"11","published-print":{"date-parts":[[2019,11]]}},"alternative-id":["10.1111\/epi.16363"],"URL":"https:\/\/doi.org\/10.1111\/epi.16363","archive":["Portico"],"relation":{},"ISSN":["0013-9580","1528-1167"],"issn-type":[{"value":"0013-9580","type":"print"},{"value":"1528-1167","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,10,20]]},"assertion":[{"value":"2019-05-08","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-09-16","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-10-20","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}