{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,22]],"date-time":"2026-07-22T03:42:28Z","timestamp":1784691748064,"version":"3.55.0"},"reference-count":18,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2018,8,10]],"date-time":"2018-08-10T00:00:00Z","timestamp":1533859200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100011102","name":"Seventh Framework Programme","doi-asserted-by":"publisher","award":["602391"],"award-info":[{"award-number":["602391"]}],"id":[{"id":"10.13039\/100011102","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Epilepsia"],"published-print":{"date-parts":[[2018,9]]},"abstract":"Summary<\/jats:title>Tuberous sclerosis complex (TSC<\/jats:styled-content>) is the most common cause of West syndrome (WS<\/jats:styled-content>). Currently available treatment options are ineffective in the majority of affected infants and\/or associated with potential serious side effects. Based on the assumption that mTOR<\/jats:styled-content> overactivation results in increased neuroexcitability in TSC<\/jats:styled-content>, mTOR inhibitors have been studied as antiseizure therapy. As a result, everolimus recently received approval for the adjunctive treatment of patients aged \u22652\u00a0years with refractory TSC<\/jats:styled-content>\u2010associated focal and secondary generalized seizures. However, efficacy and safety data for infants with TSC<\/jats:styled-content>\u2010associated WS<\/jats:styled-content> are still lacking. Therefore, a prospective open\u2010label observational study was initiated at our center, to evaluate everolimus add\u2010on treatment in infants with TSC<\/jats:styled-content>\u2010associated WS<\/jats:styled-content>, previously refractory to standard treatment. For this preliminary report, data from four male infants with TSC<\/jats:styled-content>2 and a median observation period of 13 (range = 8\u201042) months after treatment initiation were analyzed. Two infants showed electroclinical remission until day 14 after everolimus treatment initiation. In one additional infant, hypsarrhythmia resolved. No relapse after initial response was documented. Developmental progress improved in three infants. Tolerability was similar to that described in older children. According to our preliminary results, everolimus appears to have the potential to treat successfully both spasms and hypsarrhythmia in infants with TSC<\/jats:styled-content>\u2010associated WS<\/jats:styled-content>, contributing to better developmental progress.<\/jats:p>","DOI":"10.1111\/epi.14529","type":"journal-article","created":{"date-parts":[[2018,8,11]],"date-time":"2018-08-11T05:18:48Z","timestamp":1533964728000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":28,"title":["Everolimus in infants with tuberous sclerosis complex\u2013related West syndrome: First results from a single\u2010center prospective observational study"],"prefix":"10.1111","volume":"59","author":[{"given":"Sharon","family":"Samueli","sequence":"first","affiliation":[{"name":"Department of Pediatric and Adolescent Medicine Medical University of Vienna Vienna Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1518-8646","authenticated-orcid":false,"given":"Anastasia","family":"Dressler","sequence":"additional","affiliation":[{"name":"Department of Pediatric and Adolescent Medicine Medical University of Vienna Vienna Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gudrun","family":"Gr\u00f6ppel","sequence":"additional","affiliation":[{"name":"Department of Pediatric and Adolescent Medicine Medical University of Vienna Vienna Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Theresa","family":"Scholl","sequence":"additional","affiliation":[{"name":"Department of Pediatric and Adolescent Medicine Medical University of Vienna Vienna Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martha","family":"Feucht","sequence":"additional","affiliation":[{"name":"Department of Pediatric and Adolescent Medicine Medical University of 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