{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,13]],"date-time":"2026-04-13T23:47:53Z","timestamp":1776124073503,"version":"3.50.1"},"reference-count":46,"publisher":"MDPI AG","issue":"1","license":[{"start":{"date-parts":[[2023,1,3]],"date-time":"2023-01-03T00:00:00Z","timestamp":1672704000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Medical Research Agency, Poland","award":["2019\/ABM\/01\/00034"],"award-info":[{"award-number":["2019\/ABM\/01\/00034"]}]},{"name":"Medical Research Agency, Poland","award":["2020\/ABM\/01\/00054"],"award-info":[{"award-number":["2020\/ABM\/01\/00054"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background: mTOR inhibitors are a novel pharmacotherapy recommended for subependymal giant astrocytomas, refractory epilepsy, and the treatment of the other clinical manifestations of tuberous sclerosis complex (TSC). Clinical trials on everolimus proved it to be effective and safe in children. Despite its common use in clinical practice, the research on sirolimus is limited. This study is the first to determine and assess the severity of the adverse effects (AEs) of sirolimus administered to children with TSC under two years of age. Methods: We performed a bicenter retrospective data analysis of medical records of individuals with TSC who initiated therapy with sirolimus under the age of two. Results: Twenty-one patients were included in the study. At least one AE was reported in all participants. The most prevalent AEs were anemia, thrombocytosis, and hyperlipidemia. Infections and mouth ulcerations, often reported in the studies on older patients, were infrequent and of mild or moderate grade. Conclusions: Adverse effects associated with sirolimus use in infants and young children with TSC are frequent yet not life- or health-threatening. Further multicenter prospective clinical trials should determine the long-term safety of sirolimus.<\/jats:p>","DOI":"10.3390\/jcm12010365","type":"journal-article","created":{"date-parts":[[2023,1,3]],"date-time":"2023-01-03T02:05:53Z","timestamp":1672711553000},"page":"365","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["Safety of Sirolimus in Patients with Tuberous Sclerosis Complex under Two Years of Age\u2014A Bicenter Retrospective Study"],"prefix":"10.3390","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0776-193X","authenticated-orcid":false,"given":"Dominika","family":"\u015amia\u0142ek","sequence":"first","affiliation":[{"name":"Department of Pediatric Neurology, Medical University of Warsaw, 02-091 Warsaw, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3350-6326","authenticated-orcid":false,"given":"Sergiusz","family":"J\u00f3\u017awiak","sequence":"additional","affiliation":[{"name":"Research Department, The Children\u2019s Memorial Health Institute, 04-736 Warsaw, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katarzyna","family":"Kotulska","sequence":"additional","affiliation":[{"name":"Department of Neurology and Epileptology, The Children\u2019s Memorial Health Institute, 04-736 Warsaw, Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2023,1,3]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1016\/S0140-6736(08)61279-9","article-title":"Tuberous Sclerosis","volume":"372","author":"Curatolo","year":"2008","journal-title":"Lancet"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1111\/j.1528-1167.2007.01035.x","article-title":"Tuberous Sclerosis Complex and Epilepsy: Recent Developments and Future Challenges","volume":"48","author":"Holmes","year":"2007","journal-title":"Epilepsia"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1042\/BJ20080281","article-title":"The TSC1-TSC2 Complex: A Molecular Switchboard Controlling Cell Growth","volume":"412","author":"Huang","year":"2008","journal-title":"Biochem. 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