{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,11]],"date-time":"2026-10-11T02:09:51Z","timestamp":1791684591291,"version":"4.3.4"},"reference-count":6,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2022,3,7]],"date-time":"2022-03-07T00:00:00Z","timestamp":1646611200000},"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":["Euro J of Neurology"],"published-print":{"date-parts":[[2022,4]]},"abstract":"Abstract<\/jats:title>Background and purpose<\/jats:title>To provide further evidence for sirolimus, a mammalian target of rapamycin inhibitor, as a treatment strategy for patients with inclusion body myositis (IBM).<\/jats:p><\/jats:sec>Methods<\/jats:title>We acquired longitudinal clinical data and immunological assessments of CD8+<\/jats:sup> T\u2010cell subsets in peripheral blood for evaluation of potential anti\u2010inflammatory treatment effects of sirolimus.<\/jats:p><\/jats:sec>Results<\/jats:title>Therapy with sirolimus 2\u00a0mg\/day by mouth led to rapid and sustained clinical improvement of motor symptoms for an observation period of more than 1\u00a0year. Treatment was well tolerated, with no occurrence of adverse effects. We did not observe a meaningful alteration of CD8+<\/jats:sup> T\u2010cell subsets in our patient after 9 and 12\u00a0months compared to baseline.<\/jats:p><\/jats:sec>Conclusions<\/jats:title>The significant and persistent clinical improvement highlights the use of sirolimus as a potential treatment option in patients with IBM. In light of the lack of immunological treatment effects observed for cytotoxic CD8+<\/jats:sup> T cells, further studies should investigate the potential myoprotective effects of sirolimus.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ene.15231","type":"journal-article","created":{"date-parts":[[2022,3,7]],"date-time":"2022-03-07T13:23:12Z","timestamp":1646659392000},"page":"1284-1287","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Sirolimus leads to rapid and sustained clinical improvement of motor deficits in a patient with inclusion body myositis"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3080-2277","authenticated-orcid":false,"given":"Marc","family":"Pawlitzki","sequence":"first","affiliation":[{"name":"Department of Neurology, Institute of Translational Neurology University of M\u00fcnster M\u00fcnster Germany"},{"name":"Department of Neurology University Hospital D\u00fcsseldorf D\u00fcsseldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Nelke","sequence":"additional","affiliation":[{"name":"Department of Neurology University Hospital D\u00fcsseldorf D\u00fcsseldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melanie","family":"Korsen","sequence":"additional","affiliation":[{"name":"Department of Neurology University Hospital D\u00fcsseldorf D\u00fcsseldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sven G.","family":"Meuth","sequence":"additional","affiliation":[{"name":"Department of Neurology University Hospital D\u00fcsseldorf D\u00fcsseldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tobias","family":"Ruck","sequence":"additional","affiliation":[{"name":"Department of Neurology University Hospital D\u00fcsseldorf D\u00fcsseldorf Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,3,7]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awl020"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2665-9913(20)30280-0"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2013.08.007"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00401-015-1384-5"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1177\/1759720X19886494"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0122888"}],"container-title":["European Journal of Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/ene.15231","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1111\/ene.15231","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/ene.15231","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,25]],"date-time":"2023-08-25T22:57:07Z","timestamp":1693004227000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/ene.15231"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,3,7]]},"references-count":6,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2022,4]]}},"alternative-id":["10.1111\/ene.15231"],"URL":"https:\/\/doi.org\/10.1111\/ene.15231","archive":["Portico"],"relation":{},"ISSN":["1351-5101","1468-1331"],"issn-type":[{"value":"1351-5101","type":"print"},{"value":"1468-1331","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,3,7]]},"assertion":[{"value":"2021-11-27","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-12-21","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-03-07","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}