{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T16:11:16Z","timestamp":1791389476389,"version":"4.3.3"},"reference-count":42,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2022,2,8]],"date-time":"2022-02-08T00:00:00Z","timestamp":1644278400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100006769","name":"Russian Science Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100006769","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Pediatr."],"abstract":"<jats:p>JAK-inhibitors are small molecules blocking the JAK-STAT pathway that have proven effective in the treatment of different immune-mediated diseases in adults and juvenile idiopathic arthritis (JIA).<\/jats:p><jats:sec><jats:title>Aim of Study<\/jats:title><jats:p>To evaluate the safety and efficacy of tofacitinib in children with different rheumatic diseases.<\/jats:p><\/jats:sec><jats:sec><jats:title>Material and Methods<\/jats:title><jats:p>We extracted information from 24 children with the following diagnosis: JIA (<jats:italic>n<\/jats:italic> = 15), undifferentiated systemic autoinflammatory diseases (SAIDs) (<jats:italic>n<\/jats:italic> = 7), and juvenile dermatomyositis (JDM) (<jats:italic>n<\/jats:italic> = 2) who have been treated with tofacitinib for a period of longer than 6 months. The treatment outcomes were classified according to the opinion of the attending physicians as having a complete response (CR), i.e., the absence of disease activity, or a partial response (PR)\u2014a significant improvement of symptoms and disease activity, or no response (NR)\u2014no changes in disease activity.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>CR was achieved in 10\/24 patients; 7\/15 among JIA patients, 1\/2 among JDM patients, 4\/7 among SAID patients, and PR in 5\/15 of JIA, 1\/2 of JDM, and 3\/7 of SAID patients. Three non-responders with JIA discontinued tofacitinib. Corticosteroids were successfully tapered off in 11\/14 patients and discontinued in 2\/14 patients. Four patients had side effects not requiring treatment discontinuation: liver enzyme elevation (<jats:italic>n<\/jats:italic> = 2), hypercholesterolemia (<jats:italic>n<\/jats:italic> = 1), lymphadenitis (<jats:italic>n<\/jats:italic> = 1).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>JAK-inhibitors are effective new therapies for the treatment of multiple immune-mediated diseases. Our experience has shown the best results in patients with JIA and JIA-associated alopecia, and type I interferonopathies. More data from randomized controlled clinical trials are needed to use JAK-inhibitors safely in pediatric rheumatic diseases.<\/jats:p><\/jats:sec>","DOI":"10.3389\/fped.2022.820586","type":"journal-article","created":{"date-parts":[[2022,2,8]],"date-time":"2022-02-08T08:08:55Z","timestamp":1644307735000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":37,"title":["The Safety and Efficacy of Tofacitinib in 24 Cases of Pediatric Rheumatic Diseases: Single Centre Experience"],"prefix":"10.3389","volume":"10","author":[{"given":"Mikhail M.","family":"Kostik","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rinat K.","family":"Raupov","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Evgeny N.","family":"Suspitsin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugenia 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