AUTHOR=Kostik Mikhail M. , Raupov Rinat K. , Suspitsin Evgeny N. , Isupova Eugenia A. , Gaidar Ekaterina V. , Gabrusskaya Tatyana V. , Kaneva Maria A. , Snegireva Ludmila S. , Likhacheva Tatyana S. , Miulkidzhan Rimma S. , Kosmin Artem V. , Tumakova Anastasia V. , Masalova Vera V. , Dubko Margarita F. , Kalashnikova Olga V. , Aksentijevich Ivona , Chasnyk Vyacheslav G. TITLE=The Safety and Efficacy of Tofacitinib in 24 Cases of Pediatric Rheumatic Diseases: Single Centre Experience JOURNAL=Frontiers in Pediatrics VOLUME=10 YEAR=2022 URL=https://www.frontiersin.org/journals/pediatrics/articles/10.3389/fped.2022.820586 DOI=10.3389/fped.2022.820586 ISSN=2296-2360 ABSTRACT=
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).
To evaluate the safety and efficacy of tofacitinib in children with different rheumatic diseases.
We extracted information from 24 children with the following diagnosis: JIA (
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 (
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.