The Safety and Efficacy of Tofacitinib in 24 Cases of Pediatric Rheumatic Diseases: Single Centre Experience.

Autor:	Kostik MM; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Raupov RK; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia.; Saint Petersburg State Health Care Establishment the City Hospital No 40 of the Resort District, Saint-Petersburg, Russia., Suspitsin EN; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia.; Laboratory of the Molecular Oncology, N.N. Petrov's Institute of Oncology, Saint-Petersburg, Russia., Isupova EA; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Gaidar EV; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Gabrusskaya TV; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Kaneva MA; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Snegireva LS; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Likhacheva TS; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Miulkidzhan RS; Laboratory of the Molecular Oncology, N.N. Petrov's Institute of Oncology, Saint-Petersburg, Russia., Kosmin AV; Laboratory of the Molecular Oncology, N.N. Petrov's Institute of Oncology, Saint-Petersburg, Russia., Tumakova AV; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Masalova VV; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Dubko MF; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Kalashnikova OV; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia., Aksentijevich I; Inflammatory Disease Section, National Human Genome Research Institute, Bethesda, MD, United States., Chasnyk VG; Hospital Pediatry, Saint-Petersburg State Pediatric Medical University, Saint-Petersburg, Russia.
Jazyk:	angličtina
Zdroj:	Frontiers in pediatrics [Front Pediatr] 2022 Feb 08; Vol. 10, pp. 820586. Date of Electronic Publication: 2022 Feb 08 (Print Publication: 2022).
DOI:	10.3389/fped.2022.820586
Abstrakt:	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). Aim of Study: To evaluate the safety and efficacy of tofacitinib in children with different rheumatic diseases. Material and Methods: We extracted information from 24 children with the following diagnosis: JIA ( n = 15), undifferentiated systemic autoinflammatory diseases (SAIDs) ( n = 7), and juvenile dermatomyositis (JDM) ( n = 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)-a significant improvement of symptoms and disease activity, or no response (NR)-no changes in disease activity. Results: 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 ( n = 2), hypercholesterolemia ( n = 1), lymphadenitis ( n = 1). Conclusion: 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. Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. (Copyright © 2022 Kostik, Raupov, Suspitsin, Isupova, Gaidar, Gabrusskaya, Kaneva, Snegireva, Likhacheva, Miulkidzhan, Kosmin, Tumakova, Masalova, Dubko, Kalashnikova, Aksentijevich and Chasnyk.)
Databáze:	MEDLINE
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