دورية أكاديمية
أعرض في EDS

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

التفاصيل البيبلوغرافية
العنوان: The Safety and Efficacy of Tofacitinib in 24 Cases of Pediatric Rheumatic Diseases: Single Centre Experience.
المؤلفون: 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.
المصدر: Frontiers in pediatrics [Front Pediatr] 2022 Feb 08; Vol. 10, pp. 820586. Date of Electronic Publication: 2022 Feb 08 (Print Publication: 2022).
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: Frontiers Media SA Country of Publication: Switzerland NLM ID: 101615492 Publication Model: eCollection Cited Medium: Print ISSN: 2296-2360 (Print) Linking ISSN: 22962360 NLM ISO Abbreviation: Front Pediatr Subsets: PubMed not MEDLINE
أسماء مطبوعة: Original Publication: Lausanne : Frontiers Media SA, [2013]-
مستخلص: 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.)
References: N Engl J Med. 2012 Aug 9;367(6):495-507. (PMID: 22873530)
Nat Genet. 2014 May;46(5):503-509. (PMID: 24686847)
J Clin Invest. 2018 Jul 2;128(7):3041-3052. (PMID: 29649002)
Clin Pharmacokinet. 2019 Jan;58(1):15-37. (PMID: 29752633)
Int Immunopharmacol. 2020 Mar;80:106210. (PMID: 31972425)
Annu Rev Med. 2015;66:311-28. (PMID: 25587654)
Autoimmun Rev. 2019 Nov;18(11):102390. (PMID: 31520803)
N Engl J Med. 2001 Apr 5;344(14):1031-7. (PMID: 11287972)
Arthritis Care Res (Hoboken). 2011 Jul;63(7):929-36. (PMID: 21717596)
Ann Rheum Dis. 2020 Jun;79(6):842-844. (PMID: 32079571)
J Am Acad Dermatol. 2017 Jul;77(1):167-170. (PMID: 28619556)
Ann Rheum Dis. 2015 Feb;74(2):333-40. (PMID: 25431052)
World J Pediatr. 2020 Feb;16(1):44-51. (PMID: 31377974)
J Am Acad Dermatol. 2019 Apr;80(4):1164-1166. (PMID: 30630026)
Cancer Res. 2018 Jul 1;78(13):3458-3468. (PMID: 29716915)
Clin Immunol. 2020 Mar;212:108273. (PMID: 31626957)
Ann Rheum Dis. 2020 Oct 28;:. (PMID: 33115762)
N Engl J Med. 2012 Mar 1;366(9):799-807. (PMID: 22375971)
Dermatol Ther (Heidelb). 2021 Jun;11(3):733-750. (PMID: 33856640)
Pediatrics. 2019 May;143(5):. (PMID: 30948682)
Arthritis Rheumatol. 2021 May;73(5):858-865. (PMID: 33258553)
Pediatr Rheumatol Online J. 2019 May 2;17(1):19. (PMID: 31046790)
Arthritis Res Ther. 2011;13(6):249. (PMID: 22192711)
Rheumatology (Oxford). 2021 Sep 1;60(9):3986-4000. (PMID: 34117886)
Arthritis Rheumatol. 2021 Jul;73(7):1108-1123. (PMID: 34101376)
J Clin Immunol. 2018 Nov;38(8):844-846. (PMID: 30443754)
Lancet. 2021 Nov 27;398(10315):1984-1996. (PMID: 34767764)
Pediatr Res. 2020 Mar;87(4):745-752. (PMID: 31641281)
Ital J Pediatr. 2017 Jun 14;43(1):56. (PMID: 28615030)
J Eur Acad Dermatol Venereol. 2020 Dec;34(12):2898-2901. (PMID: 32531131)
Gastroenterol Hepatol. 2021 Jan;44(1):39-48. (PMID: 32829958)
Ann Rheum Dis. 2019 Mar;78(3):431-433. (PMID: 30282666)
Ann Rheum Dis. 2020 Jun;79(6):685-699. (PMID: 31969328)
Rheumatology (Oxford). 2019 Feb 1;58(Suppl 1):i43-i54. (PMID: 30806709)
Arthritis Care Res (Hoboken). 2019 Oct;71(10):1285-1299. (PMID: 31436026)
Arthritis Rheum. 2009 May 15;61(5):658-66. (PMID: 19405003)
Nat Rev Drug Discov. 2017 Dec;16(12):843-862. (PMID: 29104284)
Ann Rheum Dis. 2020 Jun;79(6):700-712. (PMID: 32434812)
Pediatr Dermatol. 2021 Jan;38(1):103-108. (PMID: 33099833)
Pediatr Rheumatol Online J. 2020 May 12;18(1):37. (PMID: 32398023)
فهرسة مساهمة: Keywords: JAK-inhibitors; alopecia; interferon type-I; interferonopathy; juvenile dermatomyositis; juvenile idiopathic arthritis; tofacitinib
تواريخ الأحداث: Date Created: 20220225 Latest Revision: 20220502
رمز التحديث: 20231215
مُعرف محوري في PubMed: PMC8861449
DOI: 10.3389/fped.2022.820586
PMID: 35211430
قاعدة البيانات: MEDLINE
الوصف
تدمد:2296-2360
DOI:10.3389/fped.2022.820586