Short-term effectiveness of baricitinib in children with refractory and/or severe juvenile dermatomyositis

ObjectiveTo determine the short-term effectiveness safety of baricitinib in children with refractory and/or severe juvenile dermatomyositis (rsJDM) in a real-world setting.MethodsThis was a single-center retrospective study, including 20 children with rsJDM. They were all treated using baricitinib c...

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Main Authors: Zhaoling Wang, Qi Zheng, Wenjie Xuan, Xisheng Xu, Meiping Lu, Jianqiang Wu, Lixia Zou, Yiping Xu, Xuefeng Xu
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-09-01
Series:Frontiers in Pediatrics
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fped.2022.962585/full
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author Zhaoling Wang
Qi Zheng
Wenjie Xuan
Xisheng Xu
Meiping Lu
Jianqiang Wu
Lixia Zou
Yiping Xu
Xuefeng Xu
author_facet Zhaoling Wang
Qi Zheng
Wenjie Xuan
Xisheng Xu
Meiping Lu
Jianqiang Wu
Lixia Zou
Yiping Xu
Xuefeng Xu
author_sort Zhaoling Wang
collection DOAJ
description ObjectiveTo determine the short-term effectiveness safety of baricitinib in children with refractory and/or severe juvenile dermatomyositis (rsJDM) in a real-world setting.MethodsThis was a single-center retrospective study, including 20 children with rsJDM. They were all treated using baricitinib combined with steroids and other immunosuppressive agents. The childhood myositis assessment scale (CMAS) and PRINTO remission criteria were used to evaluate the disease severity and treatment outcome at 0, 4, 12, and 24 weeks after initiation of baricitinib.ResultsThe skin rash improved in 95% of patients (19/20) at week 24, with a significant decrease of skin-DAS at weeks 12 (6.0 vs. 2.0, p < 0.05] and week 24 [6.0 vs. 1.0, p < 0.05) by median statistics. The CMAS score increased significantly at week 12 (41.0 [29.0, 44.0] vs. 46.0 [42.0, 52.0], p < 0.05) and week 24 (41.0 [29.0, 44.0] vs. 50.0 [45.0, 52.0], p < 0.05), as did the manual muscle testing (MMT)-8 score at week 24 (73.0 [610, 76.0] vs. 79.0 [77.0, 80.0], p < 0.05). At 24 weeks, the complete response (CR) and partial response (PR) were achieved in 75% (15/20) and 15% (3/20), respectively. The dose of corticosteroids (CS) decreased by 37% from the baseline (0.53 [0.42, 1.00] mg/kg) to week 12 (0.33 [0.18, 0.40] mg/kg) (p < 0.05), and by 49% at week 24 (p < 0.05). No serious side effects were observed.ConclusionBaricitinib combined with traditional immunosuppressants treatment was efficacious in rsJDM. Add-on therapy of baricitinib was helpful for tapering CS dose. No serious side effects were observed in this study.
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spelling doaj.art-68a9c32f89b9424f9cd5a851d9f98a1e2022-12-22T04:03:23ZengFrontiers Media S.A.Frontiers in Pediatrics2296-23602022-09-011010.3389/fped.2022.962585962585Short-term effectiveness of baricitinib in children with refractory and/or severe juvenile dermatomyositisZhaoling Wang0Qi Zheng1Wenjie Xuan2Xisheng Xu3Meiping Lu4Jianqiang Wu5Lixia Zou6Yiping Xu7Xuefeng Xu8Department of Rheumatology Immunology and Allergy, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, ChinaDepartment of Rheumatology Immunology and Allergy, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, ChinaDepartment of Pediatric, Shaoxing People’s Hospital, Shaoxing, ChinaDepartment of Rheumatology Immunology and Allergy, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, ChinaDepartment of Rheumatology Immunology and Allergy, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, ChinaDepartment of Rheumatology Immunology and Allergy, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, ChinaDepartment of Rheumatology Immunology and Allergy, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, ChinaDepartment of Rheumatology Immunology and Allergy, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, ChinaDepartment of Rheumatology Immunology and Allergy, Children’s Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, ChinaObjectiveTo determine the short-term effectiveness safety of baricitinib in children with refractory and/or severe juvenile dermatomyositis (rsJDM) in a real-world setting.MethodsThis was a single-center retrospective study, including 20 children with rsJDM. They were all treated using baricitinib combined with steroids and other immunosuppressive agents. The childhood myositis assessment scale (CMAS) and PRINTO remission criteria were used to evaluate the disease severity and treatment outcome at 0, 4, 12, and 24 weeks after initiation of baricitinib.ResultsThe skin rash improved in 95% of patients (19/20) at week 24, with a significant decrease of skin-DAS at weeks 12 (6.0 vs. 2.0, p < 0.05] and week 24 [6.0 vs. 1.0, p < 0.05) by median statistics. The CMAS score increased significantly at week 12 (41.0 [29.0, 44.0] vs. 46.0 [42.0, 52.0], p < 0.05) and week 24 (41.0 [29.0, 44.0] vs. 50.0 [45.0, 52.0], p < 0.05), as did the manual muscle testing (MMT)-8 score at week 24 (73.0 [610, 76.0] vs. 79.0 [77.0, 80.0], p < 0.05). At 24 weeks, the complete response (CR) and partial response (PR) were achieved in 75% (15/20) and 15% (3/20), respectively. The dose of corticosteroids (CS) decreased by 37% from the baseline (0.53 [0.42, 1.00] mg/kg) to week 12 (0.33 [0.18, 0.40] mg/kg) (p < 0.05), and by 49% at week 24 (p < 0.05). No serious side effects were observed.ConclusionBaricitinib combined with traditional immunosuppressants treatment was efficacious in rsJDM. Add-on therapy of baricitinib was helpful for tapering CS dose. No serious side effects were observed in this study.https://www.frontiersin.org/articles/10.3389/fped.2022.962585/fullbaricitinibjuvenile dermatomyositisrefractoryseveretreatment
spellingShingle Zhaoling Wang
Qi Zheng
Wenjie Xuan
Xisheng Xu
Meiping Lu
Jianqiang Wu
Lixia Zou
Yiping Xu
Xuefeng Xu
Short-term effectiveness of baricitinib in children with refractory and/or severe juvenile dermatomyositis
Frontiers in Pediatrics
baricitinib
juvenile dermatomyositis
refractory
severe
treatment
title Short-term effectiveness of baricitinib in children with refractory and/or severe juvenile dermatomyositis
title_full Short-term effectiveness of baricitinib in children with refractory and/or severe juvenile dermatomyositis
title_fullStr Short-term effectiveness of baricitinib in children with refractory and/or severe juvenile dermatomyositis
title_full_unstemmed Short-term effectiveness of baricitinib in children with refractory and/or severe juvenile dermatomyositis
title_short Short-term effectiveness of baricitinib in children with refractory and/or severe juvenile dermatomyositis
title_sort short term effectiveness of baricitinib in children with refractory and or severe juvenile dermatomyositis
topic baricitinib
juvenile dermatomyositis
refractory
severe
treatment
url https://www.frontiersin.org/articles/10.3389/fped.2022.962585/full
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