Janus kinase inhibitor significantly improved rash and muscle strength in juvenile dermatomyositis

Ding, Yuchuan; Huang, Baozhen; Wang, Yuan; Hou, Jun; Chen, Ying; Zhou, Zixiang; Li, Jianguo

doi:10.1136/annrheumdis-2020-218582

Cited by 23 publications

(20 citation statements)

References 6 publications

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“…They have shown efficacy in adult rheumatic conditions and are included in clinical guidelines of rheumatoid arthritis ( 15 ), psoriatic arthritis ( 16 ), ankylosing spondylitis ( 17 ). Currently, the experience of JAK-inhibitors in the pediatric population is scarce and limited to a single randomized placebo-controlled study in JIA and several studies in small groups of patients with immune-mediated diseases ( 18 , 19 ). The largest randomized, double-blind, placebo-controlled withdrawal study of tofacitinib in JIA showed that patients with polyarticular course of JIA treated with tofacitinib have significantly lower rate of disease flare at 44 weeks compared to placebo-group (29% vs 52.9%, p=0.0031).…”

Section: Discussionmentioning

confidence: 99%

“…Ding and colleges reported successful management of 24/25 children refractory JDM with JAK-inhibitors (18 patients were treated with ruxolitinib and seven patients with tofacitinib). Complete skin rash resolution was achieved in 67% cases, 28% of patients discontinued steroids, and 7/10 patients with decreased muscle strength had improved CMAS score ( 18 ). The prospective, open-label clinical trial of tofacitinib in 10 adults with refractory dermatomyositis demonstrated efficacy of JAK-inhibitors in all patients ( 32 ).…”

Section: Discussionmentioning

confidence: 99%

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

et al. 2022

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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 StudyTo evaluate the safety and efficacy of tofacitinib in children with different rheumatic diseases.Material and MethodsWe 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.ResultsCR 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).ConclusionJAK-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.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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

et al. 2022

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“…Recently, advances in the understanding of the immunopathology and genetics in JDM may help to specify new treatment approaches, especially when conventional therapy is not successful. An upregulated type 1 interferon signature is strongly associated with JDM, which could present the possibility for the treatment of most severe cases with JAK inhibitors ( 17 , 18 ). However, the safety and efficacy of this therapy still need to be studied in clinical trials.…”

Section: Discussionmentioning

confidence: 99%

Is Anti-NXP2 Autoantibody a Risk Factor for Calcinosis and Poor Outcome in Juvenile Dermatomyositis Patients? Case Series

et al. 2022

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Juvenile dermatomyositis (JDM) has a wide spectrum of clinical presentations. In the last decade, several myositis-specific antibodies have been identified in patients with JDM and connected with specific organ involvement or specific clinical picture. It has been published that the presence of anti-NXP2 autoantibodies presents a risk for calcinosis in patients with JDM. We aimed to investigate the prevalence of calcinosis and response to the treatment in JDM patients with anti-NXP2. In a retrospective, multinational, multicenter study, data on 26 JDM (19 F, 7 M) patients with positive anti-NXP2 were collected. The mean age at disease presentation was 6.5 years (SD 3.7), the median diagnosis delay was 4 months (range 0.5–27 months). Patients were divided into two groups (A and B) based on the presence of calcinosis, which occurred in 42% of anti-NXP2 positive JDM patients (group A). Four patients already had calcinosis at presentation, one developed calcinosis after 4 months, and 6 developed calcinosis later in the disease course (median 2 years, range 0.8–7.8). The differences in laboratory results were not statistically significant between the groups. The mean age at disease presentation (5.2/7.5 years) trended toward being younger in group A. Children with calcinosis were treated with several combinations of drugs. In four cases, rituximab and, in one case, anti-TNF alpha agents were used successfully. Disease outcome (by evaluation of the treating physician) was excellent in four, good in two, stable in two, and poor in three patients. None of the patients from group B had a poor disease outcome. In conclusion, JDM patients with anti-NXP2 are prone to develop calcinosis, especially if they present with the disease early, before 5 years of age. The development of calcinosis is associated with worse disease outcomes. The combination of several immunomodulatory drugs and biologic drugs can stop calcinosis progression; however, there are no evidence-based therapies for treating calcinosis in JDM patients.

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“…JAK inhibitors competitively inhibit the ATP binding site on JH1. [5][6][7] Tofacitinib (CP-690550), formerly known as tasocitinib, is the first FDA-approved JAK inhibitor for treatment of an autoimmune disease, and was initially studied as an antirejection agent in organ transplantation. Tofacitinib primarily inhibits JAK1 and JAK3, which are found on T cells, B cells, natural killer cells and mast cells.…”

Section: Biology Of Tofacitinibmentioning

confidence: 99%

Tofacitinib in paediatric dermatoses: a narrative review

Agrawal

Panda

Das

2022

Clin Experimental Derm

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Tofacitinib has revolutionized the treatment of numerous dermatological conditions in different age groups. However, evidence for its effectiveness, safety and tolerability in the paediatric population is limited. We performed a literature search, which showed that oral tofacitinib is a reliable option in refractory juvenile dermatomyositis, severe alopecia areata, atopic dermatitis and psoriasis. Topical tofacitinib is an effective option in vitiligo and halo naevus. The risk-benefit ratio should be assessed prior to consideration of this molecule. In this narrative review, we have attempted to present a summary of the evidence of using tofacitinib (oral and topical) in paediatric dermatoses.

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Janus kinase inhibitor significantly improved rash and muscle strength in juvenile dermatomyositis

Cited by 23 publications

References 6 publications

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

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

Is Anti-NXP2 Autoantibody a Risk Factor for Calcinosis and Poor Outcome in Juvenile Dermatomyositis Patients? Case Series

Tofacitinib in paediatric dermatoses: a narrative review

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