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Open Access 14-04-2025 | Juvenile Dermatomyositis | Research Letter

Tofacitinib: a promising agent for the treatment of persistent rashes in juvenile dermatomyositis

Authors: Zhao-Yu Chen, Tian-Nan Zhang, Ji Li, Zhen-Jie Zhang, Hong-Mei Song

Published in: World Journal of Pediatrics

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Excerpt

Juvenile dermatomyositis (JDM) is a chronic pediatric autoimmune disease characterized by proximal muscle weakness and distinctive skin manifestations [1, 2]. The first-line treatment for JDM involves the combination of glucocorticoids with either methotrexate (MTX) or cyclosporine [3, 4]. For severe and refractory juvenile dermatomyositis, options such as tacrolimus, mycophenolate mofetil (MMF), cyclophosphamide, rituximab, tumor necrosis factor-α inhibitors, and intravenous immunoglobulin (IVIG) may be considered [47]. Recently, several studies have reported successful management with Janus kinase (JAK) inhibitor in patients with severe or refractory JDM [810]. However, in clinical practice, although the standard treatment regimen, glucocorticoids plus MTX, effectively manages myositis in patients with JDM, it often fails to adequately address persistent rashes [11]. Although a previous survey conducted by the Childhood Arthritis and Rheumatology Research Alliance identified IVIG, MMF, and cyclosporine as typical treatments for JDM patients with persistent skin rash even after complete resolution of muscle involvement, there is still a lack of support from prospective data collection and statistical methods that account for nonrandom treatment assignment [11]. To date, no appropriate treatment regimen for such persistent rashes in JDM patients has been reported, making these cases particularly challenging. The question arises whether JAK inhibitors would exhibit similarly beneficial therapeutic effects in JDM patients with controlled myositis activity but persistent rashes. In this report, we detail our therapeutic experience with tofacitinib (TOF) in eight patients with refractory JDM. Although these patients had controlled myositis, they continued to suffer from persistent rashes, which were relieved significantly following the introduction of TOF. …
Literature
1.
go back to reference Rider LG, Aggarwal R, Pistorio A, Bayat N, Erman B, Feldman BM, et al. 2016 American college of rheumatology/European league against rheumatism criteria for minimal, moderate, and major clinical response in juvenile dermatomyositis: an international myositis assessment and clinical studies group/paediatric rheumatology international trials organisation collaborative initiative. Arthritis Rheumatol. 2017;69:911–23.CrossRefPubMedPubMedCentral Rider LG, Aggarwal R, Pistorio A, Bayat N, Erman B, Feldman BM, et al. 2016 American college of rheumatology/European league against rheumatism criteria for minimal, moderate, and major clinical response in juvenile dermatomyositis: an international myositis assessment and clinical studies group/paediatric rheumatology international trials organisation collaborative initiative. Arthritis Rheumatol. 2017;69:911–23.CrossRefPubMedPubMedCentral
2.
go back to reference Bellutti Enders F, Bader-Meunier B, Baildam E, Constantin T, Dolezalova P, Feldman BM, et al. Consensus-based recommendations for the management of juvenile dermatomyositis. Ann Rheum Dis. 2017;76:329–40.CrossRefPubMed Bellutti Enders F, Bader-Meunier B, Baildam E, Constantin T, Dolezalova P, Feldman BM, et al. Consensus-based recommendations for the management of juvenile dermatomyositis. Ann Rheum Dis. 2017;76:329–40.CrossRefPubMed
3.
go back to reference Ruperto N, Pistorio A, Oliveira S, Zulian F, Cuttica R, Ravelli A, et al. Paediatric Rheumatology International Trials Organisation (PRINTO). Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: a randomised trial. Lancet. 2016;387:671–8.CrossRefPubMed Ruperto N, Pistorio A, Oliveira S, Zulian F, Cuttica R, Ravelli A, et al. Paediatric Rheumatology International Trials Organisation (PRINTO). Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: a randomised trial. Lancet. 2016;387:671–8.CrossRefPubMed
4.
go back to reference Wu JQ, Lu MP, Reed AM. Juvenile dermatomyositis: advances in clinical presentation, myositis-specific antibodies and treatment. World J Pediatr. 2020;16:31–43.CrossRefPubMed Wu JQ, Lu MP, Reed AM. Juvenile dermatomyositis: advances in clinical presentation, myositis-specific antibodies and treatment. World J Pediatr. 2020;16:31–43.CrossRefPubMed
5.
go back to reference Deakin CT, Campanilho-Marques R, Simou S, Moraitis E, Wedderburn LR, Pullenayegum E, et al. Efficacy and safety of cyclophosphamide treatment in severe juvenile dermatomyositis shown by marginal structural modeling. Arthritis Rheumatol. 2018;70:785–93.CrossRefPubMedPubMedCentral Deakin CT, Campanilho-Marques R, Simou S, Moraitis E, Wedderburn LR, Pullenayegum E, et al. Efficacy and safety of cyclophosphamide treatment in severe juvenile dermatomyositis shown by marginal structural modeling. Arthritis Rheumatol. 2018;70:785–93.CrossRefPubMedPubMedCentral
6.
go back to reference Goswami RP, Haldar SN, Chatterjee M, Vij P, van der Kooi AJ, Lim J, et al. Efficacy and safety of intravenous and subcutaneous immunoglobulin therapy in idiopathic inflammatory myopathy: a systematic review and meta-analysis. Autoimmun Rev. 2022;21:102997.CrossRefPubMed Goswami RP, Haldar SN, Chatterjee M, Vij P, van der Kooi AJ, Lim J, et al. Efficacy and safety of intravenous and subcutaneous immunoglobulin therapy in idiopathic inflammatory myopathy: a systematic review and meta-analysis. Autoimmun Rev. 2022;21:102997.CrossRefPubMed
7.
go back to reference Campanilho-Marques R, Deakin CT, Simou S, Papadopoulou C, Wedderburn LR, Pilkington CA, et al. Retrospective analysis of infliximab and adalimumab treatment in a large cohort of juvenile dermatomyositis patients. Arthritis Res Ther. 2020;22:79.CrossRefPubMedPubMedCentral Campanilho-Marques R, Deakin CT, Simou S, Papadopoulou C, Wedderburn LR, Pilkington CA, et al. Retrospective analysis of infliximab and adalimumab treatment in a large cohort of juvenile dermatomyositis patients. Arthritis Res Ther. 2020;22:79.CrossRefPubMedPubMedCentral
8.
go back to reference Huang B, Wang X, Niu Y, Ding Y, Wang X, Tan Q, et al. Long-term follow-up of Janus-kinase inhibitor and novel active disease biomarker in juvenile dermatomyositis. Rheumatology. 2023;62:1227–37.CrossRefPubMed Huang B, Wang X, Niu Y, Ding Y, Wang X, Tan Q, et al. Long-term follow-up of Janus-kinase inhibitor and novel active disease biomarker in juvenile dermatomyositis. Rheumatology. 2023;62:1227–37.CrossRefPubMed
9.
go back to reference Yu Z, Wang L, Quan M, Zhang T, Song H. Successful management with Janus kinase inhibitor tofacitinib in refractory juvenile dermatomyositis: a pilot study and literature review. Rheumatology. 2021;60:1700–7.CrossRefPubMed Yu Z, Wang L, Quan M, Zhang T, Song H. Successful management with Janus kinase inhibitor tofacitinib in refractory juvenile dermatomyositis: a pilot study and literature review. Rheumatology. 2021;60:1700–7.CrossRefPubMed
10.
go back to reference Ding Y, Huang B, Wang Y, Hou J, Chi Y, Zhou Z, et al. Janus kinase inhibitor significantly improved rash and muscle strength in juvenile dermatomyositis. Ann Rheum Dis. 2021;80:543–5.CrossRefPubMed Ding Y, Huang B, Wang Y, Hou J, Chi Y, Zhou Z, et al. Janus kinase inhibitor significantly improved rash and muscle strength in juvenile dermatomyositis. Ann Rheum Dis. 2021;80:543–5.CrossRefPubMed
11.
go back to reference Huber A, Kim S, Reed A, Carrasco R, Feldman B, Hong S, et al.; Juvenile Dermatomyositis Research Committee of the Childhood Arthritis and Rheumatology Research Alliance. Childhood arthritis and rheumatology research alliance consensus clinical treatment plans for juvenile dermatomyositis with persistent skin rash. J Rheumatol. 2017;44:110–6.CrossRefPubMed Huber A, Kim S, Reed A, Carrasco R, Feldman B, Hong S, et al.; Juvenile Dermatomyositis Research Committee of the Childhood Arthritis and Rheumatology Research Alliance. Childhood arthritis and rheumatology research alliance consensus clinical treatment plans for juvenile dermatomyositis with persistent skin rash. J Rheumatol. 2017;44:110–6.CrossRefPubMed
12.
go back to reference Bohan A, Peter JB. Polymyositis and dermatomyositis (first of two parts). N Engl J Med. 1975;292:344–7.CrossRefPubMed Bohan A, Peter JB. Polymyositis and dermatomyositis (first of two parts). N Engl J Med. 1975;292:344–7.CrossRefPubMed
13.
go back to reference Bohan A, Peter JB. Polymyositis and dermatomyositis (second of two parts). N Engl J Med. 1975;292:403–7.CrossRefPubMed Bohan A, Peter JB. Polymyositis and dermatomyositis (second of two parts). N Engl J Med. 1975;292:403–7.CrossRefPubMed
14.
go back to reference You H, Xu D, Zhao J, Li J, Wang Q, Tian X, et al. JAK inhibitors: prospects in connective tissue diseases. Clin Rev Allergy Immunol. 2020;59:334–51.CrossRefPubMed You H, Xu D, Zhao J, Li J, Wang Q, Tian X, et al. JAK inhibitors: prospects in connective tissue diseases. Clin Rev Allergy Immunol. 2020;59:334–51.CrossRefPubMed
15.
go back to reference Ruperto N, Pistorio A, Oliveira S, Zulian F, Cuttica R, Ravelli A, et al. Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: a randomised trial. Lancet. 2016;387:671–8.CrossRefPubMed Ruperto N, Pistorio A, Oliveira S, Zulian F, Cuttica R, Ravelli A, et al. Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: a randomised trial. Lancet. 2016;387:671–8.CrossRefPubMed
17.
go back to reference Ekholm L, Kahlenberg JM, Barbasso Helmers S, Tjärnlund A, Yalavarthi S, Zhao W, et al. Dysfunction of endothelial progenitor cells is associated with the type I IFN pathway in patients with polymyositis and dermatomyositis. Rheumatology (Oxford). 2016;55:1987–92.CrossRefPubMed Ekholm L, Kahlenberg JM, Barbasso Helmers S, Tjärnlund A, Yalavarthi S, Zhao W, et al. Dysfunction of endothelial progenitor cells is associated with the type I IFN pathway in patients with polymyositis and dermatomyositis. Rheumatology (Oxford). 2016;55:1987–92.CrossRefPubMed
18.
go back to reference McCann LJ, Livermore P, Wilkinson MGL, Wedderburn LR. Juvenile dermatomyositis. where are we now? Clin Exp Rheumatol. 2022;40:394–403.CrossRefPubMed McCann LJ, Livermore P, Wilkinson MGL, Wedderburn LR. Juvenile dermatomyositis. where are we now? Clin Exp Rheumatol. 2022;40:394–403.CrossRefPubMed
19.
go back to reference Sabbagh S, Almeida de Jesus A, Hwang S, Kuehn HS, Kim H, Jung L, et al. Treatment of anti-MDA5 autoantibody-positive juvenile dermatomyositis using tofacitinib. Brain. 2019;142:e59.CrossRefPubMedPubMedCentral Sabbagh S, Almeida de Jesus A, Hwang S, Kuehn HS, Kim H, Jung L, et al. Treatment of anti-MDA5 autoantibody-positive juvenile dermatomyositis using tofacitinib. Brain. 2019;142:e59.CrossRefPubMedPubMedCentral
20.
go back to reference Zhang J, Sun L, Shi X, Li S, Liu C, Li X, et al. Janus kinase inhibitor, tofacitinib, in refractory juvenile dermatomyositis: a retrospective multi-central study in China. Arthritis Res Ther. 2023;25:204.CrossRefPubMedPubMedCentral Zhang J, Sun L, Shi X, Li S, Liu C, Li X, et al. Janus kinase inhibitor, tofacitinib, in refractory juvenile dermatomyositis: a retrospective multi-central study in China. Arthritis Res Ther. 2023;25:204.CrossRefPubMedPubMedCentral
21.
go back to reference Sener S, Cam V, Ozen S, Batu ED. Treatment with Janus kinase inhibitors in juvenile dermatomyositis: a review of the literature. Semin Arthritis Rheum. 2024;66:152426.CrossRefPubMed Sener S, Cam V, Ozen S, Batu ED. Treatment with Janus kinase inhibitors in juvenile dermatomyositis: a review of the literature. Semin Arthritis Rheum. 2024;66:152426.CrossRefPubMed
Metadata
Title
Tofacitinib: a promising agent for the treatment of persistent rashes in juvenile dermatomyositis
Authors
Zhao-Yu Chen
Tian-Nan Zhang
Ji Li
Zhen-Jie Zhang
Hong-Mei Song
Publication date
14-04-2025
Publisher
Springer Nature Singapore
Published in
World Journal of Pediatrics
Print ISSN: 1708-8569
Electronic ISSN: 1867-0687
DOI
https://doi.org/10.1007/s12519-025-00901-x

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