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Pediatric Rheumatology
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Open Access 25-02-2025 | Juvenile Dermatomyositis | Letter to the Editor

Successful treatment of an anti-MDA5 antibody-positive Juvenile Dermatomyositis patient with refractory interstitial lung disease using tofacitinib

Authors: Valentina Natoli, Serena Palmeri, Ana Isabel Rebollo-Giménez, Caterina Matucci-Cerinic, Paola Bocca, Roberta Caorsi, Stefano Volpi, Riccardo Papa, Alessandro Consolaro, Marco Gattorno, Angelo Ravelli, Silvia Rosina

Published in: Pediatric Rheumatology | Issue 1/2025

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Excerpt

Juvenile dermatomyositis (JDM) is a rare autoimmune disorder affecting children, mainly characterized by skin rash and muscle weakness [1]. Anti-MDA5 antibody-positive JDM represents a distinct disease phenotype with a high risk of developing life-threatening progressive interstitial lung disease (ILD) [2]. The pivotal role of the type I interferon (IFN) pathway in the pathogenesis of anti-MDA5 positive JDM has prompted the exploration of Janus kinase (JAK) inhibitors as a therapeutic option [3]. Nevertheless, while the efficacy and safety of JAK inhibitors have been established in clinical trials including adult-onset anti-MDA5-antibody positive DM [4], evidence in paediatric cases with progressive or refractory ILD is limited. In this report we describe the efficacy and safety of tofacitinib, a JAK 1/3 inhibitor, in treating an anti-MDA5-positive JDM young girl with refractory ILD, highlighting the potential of JAK inhibitors as targeted therapy in these patients. …
Literature
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Papadopoulou C, Chew C, Wilkinson MGL, McCann L, Wedderburn LR. Juvenile idiopathic inflammatory myositis: an update on pathophysiology and clinical care. Nat Rev Rheumatol. 2023;19(6):343–62.CrossRefPubMedPubMedCentral
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Kobayashi I, Okura Y, Yamada M, Kawamura N, Kuwana M, Ariga T. Anti-melanoma differentiation-associated gene 5 antibody is a diagnostic and predictive marker for interstitial lung diseases associated with juvenile dermatomyositis. J Pediatr. 2011;158(4):675–7.CrossRefPubMed
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Paik JJ, Lubin G, Gromatzky A, Paul N, Mudd J, Ponda MP, Christopher-Stine L. Use of JAK inhibitors in Dermatomyositis: a systematic literature review. Clin Exp Rheumatol. 2023;41(2):348.PubMed
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Wang Y, Luo J, Lv X, Li Y, An Q, Mo L, et al. Tofacitinib for new-onset adult patients with anti-melanoma differentiation-associated 5 gene antibody positive dermatomyositis. Clin Rheumatol. 2023;42(7):1847–53.CrossRefPubMed
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Kurasawa K, Arai S, Namiki Y, Tanaka A, Takamura Y, Owada T, et al. Tofacitinib for refractory interstitial lung diseases in anti-melanoma differentiation-associated 5 gene antibody-positive dermatomyositis. Rheumatology (Oxford). 2018;57(12):2114–9.CrossRefPubMed
Metadata
Title
Successful treatment of an anti-MDA5 antibody-positive Juvenile Dermatomyositis patient with refractory interstitial lung disease using tofacitinib
Authors
Valentina Natoli
Serena Palmeri
Ana Isabel Rebollo-Giménez
Caterina Matucci-Cerinic
Paola Bocca
Roberta Caorsi
Stefano Volpi
Riccardo Papa
Alessandro Consolaro
Marco Gattorno
Angelo Ravelli
Silvia Rosina
Publication date
25-02-2025
Publisher
BioMed Central
Published in
Pediatric Rheumatology / Issue 1/2025
Electronic ISSN: 1546-0096
DOI
https://doi.org/10.1186/s12969-024-01044-5

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