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Open Access 01-12-2021 | Juvenile Dermatomyositis | Review

JAK inhibitors: a potential treatment for JDM in the context of the role of interferon-driven pathology

Authors: Meredyth G. Ll Wilkinson, Claire T. Deakin, Charalampia Papadopoulou, Despina Eleftheriou, Lucy R. Wedderburn

Published in: Pediatric Rheumatology | Issue 1/2021

Abstract

Juvenile Idiopathic Inflammatory Myopathies (IIM) are a group of rare diseases that are heterogeneous in terms of pathology that can include proximal muscle weakness, associated skin changes and systemic involvement. Despite options for treatment, many patients continue to suffer resistant disease and lasting side-effects. Advances in the understanding of the immunopathology and genetics underlying IIM may specify new therapeutic targets, particularly where conventional treatment has not achieved a clinical response. An upregulated type I interferon signature is strongly associated with disease and could be a prime target for developing more specific therapeutics. There are multiple components of the IFN pathway that could be targeted for blockade therapy.

Downstream of the cytokine receptor complexes are the Janus kinase-signal transducers and activators of transcription (JAK-STAT) pathway, which consists of JAK1–3, TYK2, and STAT1–6. Therapeutic inhibitors have been developed to target components of this pathway. Promising results have been observed in case studies reporting the use of the JAK inhibitors, Baricitinib, Tofacitinib and Ruxolitinib in the treatment of refractory Juvenile Dermatomyositis (JDM). There is still the question of safety and efficacy for the use of JAK inhibitors in JDM that need to be addressed by clinical trials. Here we review the future for the use of JAK inhibitors as a treatment for JDM.

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Title: JAK inhibitors: a potential treatment for JDM in the context of the role of interferon-driven pathology
Authors: Meredyth G. Ll Wilkinson
Claire T. Deakin
Charalampia Papadopoulou
Despina Eleftheriou
Lucy R. Wedderburn
Publication date: 01-12-2021
Publisher: BioMed Central
Keywords: Juvenile Dermatomyositis
Pathology
Dermatomyositis
Interferon
Ruxolitinib
Published in: Pediatric Rheumatology / Issue 1/2021
Electronic ISSN: 1546-0096
DOI: https://doi.org/10.1186/s12969-021-00637-8

At a glance: The STEP trials

Springer Medicine

JAK inhibitors: a potential treatment for JDM in the context of the role of interferon-driven pathology

Abstract

At a glance: The STEP trials

Springer Medicine

Abstract

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