Personalised medicine in juvenile dermatomyositis: From novel insights in disease mechanisms to changes in clinical practice
- PMID: 39174374
- DOI: 10.1016/j.berh.2024.101976
Personalised medicine in juvenile dermatomyositis: From novel insights in disease mechanisms to changes in clinical practice
Abstract
Juvenile dermatomyositis is characterized by childhood-onset chronic inflammation of the muscles and skin, with potential involvement of other organs. Patients are at risk for long-term morbidity due to insufficient disease control and steroid-related toxicity. Personalised treatment is challenged by a lack of validated tools that can reliably predict treatment response and monitor ongoing (subclinical) inflammation, and by a lack of evidence regarding the best choice of medication for individual patients. A better understanding of the involved disease mechanisms could reveal potential biomarkers and novel therapeutic targets. In this review, we highlight the most relevant immune and non-immune mechanisms, elucidating the effects of interferon overexpression on tissue alongside the interplay between the interferon signature, mitochondrial function, and immune cells. We review mechanism-based biomarkers that are promising for clinical implementation, and the latest advances in targeted therapy development. Finally, we discuss key steps needed for translating these discoveries into clinical practice.
Keywords: Biomarkers; Interferon; Juvenile dermatomyositis; Personalised medicine.
Copyright © 2024. Published by Elsevier Ltd.
Conflict of interest statement
Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Saskia R. Veldkamp reports financial support was provided by Prinses Beatrix Spierfonds, the Dutch (neuro)muscular disease foundation, the Netherlands. Saskia R. Veldkamp reports financial support was provided by BAS stichting. Saskia R. Veldkamp reports a relationship with JDM Working Party (Basic Science Lead) of the Pediatric Rheumatology European Society that includes: board membership. Annet van Royen-Kerkhof reports a relationship with Global Conference on Myositis 2024 (Scientific Committee) that includes: board membership. Marc H.A. Jansen reports a relationship with Dutch Association for Pediatrics, Myositis guideline Working Group that includes: board membership. If there are other authors, they declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
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