Personalised medicine in juvenile dermatomyositis: From novel insights in disease mechanisms to changes in clinical practice

被引:0
|
作者
Veldkamp, Saskia R. [1 ]
van Wijk, Femke [1 ]
van Royen-Kerkhof, Annet [2 ]
Jansen, Marc H. A. [2 ,3 ]
机构
[1] Univ Utrecht, Univ Med Ctr Utrecht, Ctr Translat Immunol, Utrecht, Netherlands
[2] Univ Med Ctr Utrecht, Wilhelmina Childrens Hosp, Dept Pediat Immunol & Rheumatol, Utrecht, Netherlands
[3] Lundlaan 6, NL-3584 EA Utrecht, Netherlands
来源
关键词
Juvenile dermatomyositis; Personalised medicine; Interferon; Biomarkers; IDIOPATHIC INFLAMMATORY MYOPATHIES; INDUCIBLE GENE-EXPRESSION; MUSCLE BIOPSY; PERIPHERAL-BLOOD; AMYOPATHIC DERMATOMYOSITIS; ADULT DERMATOMYOSITIS; MONOCLONAL-ANTIBODY; LYMPHOCYTE SUBSETS; PROGNOSTIC-FACTORS; IMMUNE-MECHANISMS;
D O I
10.1016/j.berh.2024.101976
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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.
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页数:18
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