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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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