幼年皮肌炎的个性化医疗：从疾病机制的新见解到临床实践的改变。

IF 4.5 2区医学 Q1 RHEUMATOLOGY

Best Practice & Research in Clinical Rheumatology Pub Date : 2024-09-01 DOI:10.1016/j.berh.2024.101976

Saskia R. Veldkamp , Femke van Wijk , Annet van Royen-Kerkhof , Marc HA. Jansen

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引用次数: 0

摘要

幼年皮肌炎的特征是儿童时期发病的肌肉和皮肤慢性炎症，并可能累及其他器官。由于疾病控制不佳和类固醇相关毒性，患者面临长期发病的风险。由于缺乏能够可靠预测治疗反应和监测持续（亚临床）炎症的有效工具，以及缺乏针对个体患者最佳药物选择的证据，个性化治疗面临挑战。更好地了解相关疾病机制可以揭示潜在的生物标志物和新的治疗靶点。在这篇综述中，我们重点介绍了最相关的免疫和非免疫机制，阐明了干扰素过度表达对组织的影响，以及干扰素特征、线粒体功能和免疫细胞之间的相互作用。我们回顾了有望用于临床的基于机制的生物标记物，以及靶向疗法开发的最新进展。最后，我们将讨论将这些发现转化为临床实践所需的关键步骤。

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

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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来源期刊

Best Practice & Research in Clinical Rheumatology 医学-风湿病学

CiteScore

9.40

自引率

0.00%

发文量

审稿时长

27 days

期刊介绍： Evidence-based updates of best clinical practice across the spectrum of musculoskeletal conditions. Best Practice & Research: Clinical Rheumatology keeps the clinician or trainee informed of the latest developments and current recommended practice in the rapidly advancing fields of musculoskeletal conditions and science. The series provides a continuous update of current clinical practice. It is a topical serial publication that covers the spectrum of musculoskeletal conditions in a 4-year cycle. Each topic-based issue contains around 200 pages of practical, evidence-based review articles, which integrate the results from the latest original research with current clinical practice and thinking to provide a continuous update. Each issue follows a problem-orientated approach that focuses on the key questions to be addressed, clearly defining what is known and not known. The review articles seek to address the clinical issues of diagnosis, treatment and patient management. Management is described in practical terms so that it can be applied to the individual patient. The serial is aimed at the physician in both practice and training.