PERIPHERAL MARKERS OF IMMUNE DYSREGULATION AS A MARKER OF JUVENILE DERMATOMYOSIT

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。青少年皮肌炎（JDMS）是一种罕见的多系统自身免疫性疾病，每年约有100万儿童中有3人患病。虽然JDMS的病因尚不清楚，但遗传易感宿主的某些感染可能引发免疫介导的肌炎[1-4]。尽管进行了广泛的研究，但青少年皮肌炎的发病机制仍不清楚。此外，尽管在过去40年里，JDMS的治疗方法有所改善，但患者仍然遭受与疾病过程相关的相当大的发病率和与药物治疗相关的毒性。目的：本研究将检测JDMS患者的特异性外周淋巴细胞标志物。此外，我们将确定这些外周免疫标志物与JDMS患者的临床病程之间是否存在相关性。目的1：开展一项前瞻性队列研究，描述青少年皮肌炎（JDMS）的外周血淋巴细胞标志物。目的：测定JDMS患儿外周血淋巴细胞表面标志物的表达。特异性目的2：将外周淋巴细胞标志物与JDMS患者的临床疾病活动性联系起来。我们假设周围淋巴细胞标记物在青少年皮肌炎（JDMS）中发生改变，并在疾病结束时恢复。基于这一假设，我们可能能够将外周免疫标志物与JDMS患者的疾病活动性、治疗反应和自身免疫缓解联系起来。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Introduction Juvenile Dermatomyositis (JDMS) is a rare, multisystem autoimmune disease that affects about 3 in 1 million children each year. Although the cause of JDMS is unknown, certain infections in genetically susceptible hosts may trigger immune mediated myositis [1-4]. Despite extensive research, the pathogenesis of Juvenile Dermatomyositis remains unknown. In addition, although treatments for JDMS have improved over the past 4 decades, patients continue to suffer considerable morbidity related to the disease process and toxicities related to medical treatments. Objectives The proposed study will examine specific peripheral lymphocyte markers in patients with JDMS. Further, we will determine whether there is a correlation between these peripheral immunologic markers and the clinical course of patients with JDMS. Aims Specific Aim 1: To conduct a prospective cohort study to describe peripheral lymphocyte markers in Juvenile Dermatomyositis (JDMS). To determine the surface markers expressed on the peripheral lymphocytes of children with JDMS. Specific Aim 2: To correlate peripheral lymphocyte markers with the clinical disease activity in JDMS patients. We hypothesize that peripheral lymphocyte markers are altered in Juvenile Dermatomyositis (JDMS) and regained at the end of disease. Based on this hypothesis we may be able to correlate peripheral immune markers with disease activity, response to therapy and remission of autoimmunity in patients with JDMS.