PERIPHERAL MARKERS OF IMMUNE DYSREGULATION AS A MARKER OF JUVENILE DERMATOMYOSIT

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。幼年型皮肌炎（JDMS）是一种罕见的多系统自身免疫性疾病，每年约有3/100万儿童受到影响。 虽然JDMS的病因尚不清楚，但遗传易感宿主中的某些感染可能引发免疫介导的肌炎[1-4]。尽管广泛的研究，幼年皮肌炎的发病机制仍然不明。此外，尽管JDMS的治疗在过去40年中得到了改善，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。目的本研究旨在检测JDMS患者外周血淋巴细胞特异性标志物。此外，我们将确定这些外周免疫学标志物与JDMS患者的临床病程之间是否存在相关性。 具体目的1：进行一项前瞻性队列研究，以描述幼年型皮肌炎（JDMS）的外周淋巴细胞标志物。 目的：检测JDMS患儿外周血淋巴细胞表面标志物的表达。 具体目标2：外周血淋巴细胞标志物与临床疾病的相关性 JDMS患者的活动。 我们假设幼年型皮肌炎（JDMS）患者外周血淋巴细胞标志物发生改变，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、对治疗的反应和自身免疫的缓解相关联。

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.