Epidemiology of Lupus: Longitudinal Studies in Population-Based Cohorts - 2022

项目摘要 系统性红斑狼疮是一种全身性自身免疫性疾病，是终身的，可以危及生命。 高达15-20%的SLE患者在儿童期（<18岁）被诊断。尽管据估计， 在美国，约有10，000名儿童患有儿科发病的SLE（pSLE），其自然史、治疗和 对这一弱势儿童群体的成果尚未明确界定。 我们建议使用完善的儿童关节炎和风湿病研究联盟（CARRA） pSLE登记研究，目前包括870例纵向随访长达10年的患者（目标是招募 1300-1400名参与者），以彻底评估生物学/疾病相关因素，以及非 生物因素，可能影响短期和长期pSLE结果。该提案将解决关键差距 pSLE知识，包括狼疮性肾炎治疗的描述以及 神经精神pSLE，pSLE短期和长期发病率的两个主要原因。此外，我们将 描述患者报告的重要结局，包括疼痛、生活质量和残疾，以及长期 结果，包括向成年的过渡。 研究结果将通过患者/家庭伙伴关系传播，目的是发展未来 改善pSLE患者的健康和福祉的干预措施。

PROJECT SUMMARY Systemic Lupus Erythematosus is a systemic autoimmune condition which is life long and can be life threatening. Up to 15-20% of all SLE patients are diagnosed in childhood (<18 years of age). Although it is estimated that up to 10,000 children in the United States have pediatric-onset SLE (pSLE), the natural history, treatment and outcomes for this vulnerable population of children have not been well defined. We propose to use the well-established Childhood Arthritis and Rheumatology Research Alliance (CARRA) pSLE registry, which currently includes 870 patients followed longitudinally for up to ten years (goal of enrolling 1300-1400 participants), in order to thoroughly assess the biologic/disease related factors, as well as the non- biologic factors, which can impact short and long-term pSLE outcomes. This proposal will address critical gaps in pSLE knowledge including description of treatments for lupus nephritis and the prevalence and outcomes of neuropsychiatric pSLE, the two primary causes of pSLE short and long-term morbidity. In addition, we will describe important patient reported outcomes including pain, quality of life and disability, and long-term outcomes, including transition to adulthood. Research findings will be disseminated through patient/family partnerships with a goal of developing future interventions to improve the health and well- being of patients with pSLE.