Cohort Study of Pediatric SLE

描述(由申请者提供)：这项有指导的K23奖项的主要目标是在临床研究中获得必要的技能，这些技能是成为儿科风湿病学的独立研究员所需的，这是一个严重缺乏服务的领域。儿童系统性红斑狼疮(SLE)的临床和实验室研究的当地环境非常出色，包括具有既定互动研究计划的高度成功的导师。这项建议的科学目的是为了促进Sule博士的最终职业目标：改善SLE儿童患者的护理和临床结果。苏勒博士将与她的导师以及临床和实验室合作者密切合作，建立一个儿童系统性红斑狼疮队列(SA 1)。她将定义儿童系统性红斑狼疮患者疾病活动性(SA2)和疾病损害(SA3)的预测因子。然后，她将把这些预测因素与约翰霍普金斯大学已经建立的、定义明确的成人系统性红斑狼疮队列进行比较。我们预计这两组人的临床和实验室价值会有所不同。例如，在儿科患者中，实验室数值的变化(如白蛋白和补体水平降低，或dsDNA增加)可能只发生在疾病爆发之前。这种模式在成人患者中可能不会观察到。这些在相同建立的、预期的成人和儿科队列中的比较将提供重要的见解，据我们所知，从未进行过。即使没有观察到差异，建立预期的儿童系统性红斑狼疮队列也是重要的，因为它将阐明当前治疗时代儿童系统性红斑狼疮的自然历史。这一队列的建立将允许确定未来干预措施的目标，以改善这些患者的结果。在已建立的成人系统性红斑狼疮队列中进行的研究已经实现了这一目标。我们预计，Sule博士将在K23奖项期间建立的儿科队列将在儿科SLE方面具有同样的价值，并将成为未来儿科风湿病独立临床研究的杰出载体。通俗语言：儿科风湿病的目标是预防我们还无法“治愈”的自身免疫性疾病的活动和损害，以便儿童能够过上健康的生活。为了做到这一点，我们需要定义疾病活动的预测因素，以便我们能够进行干预，将疾病损害降至最低。我们建议建立一个儿童系统性红斑狼疮队列，这将导致对儿童狼疮的更好的了解，并改变这些儿童的生活。

DESCRIPTION (provided by applicant): The primary objective of this mentored K23 award is to acquire the necessary skills in clinical research that are needed to become an independent investigator in Pediatric Rheumatology, a critically underserved area. The local environment for clinical and laboratory research in pediatric systemic lupus erythematosus (SLE) is outstanding, and includes highly accomplished mentors with established, interactive research programs. The scientific aims of this proposal are intended to further Dr. Sule's ultimate career goal: to improve the care and clincal outcomes for pediatric patients with SLE. Working closely with her mentors as well as clinical and laboratory collaborators,Dr. Sule will establish a pediatric SLE cohort (SA 1). She will define predictors of disease activity (SA 2) and disease damage (SA 3) in pediatric patients with SLE. She will then compare these predictors to the already established and well-defined adult SLE cohort at Johns Hopkins. We expect that there will be differences in clinical and laboratory values between these two groups. For example, in pediatric patients, changes in laboratory values (such as decreased albumin and complement levels, or increased dsDNA) may only occur prior to disease flares. This pattern may not be observed in adult patients. These comparisons in identically established, prospective adult and pediatric cohorts will provide significant insights, and, to our knowledge, have never been performed. Even if no differences are observed, the establishment of the prospective pediatric SLE cohort is important as it will elucidate the natural history of pediatric SLE in the current treatment era. The establishment of this cohort will allow identification of targets for future interventions to improve outcomes in these patients. Studies in established adult SLE cohorts have accomplished this goal. We expect that the pediatric cohort that Dr. Sule will establish during the K23 award will bejust as valuable in pediatric SLE and will be an outstanding vehicle for future independent clinical research in Pediatric Rheumatology. Lay Language: The goal in Pediatric Rheumatology is to prevent activity and damage from autoimmune diseases that we cannot yet "cure" so that children can lead healthy lives. In order to do this, we need to define predictors of disease activity so that we can intervene with treatments that minimize disease damage. We propose to establish a pediatric SLE cohort, which will lead to a better understanding of pediatric lupus and make a difference in these childrens1 lives.