Cohort Study of Pediatric SLE

描述（由申请人提供）：该K23导师奖的主要目标是获得临床研究所需的必要技能，成为儿科风湿病学领域的独立研究者，这是一个服务严重不足的领域。当地儿童系统性红斑狼疮（SLE）的临床和实验室研究环境非常出色，包括具有建立的互动研究项目的高度成就的导师。该提案的科学目的是为了进一步推进苏莱博士的最终职业目标：改善小儿SLE患者的护理和临床结果。与她的导师以及临床和实验室合作者密切合作。该项目将建立一个儿童SLE队列（SA 1）。她将定义小儿SLE患者疾病活动性（SA 2）和疾病损害（SA 3）的预测因子。然后，她将这些预测因素与约翰斯·霍普金斯大学已经建立和定义明确的成人SLE队列进行比较。我们预计这两组之间的临床和实验室值会有差异。例如，在儿科患者中，实验室值的变化（如白蛋白和补体水平降低，或dsDNA升高）可能只发生在疾病发作之前。这种模式在成人患者中可能没有观察到。这些在相同建立的，前瞻性成人和儿科队列中的比较将提供重要的见解，并且据我们所知，从未进行过。即使没有观察到差异，建立前瞻性儿童SLE队列也很重要，因为它将阐明当前治疗时代儿童SLE的自然史。该队列的建立将有助于确定未来干预的目标，以改善这些患者的预后。在已建立的成人SLE队列中进行的研究已经实现了这一目标。我们期望，在K23奖项期间，苏莱博士将建立的儿科队列将在儿童SLE中发挥同样的价值，并将成为未来儿童风湿病独立临床研究的杰出载体。外行语言：儿童风湿病学的目标是预防我们还不能“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，我们需要确定疾病活动的预测因子，这样我们就可以干预治疗，将疾病损害降到最低。我们建议建立一个儿童SLE队列，这将有助于更好地了解儿童狼疮，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.