Cohort Study of Pediatric SLE

描述（由申请人提供）：这个指导K23奖的主要目标是获得必要的技能，在临床研究，需要成为一个独立的研究人员在儿科流变学，一个严重不足的领域。在儿科系统性红斑狼疮（SLE）的临床和实验室研究的当地环境是杰出的，包括高度成就的导师与既定的，互动的研究计划。本提案的科学目的旨在进一步推动Sule博士的最终职业目标：改善SLE儿科患者的护理和临床结局。Sule博士将与她的导师以及临床和实验室合作者密切合作，建立一个儿科SLE队列（SA 1）。她将定义SLE儿童患者疾病活动性（SA 2）和疾病损害（SA 3）的预测因子。然后，她将这些预测因子与约翰霍普金斯已经建立的明确定义的成人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）可能仅发生在疾病发作之前。在成人患者中可能不会观察到这种模式。在同样建立的前瞻性成人和儿童队列中进行的这些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措施的目标，以改善这些患者的结局。在已建立的成人SLE队列中的研究已经实现了这一目标。我们希望Sule博士在K23奖期间建立的儿科队列在儿科SLE中同样有价值，并将成为未来儿科流变学独立临床研究的杰出工具。Lay语言：儿科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，我们需要定义疾病活动的预测因子，这样我们就可以干预治疗，最大限度地减少疾病损害。我们建议建立一个儿童SLE队列，这将导致更好地了解儿童狼疮，并使这些儿童的生活有所不同。

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.