GDPrime

描述(由申请人提供)：本数据协调中心(DCC)申请是为了响应RFA-DK-12-014，建立一个以微小病变病(MCD)、局灶节段性肾小球硬化(FSGS)、免疫球蛋白A肾病(IGAN)和特发性膜性肾病(IMN)为重点的原发性肾小球疾病(GDPrime)联盟。总而言之，这些疾病导致严重的症状负担、慢性健康挑战以及1至20年来肾功能的渐进性丧失，导致美国每年10%的终末期肾病(ESRD)患者发病。尽管原发肾小球疾病造成了健康负担，但在疾病病理生理学、诊断、监测、预后和临床试验方面仍存在重大差距，并阻碍了对这些疾病的有效治疗。GDPrime财团将建立一个全面的研究平台，以2400个病例和2400个对照的前瞻性观察性队列研究为中心。DCC应用程序的总体目标是建立数据协调中心，为联合体和相关的辅助研究提供研究设计、数据管理和分析专业知识。GDPrime DCC将为开展GDPrime研究建立基础设施；协调对患有原发肾小球疾病和健康对照的儿童和成人进行的前瞻性纵向队列研究；确定病程，调查与非疾病对照相比患者结局的预测因素及其与疾病进展的关系。GDPrime DCC和整个联盟将为外部合作和伙伴关系建立一个全面的辅助研究计划，以促进对新的生物标记物、治疗目标、结果衡量标准以及科学界提出的尚未确定的研究的识别和评估。

DESCRIPTION (provided by applicant): This Data Coordinating Center (DCC) application is submitted in response to RFA-DK-12-014 to establish a primary glomerular disease (GDPrime) consortium with a focus on minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), immunoglobulin A nephropathy (IGAN), and idiopathic membranous nephropathy (IMN). Taken together these diseases are responsible for significant symptom burden, chronic health challenges, and progressive loss of kidney function over 1 to 20 years contributing to 10% of incident end stage kidney disease (ESRD) patients per year in the USA. Despite the health burden imposed by primary glomerular disease, significant gaps in understanding disease pathophysiology, diagnosis, monitoring, prognosis and clinical trials exists and have prevented the implementation of effective therapy for these conditions. The GDPrime consortium will establish a comprehensive research platform centered around a prospective, observational cohort study of 2400 cases and 2400 controls. The overarching goal of the DCC application is to establish the data coordinating center to provide the study design, data management and analysis expertise for the consortium and associated ancillary studies. The GDPrime DCC will establish the infrastructure for conduct of the GDPrime study; coordinate the prospective longitudinal cohort study of children and adults with primary glomerular disease and healthy controls; define disease course and investigate predictors of outcomes in patients compared with non-disease controls and their associations with disease progression. The GDPrime DCC and overall consortium will establish a comprehensive ancillary study program for extramural collaboration and partnership to advance the identification and evaluation of novel biomarkers, therapeutic targets, outcome measures and as yet undefined research proposed by the scientific community at large.