GDPrime

描述（由申请人提供）：该数据协调中心（DCC）申请是根据RFA-DK-12-014提交的，旨在建立一个原发性肾小球疾病（GDPrime）联盟，重点关注微小变化疾病（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）。综上所述，这些疾病造成了严重的症状负担、慢性健康挑战和1至20年的肾功能进行性丧失，每年在美国导致10%的终末期肾病（ESRD）患者。尽管原发性肾小球疾病带来了健康负担，但在了解疾病病理生理、诊断、监测、预后和临床试验方面存在重大差距，并阻碍了对这些疾病的有效治疗的实施。GDPrime联盟将建立一个全面的研究平台，围绕2400例病例和2400例对照的前瞻性、观察性队列研究。DCC申请的首要目标是建立数据协调中心，为联盟和相关辅助研究提供研究设计、数据管理和分析专业知识。GDPrime DCC将建立进行GDPrime研究的基础设施；协调原发性肾小球疾病儿童和成人与健康对照者的前瞻性纵向队列研究；确定病程并调查与非疾病对照者比较的患者预后预测因素及其与疾病进展的关系。GDPrime DCC和整个联合体将建立一个全面的辅助研究项目，用于校外合作和伙伴关系，以推进新的生物标志物、治疗靶点、结果测量和科学界提出的尚未定义的研究的鉴定和评估。

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.