CureGN

摘要 原发肾小球疾病，包括微小病变病(MCD)、局灶节段性肾小球硬化 FSGS、免疫球蛋白A肾病(IGAN)和膜性肾病(MN)占终末期肾病的10%。 并且在成人和儿童中都与显著的发病率和死亡率有关。 尽管与这些异质性疾病相关的疾病负担很高，但疾病的发病机制， 自然病史、临床结果的预测因素和治疗反应仍未完全确定。至 为了解决这些知识差距，CureGN(CureGN)联盟组建了一个大型 由60多个临床研究地点、肾病学家和具有不同专业知识的科学家组成的联盟受到影响 患者和倡导团体、生物制药行业、联邦资助机构和监管机构 经纪公司。CureGN正在进行一项前瞻性的纵向观察研究，成功地招募和 保留了近2400名患有IgAN、FSGS、 MN和MCD。在CureGN的第二研究阶段，我们建议保持和增强CureGN 财团基础设施及其辅助研究计划，以加速患者相关的肾小球疾病 研究。我们将继续我们对肾小球疾病的核心前瞻性、纵向观察研究。 患者，通过招募到替换策略招募额外的受试者，以保持2400名活跃队列 参与者，同时存储和管理高质量的临床数据和生物材料。这项基础性工作是 由一个完善的研究行政结构进行，并与 数据协调中心(Arbor Research Collaborative for Health和密歇根大学)和四个 参与的临床中心(由宾夕法尼亚大学、哥伦比亚大学、 北卡罗来纳州和中西部儿科联盟)。CureGN位于多个域的连接点，这些域 合作克服改善对肾小球疾病患者护理的障碍。它现在已经成熟了 科学工作组、出版委员会和辅助研究基础设施，通过使现代 多个相关知识领域的分析方法，通过管理一个研究机会池 与NephCure Kidney International合作，通过不断完善研究工具，通过加强 职业发展活动，并通过召集患者和公私合作来验证和 实施精准医治，CureGN创造的资源势必加速 改善对肾小球疾病患者的护理。

Abstract Primary glomerular diseases, including minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), immunoglobulin A nephropathy (IGAN), and membranous nephropathy (MN), account for 10% of end- stage kidney disease and are associated with significant morbidity and mortality in both adults and children. Despite the high disease burden associated with these heterogeneous disorders, disease pathogenesis, natural history, predictors of clinical outcomes and response to therapy remain incompletely defined. To address these knowledge gaps, the CureGlomeruloNephropathy (CureGN) consortium assembled a large consortium of more than 60 clinical study sites, nephrologists, scientists with diverse expertise, affected patients and advocacy groups, the biopharmaceutical industry, federal funding agencies, and regulatory agencies. CureGN is conducting a prospective, longitudinal observational study, successfully enrolling and retaining a race and ethnicity-balanced cohort of nearly 2400 adult and pediatric participants with IgAN, FSGS, MN and MCD. In CureGN’s second study phase, we propose to maintain and enhance the CureGN Consortium infrastructure and its ancillary study program to accelerate patient-relevant glomerular disease research. We will continue our core prospective, longitudinal observational study of glomerular disease patients, enrolling additional subjects by a recruit-to-replace strategy to maintain an active cohort of 2400 participants, while banking and curating high quality clinical data and biomaterials. This foundational work is being conducted by a refined study administrative structure and a well-functioning collaboration between the Data Coordinating Center (at Arbor Research Collaborative for Health and the University of Michigan) and four Participating Clinical Centers (managed at the University of Pennsylvania, Columbia University, University of North Carolina, and the Midwest Pediatric Consortium). CureGN sits at the nexus of multiple domains that are collaborating to overcome the barriers to improving care for glomerular disease patients. By its now mature scientific working groups, publications committee, and ancillary study infrastructure, by enabling modern analytical approaches in multiple relatable knowledge domains, by administering a research opportunity pool in conjunction with NephCure Kidney International, by continuing refinement of study instruments, by enhancing career development activities, and by convening patient and public-private collaborations to validate and implement precision medicine-based therapies, the resource created by CureGN is poised to accelerate improving care of glomerular disease patients.