CureGN

摘要 原发性肾小球疾病，包括微小病变（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%。 阶段性肾病，并与成人和儿童的显著发病率和死亡率相关。 尽管与这些异质性疾病相关的高疾病负担， 自然病史、临床结果的预测因素和对治疗的反应仍然不完全确定。到 为了解决这些知识缺口，CureGlomeruloNephropathy（CureGN）联盟组织了一个大型的 由60多家临床研究中心、肾病学家、具有不同专业知识的科学家组成的联盟， 患者和倡导团体、生物制药行业、联邦资助机构和监管机构 社. CureGN正在进行一项前瞻性、纵向观察性研究，成功招募并 保留了一个种族和民族平衡的队列，其中包括近2400名患有IgAN，FSGS， MN和MCD。在CureGN的第二个研究阶段，我们建议维持和增强CureGN 联盟基础设施及其辅助研究计划，以加速患者相关肾小球疾病 research.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，通过招募替换策略招募额外受试者，以维持2400例的活跃队列 参与者，同时银行和策划高质量的临床数据和生物材料。这项基础性工作是 正在通过完善的研究行政结构和 数据协调中心（Arbor Research Collaborative for Health和密歇根大学）和四个 参与临床中心（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位于多个域的连接处， 合作克服改善肾小球疾病患者护理的障碍。到目前为止， 科学工作组、出版物委员会和辅助研究基础设施， 在多个相关知识领域的分析方法，通过管理研究机会池， 与NephCure Kidney International合作，继续完善研究工具， 职业发展活动，并通过召集病人和公私合作，以验证和 实施基于精确医学的疗法，CureGN创造的资源有望加速 改善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.