Central Hub for Kidney Precision Medicine - Administrative Core

摘要 现代生物医学研究的一个主要挑战是相对无法将 提高科学发现转化为重大治疗突破的速度。这一缺点有 在肾脏疾病中尤其引人注目，在那里随机临床试验的数量 在肾脏病学上发表的论文比所有其他内科专科都要少。慢性肾脏 疾病(CKD)是一个公共健康问题，在美国有2000多万人受到影响，几乎没有药物 除了肾素-血管紧张素系统的抑制剂外，其他药物已被证明可以减缓CKD的进展， 降低死亡率，或提高生活质量。急性肾损伤(AKI)影响高达10%的 在美国住院的患者，并与死亡率、住院时间延长、 以及CKD的后续发展或进展。目前还没有公认的治疗方法 预防或改善AKI病程的方法迫切需要帮助 定义CKD和AKI疾病亚组并确定关键细胞、途径和靶点 治疗。我们建议建立对CKD和AKI的多尺度理解，通过整合 肾脏疾病进展的细胞和器官水平特征的临床数据包括 疾病的基因组和表观基因组驱动因素的询问。我们计划建造肾脏精密 在强大的现有基础设施上，由经验丰富的 多学科团队，具有良好的成功记录。KPMP的总体目标 管理核心是通过促进成功的跨学科团队科学 KPMP指导委员会(SC)之间的沟通、协作和协调， 中央枢纽的组成部分、招募地点、组织审问地点、EEP、OSMB、IRB 也是赞助商。这个团队科学的总体目标是促进科学的严谨性和参与性。 以安全促肾精准用药。作为该模型的关键组件， 行政核心将主要负责协调KPMP的运作， 建立必要的工作组和关系，组建和操作病人 参与工作组，并管理机会池，以形成新的合作伙伴关系。这个 管理核心还将提供项目管理、预算和财务 管理中心的所有活动。行政核心将负责总体 中心日常运作、研究、研究培训和教育，以及社区外展 活动，包括信息传播

ABSTRACT A major challenge for modern biomedical research has been the relative inability to translate the increasing rate of scientific discovery into major therapeutic breakthroughs. This shortcoming has been particularly dramatic in kidney diseases, where the number of randomized clinical trials published in nephrology is fewer than all other specialties of internal medicine. Chronic kidney disease (CKD) is a public health problem affecting more than 20 million people in the US, few drugs other than inhibitors of the renin-angiotensin system have been proven to slow CKD progression, lower mortality rates, or improve quality of life. Acute kidney injury (AKI) affects up to 10% of all hospitalized patients in the US, and is associated with mortality, prolonged hospital length of stay, and subsequent development or progression of CKD. Currently there is no accepted therapeutic approach to either prevention or amelioration of the course of AKI There is a critical need to help define CKD and AKI disease subgroups and identify critical cells, pathways and targets for novel therapies. We propose to establish a multiscale understanding of CKD and AKI, by integrating clinical data with the cell and organ level features of kidney disease progression including interrogation of genomic and epigenomic drivers of disease. We plan to build the Kidney Precision Medicine Project Central Hub on strong existing infrastructure led by a highly experienced, multidisciplinary team with a strong track record of success. The overarching objective of the KPMP Administrative Core is to facilitate successful interdisciplinary team science by fostering communication, collaboration, and coordination among the KPMP Steering Committee (SC), components of the Central Hub, Recruitment Sites, Tissue Interrogation Sites, EEP, OSMB, IRB and sponsor. The overall goal of this team science is to promote scientific rigor and participant safety in order to advance kidney precision medicine. As a key component of this model, the Administrative Core will have primary responsibility for coordinating the operations of the KPMP, establishing necessary working groups and relationships, forming and operating a Patient Engagement Working Group, and administering an Opportunity Pool to form new partnerships. The Administrative Core will additionally provide project management, budgeting, and fiscal management for all Central Hub activities. The Administrative Core will be responsible for overall day-to-day center operations, research, research training and education, and community outreach activities, including information dissemination