Kidney Precision Medicine Project - Patient Engagement

项目摘要 目前对引发CKD的病理生理学和社会经济学过程的理解， 他们进展为ESRD的途径知之甚少，导致治疗有限。有一个 迫切需要进行前瞻性研究，以使用联合治疗对CKD患者进行准确的表型分析， 肾活检，临床，生物学和社会经济因素，以发展个性化护理，以改善患者 成果。在过去的四年里，我们在地方一级组建了一支高技能的跨学科团队， 临床研究人员、医生-科学家、临床医生、研究护士、患者倡导者和社区 咨询委员会。这个团队设计、实施并维持了一个非常成功、道德上健全和安全的 肾脏精准医学项目的招募网站（RS）：1）我们执行了第一个KPMP联盟 2019年9月进行了肾活检，是联盟中第一个进行30次肾活检的RS， 成功入组临床诊断为高血压或糖尿病慢性肾病的患者， 产生足够的组织用于研究方案以及病理诊断; 2）我们收集并连接 活检参与者的临床、生物学和社会经济学数据; 3）我们将努力集中在非洲- 美国和拉丁裔人群的比例非常高，以解决CKD的不成比例负担， 以及历史上缺乏将这些人群纳入临床研究，由NIH授权， 在这些组中，11例活检中有22例（73%）。在联盟层面，我们的团队成员已经做出了 对KPMP的主要组织和业务方面作出有意义的贡献。 本提案的主要目标是人群识别、患者招募和获得临床 表型数据、生物样品和肾活检。为了实现这一目标，我们将1）识别，招募，注册， 每年对来自3个大型HS的34名患者进行活检，服务超过51，996名CKD和糖尿病患者和/或 2）实施伦理健全、系统质量保证 从CKD患者中获得临床和人口统计学数据、生物样本和肾活检的过程， 纵向临床研究;和3）制定和实施以患者为中心的流程，以促进长期 参与和保留KPMP的研究参与者。我们作为招聘网站的成功记录， 从患有糖尿病或高血压的CKD患者的肾脏组织中获取， 患者人群使我们成为KPMP继续的理想场所。我们招募患者的能力 多个卫生系统与新的信息学工具，一个伦理上严格的方法，同意肾脏 活检，加上严格和安全的方法是一个强大的组合，我们带来了KPMP。我们 创新的方法，这FOA不仅将提供数据，以产生新的知识，以改善这两个 CKD的诊断和治疗，而且还可以作为未来肾脏精准医学计划的模型， 随着时间的推移而扩张。

Project Summary Abstract The current understanding of the pathophysiologic and socio-economic processes that initiate CKD and the paths by which they progress to ESRD are poorly understood, resulting in limited treatments. There is a compelling unmet need for prospective studies to accurately phenotype patients with CKD using a combination of kidney biopsy, clinical, biologic, and socioeconomic factors to develop individualized care to improve patient outcomes. Over the past four years, at the local level we assembled a highly skilled, interdisciplinary team of clinical researchers, physician-scientists, clinicians, research nurses, patient advocates and a Community Advisory Board. This team designed, implemented and sustained a highly successful, ethically sound and safe recruitment site (RS) for the Kidney Precision Medicine Project: 1) We performed the very first KPMP consortium kidney biopsy in September 2019 and were the first RS in the consortium to perform 30 kidney biopsies, successfully enrolling patients with a clinical diagnosis of hypertensive or diabetic chronic kidney disease and yielding sufficient tissue for research protocols as well as for pathological diagnosis; 2) We collected and linked clinical, biological and socioeconomic data of the biopsy participants; 3) We focused our efforts on African- American and Latinx populations at a very high rate in order to address the disproportionate burden of CKD as well as the historical lack of inclusion of these populations in cljnical research, mandated by the NIH, completing 22 out of 11 (73%) of biopsies among these groups. At the consortium level our team members have made meaningful contributions to the major organizational and operational aspects of KPMP. The main goal in this proposal will be population identification, patient recruitment and obtaining clinical phenotype data, biosamples and kidney biopsies. To accomplish this goal we will 1) Identify, recruit, enroll, and biopsy 34 patients annually from 3 large HS serving more than 51,996 patients with CKD and diabetes and/ or hypertension and follow patients longitudinally; 2) Implement ethically-sound, systematic quality-assured processes to obtain clinical and demographic data, bio-samples and kidney biopsies from patients with CKD in a longitudinal clinical study; and 3) Develop and implement patient-centered processes to promote long-term engagement and retention of study participants in KPMP. Our successful track record as a recruitment site for procurement of kidney tissue from patients with CKD attributed to diabetes or hypertension from a large, diverse patient population makes us an ideal site for the continuation of the KPMP. Our ability to recruit patients from multiple health systems with novel informatics tools, an ethically rigorous approach to consenting for kidney biopsy and coupled with a rigorous and safe methodology is a powerful combination we bring to the KPMP. Our innovative approach to this FOA will not only provide the data to generate new knowledge to improve both the diagnosis and treatment of CKD, but also serve as a model for future kidney precision medicine initiatives and their expansion over time.