Chronic Kidney Disease Precision Medicine Project

慢性肾脏病精准医疗项目

基本信息

批准号：
10223908
负责人：
ROBERT Daniel TOTO
金额：
$ 30万
依托单位：
UT SOUTHWESTERN MEDICAL CENTER
依托单位国家：
美国
项目类别：
财政年份：
2017
资助国家：
美国
起止时间：
2017-09-15 至 2023-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10223908
关键词：
Affect Atlases Biological Factors Biopsy Blood Caring Cessation of life Characteristics Chronic Kidney Failure Clinical Clinical Data Clinical Research Clinical Research Protocols Clinical Trials Cohort Studies Communities Consent Coupled Data Diabetes Mellitus Diagnosis Education Electronic Health Record End stage renal failure Enrollment Ensure Ethics Evaluation Functional Magnetic Resonance Imaging Funding Future Health Health Resources Healthcare Systems Hospitals Hypertension Image Individual Informatics Information Technology Informed Consent Infrastructure Intervention Kidney Kidney Failure Knowledge Longitudinal cohort study Medical Economics Methodology Modeling Multicenter Studies National Institute of Diabetes and Digestive and Kidney Diseases Outcome Outcomes Research Participant Patient Care Patient Recruitments Patient-Focused Outcomes Patients Phase Phenotype Population Precision Medicine Initiative Probability Process Process Assessment Prospective Studies Prospective cohort study Protocols documentation Public Health Research Research Personnel Resources Safety Sampling Site Socioeconomic Factors System Texas Time Tissues United States National Institutes of Health Urine Work base clinical phenotype clinical practice cohort collaboratory disorder subtype experience high risk improved informatics tool innovation kidney biopsy novel patient oriented patient population personalized care phenotypic data pragmatic trial precision medicine primary outcome programs radiologist recruit single photon emission computed tomography socioeconomics sound systems research therapeutic target tool translational medicine

项目摘要

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. We are currently conducting the largest pragmatic trial in CKD sponsored by the NIH Collaboratory for Health Care Systems (HCS) Research using novel electronic health records (EHR) - based informatics to identify and facilitate care for patients with CKD, diabetes and hypertension, including large numbers of underserved patients. We now propose a prospective cohort study (Precise-CKD) in patients with CKD as part of the Kidney Precision Medicine Program (KPMP). We hypothesize that our large and diverse HCS will successfully obtain kidney biopsies, biosamples and clinical data from patients with CKD and that with that approach we will identify disease subgroups and contribute to the creation of a kidney tissue atlas as part of the KPMP. In the Planning Phase UG 3 of this proposal we will use our existing, fully functioning, novel information technology tools leveraging our electronic health record systems to identify, recruit, enroll and obtain clinical data, biosamples and kidney biopsies from patients with CKD-Diabetes and CKD-Hypertension from 3 large HCS. We will engage community and patient stakeholders to develop ethically-sound, patient-centered processes to engage patients with CKD in the study and obtain consent for kidney biopsies. We will work closely with KPMP to develop common study protocols and following maximum safety precautions we will obtain high quality kidney biopsies from 40 patients. In the Implementation Phase UH3 we will increase recruitment and obtain kidney biopsies, biosamples and clinical data from an additional 400 patients with CKD-diabetes and CKD-HTN adding another large HCS. We will also expand the cohort of CKD patients likely to yield actionable results based on high risk for progression (eGFR decline >30% over 2 years), novel imaging findings (fMRI or SPECT from UTSW) or results from other KPMP investigators from UG3 suggesting probability of identifying therapeutic targets. The long-standing successful track record of our group in recruiting patients into clinical trials of CKD, the large, and diverse patient populations available for recruitment at our site, the fully functioning infrastructure for identifying and recruiting patients from multiple healthcare systems with novel informatics tools, and the ethically rigorous approach to consenting for kidney biopsy coupled with the rigorously safe methodology will facilitate our efforts to contribute to the KPMP.

目前对引发 CKD 的病理生理学和社会经济过程的理解人们对他们进展为 ESRD 的途径知之甚少，导致治疗方法有限。有一个使用组合药物准确对 CKD 患者进行表型分型的前瞻性研究的迫切需求尚未得到满足肾活检、临床、生物学和社会经济因素，以制定个性化护理以改善患者的状况结果。我们目前正在进行由 NIH 合作实验室赞助的最大规模的 CKD 实用试验使用基于新型电子健康记录 (EHR) 的信息学进行医疗保健系统 (HCS) 研究识别并促进对 CKD、糖尿病和高血压患者的护理，包括大量服务不足的患者。我们现在提出一项针对 CKD 患者的前瞻性队列研究 (Precise-CKD)，作为其中的一部分肾脏精准医学计划 (KPMP) 的成员。我们假设我们庞大且多样化的 HCS 将成功获得 CKD 患者及其相关患者的肾活检、生物样本和临床数据通过这种方法，我们将确定疾病亚组，并为创建肾脏组织图谱做出贡献，作为该研究的一部分毕马威会计师事务所。在此提案的规划阶段 UG 3 中，我们将使用现有的、功能齐全的新颖信息利用我们的电子健康记录系统来识别、招募、登记和获取临床信息的技术工具来自 3 大 CKD 糖尿病和 CKD 高血压患者的数据、生物样本和肾活检 HCS。我们将让社区和患者利益相关者参与进来，共同制定符合道德规范、以患者为中心的让 CKD 患者参与研究并获得肾活检同意的流程。我们将密切合作与 KPMP 一起制定共同的研究方案并遵循最大程度的安全预防措施，我们将获得高来自 40 名患者的高质量肾活检。在 UH3 实施阶段，我们将增加招募并获取肾脏活检、生物样本以及另外 400 名 CKD-糖尿病和 CKD-HTN 患者的临床数据，添加了另一个大型 HCS。我们还将扩大 CKD 患者队列，可能会根据进展的高风险产生可行的结果（2 年内 eGFR 下降 >30%）、新的影像学发现（来自 UTSW 的 fMRI 或 SPECT）或其他结果 UG3 的 KPMP 研究人员提出了确定治疗靶点的可能性。我们团队在招募患者参加 CKD 临床试验方面有着长期的成功记录，我们的站点可供招募的大量且多样化的患者群体、功能齐全的基础设施使用新颖的信息学工具从多个医疗保健系统中识别和招募患者，以及同意肾活检的伦理严格方法加上严格安全的方法将促进我们为 KPMP 做出贡献的努力。