Kansas Polycystic Kidney Imaging Program

描述（由申请人提供）：常染色体显性遗传性多囊肾病（ADPKD）是一种主要的发病原因，也是世界上ESRD的第四大病因，影响超过50万美国公民。亚拉巴马大学、埃默里大学、堪萨斯大学、马约诊所和华盛顿大学的研究人员于2000年联合起来创建了多囊肾病放射学研究联盟（CRISPI），并于2006年将匹兹堡大学纳入CRISP II。CRISPI和II的主要目的是：建立准确、可靠和可重复的基于磁共振的肾脏总体积（TKV）、肝囊肿体积（LCV）、肾血流量（RBF）以及囊肿生长和扩张模式的测量。基于对200例CRISP I/II受试者进行的7.3年纵向随访，我们现在可以：1）确定TKV与定性（患者报告的结局）和定量（肾功能不全）终点之间的明确关系;以及2）确定与干预研究中的TKV和LCV相关的潜在可改变风险因素。TKV最终可用作临床试验中疾病进展的替代标志物。CRISPIN的目标扩展了CRISPI/II的观察。CRISP III的总体目标是开发和增强最能预测ADPKD肾功能不全的预测模型。具体而言，目标1：扩展TKV和LCV的系列定量，以开发和测试预测肾功能不全风险的新模型。目标二：确定年龄和性别校正的RBF测量值预测TKV变化率的程度，并确定RBF和TKV是否独立预测发生肾功能不全的风险。目标3：制定方法，量化基线时肾囊肿数量、体积和地形对TKV和GFR后续病程以及肾功能不全风险的影响。目标4：扩展和分析NIDDK储存库中收集的CRISP生物样本，以改善基因型/表型和生物标志物研究，并促进辅助研究。目标5：测试强化饮食咨询在改变CRISP中观察到的钠摄入固定模式方面的可行性和有效性，并确定这种变化是否会改变TKV变化率。

DESCRIPTION (provided by applicant): Autosomal dominant polycystic kidney disease (ADPKD) is a major cause of morbidity and the fourth leading cause of ESRD in the world, affecting more than 500,000 U.S. citizens. Researchers at the University of Alabama, Emory University, University of Kansas, Mayo Clinic and Washington University joined together in 2000 to create the Consortium for Radiologic Studies of Polycystic Kidney Disease (CRISPI) and in 2006 included the University of Pittsburgh in place of Washington University for CRISP II. The primary objectives of CRISPI and II were to: establish accurate, reliable and reproducible magnetic resonance based measurements of total kidney volume (TKV), liver cyst volume (LCV), renal blood flow (RBF), and patterns of cyst growth and expansion. Based on 7.3 years of longitudinal followup in 200 CRISP l/ll participants, we can now: 1) establish an unequivocal relationship between TKV and qualitative (patient reported outcomes) and quantitative (renal insufficiency) end-points; as well as 2) identify potential modifiable risk factors associating with TKV and LCV for intervention studies. TKV ultimately may be used as a surrogate marker of disease progression in clinical trials. The goals of CRISPIN extend the observations of CRlSPI/ll. The overarching Aim for CRISP III is to develop and enhance prediction models that best predict renal insufficiency in ADPKD. Specifically, Aim 1: Extend the serial quantification of TKV and LCV to develop and test new models for predicting the risk of developing renal insufficiency. Aim 2: Determine the extent to which age and sex-adjusted measurements of RBF predict the rate of change in TKV and determine if RBF and TKV independently predict the risk of developing renal insufficiency. Aim 3: Develop methods to quantify the influence of renal cyst number, volume, and topography at baseline on the subsequent course of TKV and GFR and the risk of developing renal insufficiency. Aim 4: Expand and analyze CRISP biological samples collected in NIDDK repositories to improve genotype/phenotype and biomarker studies, and facilitate ancillary studies. Aim 5: Test the feasibility and efficacy of intensive dietary counseling in modifying the fixed pattern of sodium intake observed in CRISP and determine if this change alters the rate of TKV change.