CRISP III - Kansas Polycystic Kidney Imaging Program Supplemental Request

常染色体显性遗传性多囊肾病（ADPKD）是发病的主要原因，也是第四大病因。 这是世界上最严重的终末期肾病，影响了50多万美国公民。大学的研究人员 安娜，埃默里大学，堪萨斯大学，马约诊所和华盛顿大学联合起来， 2000年创建了多囊肾病放射学研究联盟（CRISPI），2006年 包括匹兹堡大学代替华盛顿大学参加CRISP II。主要目标 CRISPI和II的目的是：建立准确，可靠和可重复的磁共振基础 测量肾脏总体积（TKV）、肝囊肿体积（LCV）、肾血流量（RBF）和 囊肿生长和扩张。基于对200名CRISP l/ll参与者进行的7.3年纵向随访，我们 现在可以：1）确定TKV和定性（患者报告的结局）之间的明确关系 和定量（肾功能不全）终点;以及2）确定潜在的可改变的风险因素 与TKV和LCV联合进行干预研究。TKV最终可用作替代标记物 临床试验中的疾病进展。CRISPIN的目标扩展了CRISPI/II的观察。的 CRISP III的总体目标是开发和增强预测模型， ADPKD的不足。具体而言，目标1：扩展TKV和LCV的系列定量， 测试预测肾功能不全风险的新模型。目标2：确定 经年龄和性别调整的RBF测量值可预测TKV的变化率，并确定RBF和 TKV可独立预测发生肾功能不全的风险。目标3：制定方法， 基线时肾囊肿数量、体积和地形对TKV后续过程的影响， GFR和肾功能不全的风险。目的4：扩展和分析CRISP生物样品 收集在NIDDK储存库中，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修改固定饮食中的可行性和有效性 CRISP中观察到的钠摄入模式，并确定该变化是否改变TKV变化率。 相关性（参见说明）： 这些研究的结果将影响ADPKD患者的生活。开发预测标记物 疾病严重程度早，肾功能不全之前的损失将导致预期寿命的增加和改善 ADPKD患者的生活质量。

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 Alabanna, 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 ncluded 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 nsufficiency 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. RELEVANCE (See instructions): The results of these studies will impact the lives of patients with ADPKD. Developing predictive markers of disease severity early, prior to loss of renal insufficiency will result in increased life expectancy and improved quality of life in patients with ADPKD.