CRISP III - Kansas Polycystic Kidney Imaging Program Supplemental Request

常染色体显性遗传性多囊肾病(ADPKD)是发病率的主要原因，也是第四大致病原因 ESRD是世界上最严重的疾病之一，影响着50多万美国公民。密歇根大学的研究人员 阿拉巴纳大学、埃默里大学、堪萨斯大学、梅奥诊所和华盛顿大学共同参加了 2000年创建多囊肾病放射学研究联盟(Crispi)，并于2006年 将匹兹堡大学取代华盛顿大学作为CRISP II的主要目标 Crispi和II的目标是：建立准确、可靠和可重复性的磁共振基础 测量总肾体积(TKV)、肝囊肿体积(LCV)、肾血流量(RBF)及 包囊的生长和扩张。基于对200名健康的L/ll参与者7.3年的纵向随访，我们 现在可以：1)在TKV和定性(患者报告的结果)之间建立明确的关系 和量化(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最终可能被用作替代标记 临床试验中的疾病进展情况。Crispin的目标扩展了CRlSPI/11的观测范围。这个 CRISP III的首要目标是开发和增强最好地预测肾脏的预测模型 ADPKD不足。具体而言，目标1：扩大TKV和LCV的系列量化，以开发和 测试预测发生肾功能不全风险的新模型。目标2：确定以下方面的程度 经年龄和性别调整的RBF测量可预测TKV的变化率，并确定RBF和 TKV独立预测发生肾功能不全的风险。目标3：开发方法来量化 基线时肾囊肿数目、体积和地形图对TKV和TKV后续病程的影响 肾小球滤过率和发生肾功能不全的风险。目标4：扩展和分析脆的生物样本 收集在NIDDK存储库中，以改进基因/表型和生物标记物研究，并促进 辅助研究。目的5.检验强化饮食咨询在改变固定饮食习惯方面的可行性和有效性 观察脆片中钠的摄入模式，并确定这一变化是否会改变TKV的变化率。 相关性(请参阅说明)： 这些研究的结果将影响ADPKD患者的生活。发展预测标记物 病情严重程度越早，肾功能不全消失之前就会导致预期寿命的延长和改善 ADPKD患者的生活质量研究

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.