DATA COORDINATING AND IMAGING ANALYSIS CENTER (DCIAC)

多囊肾病（PKD）是一种常见的遗传性疾病，在终末期肾衰竭的主要原因中排名第四。本研究的主要目的是利用容积成像方法来识别和量化与PKD分期和PKD进展率相关的形态学指标。将对200名受试者进行为期4年的随访，分为3个队列（GFR=30-50，70- 90 ml/min）。两个参与的临床中心（PCC）将各自提供约100例受试者的数据。每个PCC将收集临床数据和标本，进行肾功能检查，并在特定的随访间隔获得每例受试者的MR和US图像。数据将交付或以电子方式传输至本申请中拟定的数据协调和成像分析中心（DCIAC）。我们的具体目标是：（1）验证磁共振（MR）容积成像方法对PKD患者肾实质和肾/肝/胰腺囊肿的准确和可靠的形态学评估;（二）比较从PKD患者的MR成像数据中获得的形态学测量值与肾功能测量值，以确定哪些形态学测量值与肾功能损害的程度和发生率相关。疾病进展;和结构变化，同时考虑临床有效性、成本效益和患者可接受性。将开发适用于异质性人群生长曲线估计的统计方法，并将其应用于表征每个队列和整个样本的PKD进展。该项目的长期目标是开发有助于缩短确定PKD患者治疗干预有效性所需的观察期的方法。

Polycystic kidney disease (PKD) is a common hereditary disease and ranks fourth as a leading cause of end-stage renal failure. The primary goal of this study is to exploit volumetric imaging methods to identify and quantify morphologic measures that are associated with the PKD stage and the rate of PKD progression. 200 subjects, divided into three cohorts (GFR=30-50, 70- 90 ml/min) will be followed for a four-year period. Two participating clinical centers (PCC) will each contribute data on approximately 100 subjects. Each PCC will collect clinical data and specimens, perform renal functional tests, and obtain MR and US images for each subject at specific follow-up intervals. The data will be delivered or electronically transferred to the data coordinating and imaging analysis center (DCIAC) proposed in this application. Our specific aims are (1) to validate magnetic resonance (MR) volumetric imaging methods for accurate and reliable morphometric assessment of the renal parenchyma and renal/hepatic/pancreatic cysts in PKD patients; (2) to compare morphologic measures obtained from MR imaging data with renal functional measures in PKD patients to determine which morphologic measures are associated with the extent of renal functional impairment and with the rate of disease progression; and structural changes, while considering clinical-effectiveness, cost- effectiveness and patient acceptability. Statistical methods appropriate for growth curve estimation with a heterogeneous population will be developed and applied to characterize the PKD progression for each cohort and for the sample as a whole. The long-term goal of this project is to develop methods that would facilitate shortening the observation period necessary to determine efficacy of treatment interventions in PKD patients.