KANSAS POLYCYSTIC KIDNEY IMAGING PROGRAM

多数常染色体显性多囊肾病（ADPKD 1型和2型）和常染色体隐性多囊肾病（ARPKD）患者发展为终末期肾衰竭。初步研究表明，囊肾的体积与其GFR反映的功能呈负相关。然而，目前还没有办法在病程早期确定PKD患者是否注定会在正常预期寿命内经历肾功能衰竭。为达致《外地流动资金条例》的目标，我们会成立一个工作小组，评估以下具体目标：目标1。收集具有明确特征的PKD （ADPKD）的个体队列，这些个体具有相对较高的进展为终末期肾脏疾病（ESRD）的风险。ADPKD受试者将从该地区选择有一个或多个进展危险因素的受试者，包括：ESRD家族史、男性、多胎妊娠、高血压、蛋白尿和血尿。ARPKD受试者将主要从没有透析的患者中选择。目标2。比较和评价快速采集MR，超声和CT在典型PKD中获得的肾脏图像的准确性和再现性。这些研究将在囊肾幻影模型中进行，以确定每种方法与其他方法相比的最佳精度和准确性，并在轻度到重度肾囊变患者中进行。PKD科目将按照下一个目标的概述顺序进行研究。目标3。评价PKD患者肾脏形态计量学与进行性疾病的其他替代标志物的顺序变化。受试者将在预定的时间间隔内进行成像，以确定：a)肾脏总体积、囊肿总体积和实质总体积的变化率；b)“功能性”实质体积变化率；c)单个囊肿体积变化率；d)肾内囊肿分布与进展因素的关系；e)与肾脏增大和实质体积相关的疾病进展替代标志物的表达变化；F)囊肿生长与囊内积液假说的关系。这些研究的成功完成将确定临床上可靠的方法，在GFR发生可测量的变化和肾超结构发生不可逆转的变化之前确定PKD的进展速度。

The majority of patients with autosomal dominant polycystic kidney disease (ADPKD types 1 and 2) and autosomal recessive polycystic kidney disease (ARPKD) develop end-stage renal failure. Preliminary studies indicate an inverse correlation between the volume of cystic kidneys and their function reflected in the GFR. However, there is presently no way to determine early in the course if an individual with PKD is destined to experience renal failure in a normal life expectancy. To meet the objectives of the RFA we will develop a PCC to evaluate the following specific aims: Aim 1. Assemble a cohort of individuals with well-characterized PKD (ADPKD) who are at relatively high risk to experience progression to end-stage-renal disease (ESRD). ADPKD subjects will be selected from the region who have one or more risk factors for progression, including: family history of ESRD, male gender, multiple pregnancies, hypertension, proteinuria and hematuria. ARPKD subjects will be selected primarily from those who have survived without dialysis beyond the first year of life. Aim 2. Compare and evaluate the accuracy and reproducibility of renal images obtained by rapid acquisition MR, ultrasound and CT in representative examples of PKD. These studies will be done in phantom models of cystic kidney in order to determine optimum precision and accuracy of each method in comparison to the other, and in selected patients with renal cystic changes ranging from mild to severe. PKD subjects will then be studied serially as outlined in the next aim. Aim 3. Evaluate sequential changes in renal morphometrics in subjects with PKD in relation to other surrogate markers for progressive disease. Subjects will be imaged at predefined intervals in order to determine: a) The rate of change in total kidney volume, total cyst volume and total parenchymal volume; b) The rate of change of "functional" parenchymal volume; c) The rate of change of individual cyst volume; d) The intra- renal distribution of cysts in relation to progression factors; e) Changes in the expression of surrogate markers of disease progression in relation to renal enlargement and parenchyma volume, and ; f) the relation of cyst growth to hypotheses of intra-cyst fluid accumulation. Successful completion of these studies will identify clinically reliable methods for determining the rate of progression of PKD before there are measurable changes in GFR and irreversible changes in renal ultra- structure.