FOCAL SEGMENTAL GLOMERULOSCLEROSIS IN YOUNG PATIENTS

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 局灶性节段性肾小球硬化约占所有特发性肾病综合征病例的10%至20%，这是一种肾脏内部结构的炎症，在儿童和成人中分别占35%。在FSGS中，蛋白质在尿液中持续释放，特别是以尿白蛋白的形式释放，尿白蛋白是一种大的蛋白质分子。大多数FSGS患者将进展为长期、慢性肾功能衰竭，这是渐进的，并导致肾脏排泄废物、浓缩尿液和保存电解质的能力逐渐丧失。在大多数患者中，FSGS对治疗干预是无效的。FSGS患者的发病率和死亡率因肾移植后这种病变相对较高的复发率而变得更加复杂。复发的FSGS患者的预后即使不比原发FSGS患者差，也是一样差的。在过去的二十年里，FSGS在城市和农村人口中的发病率都有所增加。尽管这种疾病似乎与免疫系统有关，但对皮质类固醇或免疫抑制药物的反应并不一致，这些药物会改变我们的免疫反应。这项研究由国家卫生研究所赞助，旨在制定循证治疗指南，以干预FSGS及其进展为慢性肾功能衰竭。这是一项多中心随机试验，将在全美500名儿童和年轻人中进行。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Focal segmental glomerulosclerosis causes about 10 to 20% of all cases of idiopathic nephrotic syndrome, which is an inflammation of the internal structures of the kidney, in children and 35% in adults. In FSGS, protein is persistently released in the urine, especially in the form of urine albumin, a large protein molecule. Most patients with FSGS will progress to long term, chronic renal failure which is gradual and results in a progressive loss of the ability of the kidneys to excrete wastes, concentrate urine, and conserve electrolytes. In the majority of patients, FSGS is refractory to therapeutic interventions. The morbidity and mortality for patients with FSGS is compounded by the relatively high recurrence rate for this lesion following renal transplantation. The outcomes for patients with recurrent FSGS are as poor if not worse than those for patients with primary FSGS. Over the past two decades there has been an increased incidence of FSGS in both urban and rural populations. Although the disorder seems to be immune system related, response to corticosteroid or immunosuppressive medications, which modify our immune response, is inconsistent. This research study is being sponsored by the National Institute of Health to develop evidence based treatment guidelines to intervene with FSGS and its progression to chronic renal failure. It is a multi-center randomized trial that will be conducted throughout the United States in 500 children and young adults.