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(FSGS)占儿童特发性肾病综合征的10-20%，占成人肾病综合征的35%。在大多数情况下，FSGS对治疗干预无效，经常导致慢性肾功能衰竭。尽管治疗FSGS的治疗干预措施已被广泛报道，但由于缺乏对照研究和研究的患者数量较少，尚未制定循证治疗指南。因此，这是第一个多中心、前瞻性、随机化的临床试验，预计将有足够大的研究人群来提供统计上有效的结果。本研究的目的是比较环孢素(CsA)、霉酚酸酯(MMF)和地塞米松(DEX)诱导激素耐药FSGS患者蛋白尿缓解的效果。研究对象为2-35岁，经活检证实的原发FSGS患者，且使用标准大剂量类固醇治疗失败。受试者将在泼尼松6个月和赖诺普利或氯沙坦18个月的基础上接受为期1年的随机治疗(CsA或MMF+DEX)。

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 (FSGS) causes ~10-20% of all cases of idiopathic nephrotic syndrome in children and ~35% of all cases in adults. In the majority of cases, FSGS is refractory to therapeutic interventions and often results in chronic renal failure. Although therapeutic interventions for the treatment of FSGS have been widely reported, evidence-based treatment guidelines have not been developed due to the lack of controlled studies and the small number of patients studied. Therefore, this is the first multi-center, prospective, randomized clinical trial which is expected to have a sufficiently large study population to provide statistically valid outcomes. The purpose of this research is to compare the effectiveness of Cyclosporine (CSA) vs. Mycophenolate Mofetil (MMF) and dexamethasone (DEX) to induce remission of proteinuria in steroid resistant FSGS. Subjects 2-35 years of age who have biopsy-proven primary FSGS and have failed treatment with standard high-dose steroids will be enrolled. Subjects will receive the randomized treatment (CSA or MMF + DEX) for 1 year in addition to prednisone for 6 months and lisinopril or losartan for 18 months.