FOCAL SEGMENTAL GLOMERULOSCLEROSIS IN YOUNG PATIENTS

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。局灶节段性肾小球硬化（FSGS）导致约10-20%的儿童特发性肾病综合征病例和约35%的成人病例。在大多数情况下，FSGS治疗干预是难治性的，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、随机临床试验，预计将有足够大的研究人群来提供统计有效的结果。本研究的目的是比较环孢素（CSA）与霉酚酸酯（MMF）和地塞米松（DEX）对类固醇耐药FSGS患者蛋白尿缓解的效果。受试者年龄2-35岁，活检证实为原发性FSGS，且使用标准大剂量类固醇治疗失败。受试者将接受1年的随机治疗（CSA或MMF + DEX），外加6个月的强的松治疗和18个月的赖诺普利或氯沙坦治疗。

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.