FOCAL SEGMENTAL GLOMERULOSCLEROSIS IN YOUNG PATIENTS

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 局灶节段性肾小球硬化症（FSGS）约占儿童特发性肾病综合征所有病例的10-20%，约占成人所有病例的35%。 在大多数情况下，FSGS是难治性的治疗干预，往往导致慢性肾衰竭。 虽然治疗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，这是第一项多中心、前瞻性、随机化临床试验，预计将有足够大的研究人群提供统计学有效的结局。 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）诱导激素抵抗的FSGS蛋白尿缓解的有效性。 将入组2-35岁的受试者，这些受试者患有活检证实的原发性FSGS，并且标准高剂量类固醇治疗失败。 受试者将接受随机化治疗（CSA或MMF + DEX）1年，此外还将接受泼尼松6个月和赖诺普利或氯沙坦18个月。

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.