FOCAL SEGMENTAL GLOMERULOSCLEROSIS IN YOUNG PATIENTS

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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.