JCM Chan

Abstract

Objectives: We aim to review the therapeutic options in ameliorating the progression of steroid resistant-focal segmental glomerulosclerosis (SR-FSGS). We shall comment on the clinical implications of candidate genes in familial cases of FSGS. Methods: Selected key references concerning SR-FSGS were analysed, together with a PubMed search of the literature from 1998 to 2009 and a search on current clinical trials registered with ClinicalTrials.gov. Findings: Treatment options consist of one or more of the following medications: vitamin E, prednisone, angiotensin-converting enzyme inhibitor, angiotensin receptor blocker. In the resistant cases, methylprednisolone, cyclophosphamide, cyclosporine, calcineurin inhibitors, rituximab, galactose, and mycophenolate mofetil have been used. In this review, the current clinical trials in SR-FSGS are critiqued. The new findings on the genetics of familial forms of FSGS are reviewed. Conclusions: The available modalities of treatment are only moderately effective. Research into the genetics of familial FSGS may finally provide insight into the pathogenesis of injury to the podocytes, central to the development of FSGS and may point to novel therapy to improve the long-term outcome of SR-FSGS.