Primary Focal Segmental Glomerulosclerosis, an Important Component of Childhood Nephrotic Syndrome: Therapeutic Options and Genetic Basis

Abstract

Introduction: We aim to review the therapeutic options in ameliorating the progression of focal segmental glomerulosclerosis (FSGS). We shall comment on the long-term prognosis and the clinical implications of candidate genes in familial cases of FSGS. Materials and Method: Key references from the past concerning FSGS were analyzed, together with a PubMed search of the literature from 1998 to 2008. Results and Discussion: Treatment of FSGS consists of one or more of the following medications: vitamin E, prednisone, angiotensin-converting enzyme inhibitor, angiotensin receptor blocker; in the severe cases, methylprednisolone, cyclo- phosphamide, cyclosporine, calcineurin inhibitors, rituximab and mycophenolate. The prognoses with lengths of follow- up to 14 years were analyzed. The genetics of familial FSGS and the strategy of kidney transplants in such cases need special attention. Conclusion: We reviewed the available modalities of treatment and the long-term prognosis. The new findings on familial forms of FSGS and the clinical implications are succinctly presented.