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

James C.M. Chan1, *, Karl S. Roth2
1 University of Vermont College of Medicine, The Barbara Bush Children’s Hospital, Maine Medical Center, Portland, Maine, USA
2 Department of Pediatrics, Creighton University, Omaha, Nebraska, USA

© 2008 Chan and Roth

open-access license: This is an open access article distributed under the terms of the Creative Commons Attribution 4.0 International Public License (CC-BY 4.0), a copy of which is available at: This license permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

* Address correspondence to this author at the University of Vermont College of Medicine, The Barbara Bush Children’s Hospital, Maine Medical Center, 22 Bramhall Street, Portland, Maine 04102-3175, USA; USA; Tel: 207- 871-2439; Fax: 207-871-6272; E-mail:



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, cyclophosphamide, cyclosporine, calcineurin inhibitors, rituximab and mycophenolate. The prognoses with lengths of followup to 14 years were analyzed. The genetics of familial FSGS and the strategy of kidney transplants in such cases need special attention.


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.

Keywords: Focal segmental glomerulosclerosis, combined therapy, long-term outcome.