Corresponding Author: James C. M. Chan, MD, University of Vermont; The Barbara Bush Children's Hospital, Maine Medical Center, 22 Bramhall Street, Portland, Maine 04102-3175, USA (Tel: 1-207-662-2439; Fax: 1-207-662-6272; Email: chanj@mmc.org)

Background: By sharing our quarter century of care on children with kidney biopsy-proven focal segmental glomerulosclerosis (FSGS), we hope to advance early recognition of this condition with its changing demographics in recent years and examine promising treatment modalities to slow its rate of progression. 

Methods: Children fitting entry and exclusion criteria, who presented to our regional nephrology center, were enrolled into this retrospective study. The same criteria for kidney biopsies were uniformly applied under the same nephrologists in over 25 years of practice. Patients exited the study at the initiation of dialysis/transplantation or death. Kidney survival rate was determined by the Kaplan-Meier method.

Results: In 43 children (21 females and 22 males) enrolled, 15 were white and 28 black. The peak age at presentation was 10 to 15 years. Hypertension and heavy proteinuria were frequently seen as presenting features. Almost half of the patients showed serum creatinine concentrations minimally elevated above normal, indicating relatively early diagnosis of FSGS in the study population. Treatment consisted of one or more of the following medications: oral alternative-day prednisone, angiotensin converting enzyme inhibitors or angiotensin receptor blockers, vitamin E, and intravenous methylprednisolone with cyclophosphamide or cyclosporine A.

Conclusions: In this study, with the early interest in FSGS, an opportunity became available to use the different treatment options over more than 2 decades. With the present combined therapy, the kidney survival was no better than the therapeutic regimen described earlier by others. Therefore attention must be focused on the efficacy and side effects of treatments which have been called into question: cyclophosphamide and intravenous methylprednisolone. We recommend a modified treatment regimen, which is cost effective and with minimal side effects.

Key words: focal segmental glomerulosclerosis; African-American children; natural history; therapy; outcome