title: Long-term treatment of focal segmental glomerulosclerosis in children with cyclosporine given as a single daily dose.
abstract: Cyclosporine (CsA) has been successfully used for treatment of children with focal segmental glomerulosclerosis (FSGS) and nephrotic syndrome (NS) for the last decade. Response rates of 50% to 100% have been reported using twice-daily dosing of 5 to 32 mg/kg/d, achieving trough blood levels of 70 to 500 ng/mL. Treatment has been associated with a high incidence of side effects, including nephrotoxicity, hypertension, gingival hyperplasia, and hirsutism. To determine whether once-daily low-dose CsA could minimize side effects and still induce remission, 21 children with biopsy-proven FSGS and NS, each treated with CsA, 4.6 +/- 0.8 mg/kg/d, with no predetermined target trough blood levels, were studied. Eleven of 21 children (52%) attained complete remission and 5 of 21 children (24%) attained partial remission, for a total response rate of 76%. Mean time to response was 2.8 +/- 0.8 months, and mean duration of therapy was 20.6 +/- 13.7 months. CsA dosage was tapered or stopped in 9 responders; 3 of these patients maintained remission at last follow-up 6 to 13 months later, and 6 patients relapsed at 1.5 to 18.7 months (mean, 8.7 months). Five of these 6 patients responded again when CsA therapy was restarted or the dosage was increased. Twelve of 16 responders were still being administered CsA at last follow-up 11 to 60 months (mean, 24.6 months) later. Five of 21 patients (24%) had no response to CsA during 2 to 27 months of therapy; 4 of these 5 patients developed end-stage renal disease after CsA therapy was stopped. Side effects of CsA therapy were minimal: 1 patient each developed new-onset hypertension or gingival hyperplasia, and no patient had hirsutism or nephrotoxicity. Single daily low-dose CsA appears to be effective for long-term treatment of children with FSGS and NS, with fewer side effects than twice-daily dosing.
authors.affiliation: Department of Pediatrics, Renal Section, Baylor College of Medicine and Texas Children's Hospital, Houston, TX 77030, USA.
authors.forename: A S
authors.initials: AS
authors.lastname: Chishti
authors.forename: J M
authors.initials: JM
authors.lastname: Sorof
authors.forename: E D
authors.initials: ED
authors.lastname: Brewer
authors.forename: A S
authors.initials: AS
authors.lastname: Kale
mesh_terms: D000293:Adolescent; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000008:administration & dosage*; D004334:Drug Administration Schedule; D004656:Enalapril / Q000627:therapeutic use; D005260:Female; D005923:Glomerulosclerosis, Focal Segmental / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D007676:Kidney Failure, Chronic / Q000209:etiology; D008297:Male; D009404:Nephrotic Syndrome / Q000150:complications / Q000188:drug therapy*; D012074:Remission Induction
chemical_list: D000806:Angiotensin-Converting Enzyme Inhibitors; D007166:Immunosuppressive Agents; D004656:Enalapril; D016572:Cyclosporine