Seven children with
steroid-resistant
focal segmental glomerulosclerosis (SR-FGS) were placed on a therapeutic protocol of
methylprednisolone (MP), oral
prednisone (
pred) and oral
cyclophosphamide (CYC) given over 16 months (regimen A). Another 5 children with SR-FGS were treated with a shorter course of intravenous CYC (monthly doses over 6 months), intravenous MP (3 consecutive daily doses) and oral
pred 2 mg/kg (alternate days) (
regimen B). With regimen A, 1 child had a short remission, and in the others, oedema subsided, the urine
protein/
creatinine ratio decreased, haematuria disappeared and the estimated glomerular filtration rate (GFR) increased. The observation period was 21-42 months and the drugs were well tolerated. With
regimen B, 2 patients went into complete remission, 1 had partial remission, 1 failed to respond and another died because of severe concurrent
infections. In the responding children, oedema cleared, the urine
protein/
creatinine ratio decreased, haematuria disappeared and the GFR rose. The follow-up was between 3 and 34 months. Minor side effects were
alopecia and transient
hypertension. Both regimens improved the quality of life of most children. Compared with regimen A,
regimen B is six times less costly with a quarter of the number of hospital visits. These observations may be of value in designing appropriate multicentre controlled trials, which have been advocated recently, for the rational and optimum management of SR-FGS.