Steroid-responsive nephrotic syndrome of childhood: a long-term study of clinical course, histopathology, efficacy of cyclophosphamide therapy, and effects on growth.

The long-term clinical course of 60 children with steroid-responsive nephrotic syndrome, observed for a minimum of 10 years from onset, was studied (mean 14.5 +/- 0.5 years). Four children had only a single episode, seven children experienced only one to three relapses early in their course, and the remaining 49 patients (82%) experienced frequently relapsing steroid-dependent disease. Nearly half of these (47%) continued to relapse into their late teens and early twenties. All 20 children treated with cyclophosphamide because of steroid-induced side effects developed complete remissions of the nephrotic syndrome. These were sustained in 70% for 9.1 +/- 0.6 years, with a reduction of disease severity in the remaining 30%. In contrast, only 48% of patients treated with prednisone alone were in remission at last follow-up (P = .06). Ten of the children treated with cyclophosphamide had the minimal change lesion prior to therapy; 90% of these had permanent remissions. Only 50% of the six children with focal glomerulosclerosis and four children with mesangial proliferation have had permanent remissions. None of the patients developed renal insufficiency. Children treated with prednisone alone were -0.93 +/- 0.3 SD below the mean for height at last follow-up. Cyclophosphamide treatment was associated with an increase in height SD scores from -0.84 +/- 0.4 to -0.28 +/- 0.3. Children with severe growth impairment demonstrated dramatic catch-up growth when treated with cyclophosphamide with SD scores increasing from -2.29 +/- 0.8 to -0.43 +/- 0.6 (P less than .05).(ABSTRACT TRUNCATED AT 250 WORDS)