Pulse cyclophosphamide therapy for steroid-resistant focal segmental glomerulosclerosis in children

In children, steroid-resistant nephritic syndrome due to focal segmental glomerulosclerosis (FSGS) is frequently a progressive condition resulting in end-stage renal disease (ESRD). We report the response of 15 patients with steroid resistant FSGS to treatment with intravenous pulse cyclophosphamide (IVCP) and oral prednisone after 4 years of follow up. Five patients had initial steroid resistance and ten patients had late steroid resistance.

All patients were treated with IVCP at a dose of 500 mg/m2/month for 6 months. Adjunctive prednisolone was given at a dose of 60 mg/m2/day for 4 weeks followed by 40 mg/m2/ on alternate days for 4 weeks and then tapered over next 4 weeks.

All patients with initial resistance to steroids showed no response to IVCP and continued to be steroid resistant. Three developed CRF during the observation period. The other ten patients with late steroid resistance responded to IVCP, but all were steroid dependent at the end of the observation period. Five could not be weaned from steroids during the IVCP treatment period. The other five patients achieved relatively prolonged remission (7 months to 24 months), but eventually become steroid dependent.

Sixty-seven percent of steroid-resistant FSGS becomes steroid dependent. Patients with initial steroid resistance did not respond to IVCP. We found no correlation between IgM deposition and the response to therapy. The side effects of IVCP were negligible. Beneficial therapy for initial steroid-resistant FSGS remains to be determined.