Intravenous pulse cyclophosphamide - is it effective in children with steroid-resistant nephrotic syndrome?

Treatment of steroid-resistant nephrotic syndrome (SRNS) remains a challenge to pediatric nephrologists. Recently, intravenous cyclophosphamide (IV-CPM) infusion was shown to be effective, safe, and economical for the treatment of SRNS, particularly minimal change disease (MCD), as it results in more sustained remissions, longer periods without proteinuria, and fewer significant side effects at a lower cumulative dose. A prospective study was conducted to evaluate IV-CPM infusions in the management of children with SRNS secondary to MCD or IgM nephropathy. Five patients with SRNS (4 IgM nephropathy and 1 MCD) received six monthly IV-CPM infusions at a dose of 500 mg/m(2). No patient achieved complete or sustained remission. Three patients attained partial remission, which was not sustained for more than 1 month post therapy. One patient progressed rapidly to end-stage renal disease during treatment. Side effects included vomiting in four patients and alopecia in one patient. Conclusion: IV-CPM pulse therapy at a dose of 500 mg/m(2) is unsuccessful in obtaining complete or sustained remission in children with SRNS secondary to IGM nephropathy or MCD. Further randomized controlled studies with higher doses are required.