Intravenous pulsed vs oral cyclophosphamide therapy in steroid dependent nephrotic syndrome

Background: No consensus exists concerning dose and route of administration of cyclophosphamide (CYC) in steroid-dependant nephrotic syndrome (SDNS).

Objective: To compare the outcome of children with SDNS treated with either intravenous (IV) or oral CYC in a single centre in Sri Lanka by reviewing data from 2002 to 2011.

Method:  One hundred and twenty seven children with SDNS with evidence of steroid toxicity received either oral or IV cyclophosphamide. Seventy two received IV cyclophosphamide in a dose of 500 mg/m² monthly for 6 months, and 55 received oral CYC in a dose of 3 mg/kg per day for 8 weeks. In both groups prednisolone was tapered over 6 months in a similar manner. Full blood counts were done weekly during oral treatment and monthly during IV treatment. Patient progress was assessed on a monthly basis for the first year and at 3 monthly intervals thereafter till 5 years.

Results: In the oral CYC group, 4 patients developed bone marrow suppression, 7 had serious infections, 15 had significant alopecia and one child died due to overwhelming sepsis. In the IV group alopecia was seen in 9 patients and one had a serious infection while bone marrow suppression was not seen. At one and 5 year follow ups there was no significant difference in the proportion who suffered a relapse in the 2 groups (p >0.05)

Conclusion: In SDNS, IV cyclophosphamide was as effective as oral CYC in inducing sustained remission and had less side effects and a smaller total dose. 

Sri Lanka Journal of Child Health, 2017; 46(4): 317-321