Safety and efficacy of intravenous pulse cyclophosphamide therapy in children with steroid-dependent nephrotic syndrome

Abstract

Background: Cyclophosphamide (Cyp) is a well-known alternative agent to spare the use of steroids and avoid the side effects that result from long-term steroid therapy in children with idiopathic nephrotic syndrome (NS). Use of Cyp typically reduces the risk of relapse in comparison with prednisolone by about 50%. Objective: To study the safety and efficacy of intravenous pulse Cyp (IV Cyp) therapy in children with steroid-dependent NS (SDNS). Materials and Methods: This was a retrospective single-center analysis from a large government hospital in South India. The data were retrieved from the records of children attending the pediatric nephrology clinic between 2005 and 2016. Children with SDNS who received the complete schedule of IV Cyp were included in the study. Children who received other alternate drugs were excluded. Results: Fifty patients with SDNS were treated with IV Cyp, seven patients did not complete the treatment and were excluded from the analysis. 24 (56%) of the 43 patients stayed in remission throughout the course of Cyp treatment and prednisolone could be stopped and were considered as Cyp responsive. The remaining 19 (44%) children had relapsed while on Cyp therapy (Cyp resistant) and required treatment with alternate immunosuppressant drugs. 19 of the 24 Cyp responsive patients were in remission 6 months after stopping Cyp treatment (Cyp sensitive) while the remaining five patients relapsed within 6 months of stopping Cyp treatment (Cyp dependent). 9 children (20.9%) were relapse-free till the time of last follow-up with a median follow-up of 2.1 years. Among 24 children, 13 (30.23%) who responded were infrequently relapsing NS and 2 (4.5%) children were frequently relapsing NS.