Category: Original Article: Efficacy and safety of oral cyclophosphamide versus mycophenolate mofetil in childhood nephrotic syndrome: An open-label comparative study.

Abstract Background: The aim is to study the efficacy and safety of oral cyclophosphamide (CYC) and mycophenolate mofetil (MMF) in children with steroid-sensitive nephrotic syndrome for the proportion of children off steroids for at least 6 months (responders). Methods: This is an open-label comparative study done in a Pediatric Nephrology clinic of a referral center on children between 3 months and 18 years with FR/SD nephrotic syndrome. Group A was patients receiving oral cyclophosphamide (100, 25% female) with a dose of 2–2.5 mg/kg once daily for 8–12 weeks of therapy. Group B was patients receiving oral Mycophenolate Mofetil (n = 61, 18% female) (dose: 800-1200 mg/m²) for at least 12 months. Responders were defined as children off steroids for at least 6 months. Results: In the CYC group, the proportion of responders was 50% compared to 54% in the MMF group (p = 0.614). The time to first relapse with CYC was 7 months (IQR 5.25–11) versus 7 months (IQR 3.5–12) with MMF (p = 0.092). The incidence relapse rate in CYC group was 1.77 relapses per patient-year compared to 1.295 relapses per patient-year in the MMF group. The difference in relapse rate was significant (-0.474; 95% CI, 0.09 to 0.86 relapses/person-year) (p-value = 0.009). Multivariate analysis showed age less than 5 years at the start of medication was a significant factor in showing a better response with MMF (p-value =0.039, OR = 2.988, CI -1.055-8.468). Conclusions: The efficacy of MMF was similar to CYC in terms of response (off steroid for 6 months) in children with FR/SD nephrotic syndrome. Whereas MMF showed a favorable response in the frequency of relapses, treatment failure. Trial registration at Clinical Trials Registry of India: (http://ctri.nic.in;CTRI/2021/06/034421) (Dt: 28/06/2021)