Randomized controlled trial to compare safety and efficacy of mycophenolate vs. cyclosporine after rituximab in children with steroid‐resistant nephrotic syndrome

Abstract Objective The comparative safety and efficacy of maintenance mycophenolate mofetil (MMF) and cyclosporine (CYC) following rituximab (RTX) in children with steroid‐resistance nephrotic syndrome are uncertain. Study Design and setting Multicenter randomized controlled trial. Patients Sixty‐six children between 2 and 6 years of age with SRNS. Interventions Patients were randomized to receive either MMF 1000 mg/m 2 /day ( n = 32) or CYC 5 mg/kg/day ( n = 34) for 12 months following RTX induction therapy (375 mg/m 2 ) given as needed for B‐cell count. Main outcomes Complete remission and adverse events (AEs). Results Complete remission was observed in 26 patients (83.1%) in the MMF group compared with 21 patients (61.7%) in the CYC group ( p = 0.02). The median time to remission was shorter in the MMF group than in the CYC group (2.64 vs. 3.4 months; hazard ratio [HR], 0.61; 95% CI, 0.74–0.90, p = 0.03). The median time to first relapse was longer in the MMF group compared with the CYC group (10.8 vs. 8.0 months; HR, 1.12; 95% CI, 1.31–1.54, p = 0.01), and this was significantly correlated with the median time to B‐cell recovery in the two groups (8.6 vs. 5.2 months in MMF and CYC, respectively, p = 0.02). The overall incidence of adverse drug events was lower in the MMF group compared with the CYC group (59.3% vs. 76.4%, p = 0.03). Conclusions MMF‐RTX is superior to CYC‐RTX in maintaining remission with fewer AEs in children with initial SRNS. Additional high‐quality randomized control trials with long‐term follow‐up are needed to identify the long‐term potential complications.