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

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 /day (n = 32) or CYC 5 mg/kg/day (n = 34) for 12 months following RTX induction therapy (375 mg/m ) 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.