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Mycophenolate mofetil versus cyclosporin A in children with frequently relapsing nephrotic syndrome
Jutta Gellermann1, Lutz Weber, Lars Pape
1Department of Pediatric Nephrology, Charité Universitätsmedizin Berlin CVK, Berlin, Germany;
Insights
Mycophenolate mofetil (MMF) showed more relapses than cyclosporin A (CsA) in children with frequently relapsing steroid-sensitive nephrotic syndrome (FR-SSNS). However, MMF may be a less nephrotoxic option with improved kidney function markers.
Area of Science:
- Pediatric Nephrology
- Immunosuppressive Therapy
- Pharmacokinetics
Background:
- Long-term corticosteroid or cyclosporin A (CsA) therapy for frequently relapsing steroid-sensitive nephrotic syndrome (FR-SSNS) in children causes severe side effects.
- Alternative immunosuppressive agents are needed to improve treatment outcomes and reduce toxicity.
Purpose of the Study:
- To compare the efficacy and safety of mycophenolate mofetil (MMF) versus CsA in pediatric patients with FR-SSNS.
- To evaluate relapse rates, time to relapse, and pharmacokinetic profiles of MMF and CsA.
- To assess the impact of MMF and CsA on renal function and other safety parameters.
Main Methods:
- A randomized, multicenter, open-label, crossover study involving 60 pediatric patients with FR-SSNS.
- 1-year treatment with MMF (target plasma mycophenolic acid trough level of 1.5-2.5 µg/ml) or CsA (target trough level of 80-100 ng/ml).
- Assessment of relapse frequency, time without relapse, pharmacokinetic profiles (AUC), blood pressure, growth, lipid levels, and renal function markers.
Main Results:
- More relapses per patient per year occurred with MMF than with CsA during the first year (P=0.03).
- Higher exposure to mycophenolic acid (AUC >50 µg⋅h/ml) was associated with fewer relapses (P<0.05).
- MMF treatment showed significant increases in cystatin clearance, estimated GFR, and hemoglobin levels compared to CsA, suggesting less nephrotoxicity.
Conclusions:
- MMF is less effective than CsA in preventing relapses in pediatric FR-SSNS patients.
- MMF may offer a safer alternative with a potentially lower nephrotoxic profile.
- Optimizing MMF dosing based on pharmacokinetic exposure (AUC) could improve its efficacy in FR-SSNS treatment.
Abstract:
The severe side effects of long-term corticosteroid or cyclosporin A (CsA) therapy complicate the treatment of children with frequently relapsing steroid-sensitive nephrotic syndrome (FR-SSNS). We conducted a randomized, multicenter, open-label, crossover study comparing the efficacy and safety of a 1-year treatment with mycophenolate mofetil (MMF; target plasma mycophenolic acid trough level of 1.5-2.5 µg/ml) or CsA (target trough level of 80-100 ng/ml) in 60 pediatric patients with FR-SSNS. We assessed the frequency of relapse as the primary endpoint and evaluated pharmacokinetic profiles (area under the curve [AUC]) after 3 and 6 months of treatment. More relapses per patient per year occurred with MMF than with CsA during the first year (P=0.03), but not during the second year (P=0.14). No relapses occurred in 85% of patients during CsA therapy and in 64% of patients during MMF therapy (P=0.06). However, the time without relapse was significantly longer with CsA than with MMF during the first year (P<0.05), but not during the second year (P=0.36). In post hoc analysis, patients with low mycophenolic acid exposure (AUC <50 µg⋅h/ml) experienced 1.4 relapses per year compared with 0.27 relapses per year in those with high exposure (AUC>50 µg⋅h/ml; P<0.05). There were no significant differences between groups with respect to BP, growth, lipid levels, or adverse events. However, cystatin clearance, estimated GFR, and hemoglobin levels increased significantly with MMF compared with CsA. These results indicate that MMF is inferior to CsA in preventing relapses in pediatric patients with FR-SSNS, but may be a less nephrotoxic treatment option.
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