Safety and efficacy of mycophenolate mofetil as first-line immunosuppressive therapy in children with steroid-dependent nephrotic syndrome

Abstract

Introduction. More than half of children with idiopathic nephrotic syndrome have relapsed or become steroid-dependent after the disease’s onset. So far they, require long-term therapy with glucocorticosteroids and/or other immunosuppressive treatment to maintain remission. Despite the widespread use of immunosuppressive agents, there is no consensus for appropriate first-line therapy. Materials and methods. A retrospective study included children presenting with steroid-dependent nephrotic syndrome who received treatment with mycophenolate mofetil in the Nephrology department between 2003 and 2018 at the National Medical Research Center for Children’s Health (Moscow). Results. In total, withdrawal of corticosteroids was recorded in 24 (85.7%) patients, while the median remission period was 23 [12; 30] months, after glucocorticosteroids cessation - 19 [10; 24.5] months, the cumulative dose of steroids was reduced from 189 [125.9; 240] to 45.5 [19.5; 68.2] mg/kg per year and relapses rate per year from 1.9 [1.35; 2.35] to 0 during a year. Among children who did not overcome steroid dependence, the treatment failed in 3 patients, and in 1 child, the steroid dosage has been reduced to the lower dose. Conclusion. Mycophenolate mofetil has a favorable efficacy due to the significant reduction of the cumulative dose of steroids and maintenance of long-term remission of nephrotic syndrome after glucocorticosteroid cessation and may be recommended as the first-line therapy in children with steroid-dependent nephrotic syndrome.