Mycophenolate mofetil in children with steroid/cyclophosphamide-resistant nephrotic syndrome

The purpose of this study was to assess the results of therapy with mycophenolate mofetil (MMF) in children with idiopathic nephrotic syndrome (INS) who were both steroid- and cyclophosphamide-resistant. Treatment lasted a minimum of 6 months, and follow-up data were collected over a 2-year period. The children were divided into two groups: Group 1 ( n = 34) comprised patients who had received cyclosporine A (CsA) before the initiation of MMF therapy; Group 2 ( n = 18) comprised patients who received only MMF. Among the 34 patients of Group 1, complete and partial remission were achieved in seven (20.6%) and 13 patients (38.6%), respectively; there was no response in 14 patients (41.2%). Among the 18 patients in Group 2, complete and partial remission occurred in five (27.8%) and six (33.3%) patients, respectively; there was no response in seven patients (38.9%). Eight patients developed chronic kidney disease. The main side-effects were gastrointestinal complaints ( n = 11, 21%), recurring severe infections ( n = 1, 1.9%), and mild thrombocytopenia/leucopenia ( n = 1, 1.9%). MMF proved to be therapeutically effective in 59.5% of the cases. These beneficial effects need to be confirmed in studies with a long-term follow-up after discontinuation of the treatment. Our statistical analysis of the results of therapy with MMF did not reveal any significant difference between its use alone or following CsA administration.