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

Pediatr Nephrol. 2010 Mar;25(3):453-60. doi: 10.1007/s00467-009-1356-x.


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.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Age of Onset
  • Anti-Inflammatory Agents / adverse effects
  • Anti-Inflammatory Agents / therapeutic use*
  • Child
  • Child, Preschool
  • Cyclophosphamide / therapeutic use*
  • Cyclosporine / therapeutic use
  • Drug Resistance
  • Ethnicity
  • Female
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Kidney Function Tests
  • Male
  • Mycophenolic Acid / adverse effects
  • Mycophenolic Acid / analogs & derivatives*
  • Mycophenolic Acid / therapeutic use
  • Nephrotic Syndrome / drug therapy*
  • Nephrotic Syndrome / pathology
  • Sex Characteristics
  • Steroids / therapeutic use*


  • Anti-Inflammatory Agents
  • Immunosuppressive Agents
  • Steroids
  • Cyclosporine
  • Cyclophosphamide
  • Mycophenolic Acid