Mycophenolate mofetil in steroid/cyclosporine-dependent/resistant nephrotic syndrome

Pediatr Nephrol. 2005 Jul;20(7):914-9. doi: 10.1007/s00467-005-1877-x. Epub 2005 May 13.


Attempts to minimize the effects of prolonged steroid use in steroid-dependent nephrotic syndrome (SDNS) and the need to overcome steroid resistance (SRNS) justifies immunosuppressant therapy. We report our experience in a cohort of patients with SD/SRNS during the administration of mycophenolate mofetil (MMF) in a prospective protocol initiated in January 2001. Twenty-six children with idiopathic nephrotic syndrome were included (21 steroid dependent and 5 steroid resistant), whose response did not change after sequential treatment with cyclophosphamide (CPM) and cyclosporine (CsA). Histopathologic patterns were: 11 minimal change disease (MCD), 1 diffuse mesangial proliferation (DMP), 13 focal segmental glomerulosclerosis (FSGS) and membranous 1 glomerulonephritis (MGN). The median age of onset of NS was 2.8 years (range 1.2-12.5), and treatment with MMF was performed at a median age of 11.4 years (range 5-17) with an initial dose of 600 mg/m(2)/12 h, adjusted to maintain levels of mycophenolic acid (MPA) at 2.5-5 mcg/ml. The planned duration of study to assess treatment efficacy was 6 months. The mean MMF dose required was 624 (SD=136) mg/m(2)/12 h (range 415-970), which maintained mean C(0)-MPA levels of 2.9 (SD=1.17) mcg/ml (range 1.2-5.9 mcg/ml). In the five patients with SRNS, only one achieved complete remission. In the patients with SDNS, steroid sparing was achieved in 15 and 9 remained in remission on MMF monotherapy. Withdrawal of MMF resulted in immediate relapse in 47%. In our study, MMF was a useful immunosuppressant due to its fewer undesirable effects and similar efficacy to other drugs used. It appears effective for the maintenance of remission in SDNS patients, with a response similar to that of CsA.

Publication types

  • Clinical Trial

MeSH terms

  • Child
  • Child, Preschool
  • Cohort Studies
  • Cyclophosphamide / therapeutic use
  • Cyclosporine / therapeutic use*
  • Dose-Response Relationship, Drug
  • Drug Resistance
  • Glucocorticoids / administration & dosage
  • Glucocorticoids / therapeutic use
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Mycophenolic Acid / analogs & derivatives*
  • Mycophenolic Acid / therapeutic use
  • Nephrotic Syndrome / drug therapy*
  • Prednisone / administration & dosage
  • Prednisone / therapeutic use
  • Prospective Studies
  • Steroids / therapeutic use*
  • Treatment Outcome


  • Glucocorticoids
  • Immunosuppressive Agents
  • Steroids
  • Cyclosporine
  • Cyclophosphamide
  • Mycophenolic Acid
  • Prednisone