Use of mycophenolate mofetil in steroid-dependent and -resistant nephrotic syndrome

Pediatr Nephrol. 2003 Aug;18(8):833-7. doi: 10.1007/s00467-003-1175-4. Epub 2003 May 28.


Cyclosporin (Cs-A) is an effective treatment for difficult cases of nephrotic syndrome (NS), but its use can be complicated by renal toxicity and a high incidence of relapses after withdrawal. We reviewed the charts of 10 Cs-A-dependent patients and 4 patients with steroid-dependent nephrotic syndrome (SDNS) not previously treated with Cs-A therapy. All patients had persistent NS, even after prior treatment with oral cyclophosphamide. Of 10 patients treated with Cs-A, 4 had surveillance renal biopsies consistent with Cs-A toxicity, and 8 of 10 had interstitial fibrosis prior to mycophenolate mofetil (MMF). Patients were treated with MMF, at 1,200 mg/m(2) per day, in an attempt to allow weaning of Cs-A and/or steroid therapy, and reduce the frequency of relapses. Overall, a significant decrease in frequency of relapses was noted after initiation of MMF therapy. In addition, 5 patients were weaned off Cs-A by 1-2 years of follow-up. One patient was weaned off Cs-A and MMF, and remained in complete remission. However, the subgroup of patients with frequently relapsing SDNS not treated with Cs-A appeared to have a reduction in the number of relapses while on MMF that did not reach statistical significance. Two patients with intractable steroid-resistant NS continued to relapse repeatedly on MMF and Cs-A therapy. We conclude that in this small, single-center, uncontrolled experience, MMF therapy in patients with Cs-A-dependent NS appears to be effective in reducing Cs-A exposure. In addition, MMF appears to significantly decrease the frequency of relapses in this patient population. Further controlled studies are warranted to better define the potential efficacy and side effects of long-term MMF therapy in this setting.

MeSH terms

  • Anti-Inflammatory Agents, Non-Steroidal / administration & dosage*
  • Anti-Inflammatory Agents, Non-Steroidal / adverse effects
  • Child
  • Child, Preschool
  • Cyclophosphamide / administration & dosage
  • Drug Resistance
  • Drug Therapy, Combination
  • Glucocorticoids / administration & dosage*
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Infant
  • Mycophenolic Acid / administration & dosage*
  • Mycophenolic Acid / adverse effects
  • Mycophenolic Acid / analogs & derivatives*
  • Nephrotic Syndrome / drug therapy*
  • Prednisone / administration & dosage*
  • Retrospective Studies
  • Secondary Prevention
  • Treatment Outcome


  • Anti-Inflammatory Agents, Non-Steroidal
  • Glucocorticoids
  • Immunosuppressive Agents
  • Cyclophosphamide
  • Mycophenolic Acid
  • Prednisone