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

Valderez Raposo de Mello1, Maira Tinte Rodrigues, Tais Helena Mastrocinque

  • 1Department of Pediatrics, Division of Pediatric Nephrology, Santa Casa de São Paulo, Rua Cesario Motta Jr, 112 ZC 01221-020 São Paulo, Brazil.

Pediatric Nephrology (Berlin, Germany)
|November 26, 2009
PubMed

Insights

Mycophenolate mofetil (MMF) shows therapeutic effectiveness in children with difficult-to-treat idiopathic nephrotic syndrome (INS). MMF is effective whether used alone or after cyclosporine A, with manageable side effects.

Area of Science:

  • Pediatric Nephrology
  • Immunosuppressive Therapy

Background:

  • Idiopathic nephrotic syndrome (INS) is a significant cause of kidney disease in children.
  • Steroid and cyclophosphamide resistance presents a therapeutic challenge in managing INS.
  • Limited treatment options exist for patients refractory to standard therapies.

Purpose of the Study:

  • To evaluate the efficacy and safety of mycophenolate mofetil (MMF) in children with steroid- and cyclophosphamide-resistant INS.
  • To compare outcomes of MMF therapy alone versus MMF following cyclosporine A (CsA) treatment.
  • To assess the long-term effects and side effects of MMF in this patient population.

Main Methods:

  • A retrospective study involving two groups of children with INS.
  • Group 1 received MMF after CsA treatment (n=34).
  • Group 2 received MMF as a standalone therapy (n=18).
  • Treatment duration was at least 6 months with a 2-year follow-up.

Main Results:

  • Overall therapeutic effectiveness of MMF was 59.5%.
  • In Group 1, complete/partial remission occurred in 59.2% of patients.
  • In Group 2, complete/partial remission occurred in 61.1% of patients.
  • No significant difference in efficacy was observed between the two groups.
  • Main side effects included gastrointestinal issues (21%) and infections (1.9%).
  • Eight patients developed chronic kidney disease.

Conclusions:

  • Mycophenolate mofetil (MMF) is a viable therapeutic option for children with steroid- and cyclophosphamide-resistant idiopathic nephrotic syndrome (INS).
  • MMF demonstrated comparable efficacy when used alone or subsequent to cyclosporine A.
  • Further long-term studies are warranted to confirm sustained benefits after treatment discontinuation.

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