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Mycophenolate mofetil in treatment of childhood steroid-resistant nephrotic syndrome

Tahar T G Gargah1, Mohamed Rachid Lakhoua

  • 1Department of Pediatric Nephrology, Charles Nicolle Hospital, Tunis, Tunisia. kitomora@yahoo.fr

Journal of Nephrology
|February 15, 2011
PubMed
Abstract

Insights

Mycophenolate mofetil (MMF) showed limited efficacy in treating pediatric steroid-resistant nephrotic syndrome (SRNS), with only one-third of patients achieving remission. Further research is needed to explore its potential in refractory cases.

Area of Science:

  • Pediatric Nephrology
  • Immunosuppressive Therapy
  • Glomerular Diseases

Background:

  • Steroid-resistant nephrotic syndrome (SRNS) presents limited treatment options.
  • Cyclosporine shows some efficacy, but mycophenolate mofetil (MMF) is a promising alternative due to its lack of nephrotoxicity.
  • This study evaluates the efficacy and tolerance of MMF in pediatric SRNS.

Observation:

  • Six pediatric patients with SRNS received MMF combined with oral prednisolone.
  • Patients had various underlying pathologies including focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD), and diffuse mesangial proliferation glomerulonephritis (DMP).
  • Treatment duration was planned for 12 weeks with specific MMF dosage adjustments to maintain mycophenolic acid levels.

Findings:

  • Only one patient (with MCD) achieved complete remission; another experienced partial remission.
  • While overall remission rates were low (one-third response), some non-responders showed reduced proteinuria and increased serum albumin.
  • No significant improvements in glomerular filtration rate were observed.

Implications:

  • MMF demonstrates a low response rate in pediatric SRNS, suggesting it may not be a primary treatment.
  • The observed benefits in proteinuria and albumin in some non-responders warrant further investigation.
  • MMF could be considered for select SRNS patients refractory to standard therapies, despite limited efficacy in this cohort.

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