Rituximab in children with resistant idiopathic nephrotic syndrome

J Am Soc Nephrol. 2012 Jun;23(6):1117-24. doi: 10.1681/ASN.2011080775. Epub 2012 May 10.


Idiopathic nephrotic syndrome resistant to standard treatments remains a therapeutic dilemma in pediatric nephrology. To test whether the anti-CD20 monoclonal antibody rituximab may benefit these patients, we conducted an open-label, randomized, controlled trial in 31 children with idiopathic nephrotic syndrome unresponsive to the combination of calcineurin inhibitors and prednisone. All children continued prednisone and calcineurin inhibitors at the doses prescribed before enrollment, and one treatment group received two doses of rituximab (375 mg/m(2) intravenously) as add-on therapy. The mean age was 8 years (range, 2-16 years). Rituximab did not reduce proteinuria at 3 months (change, -12% [95% confidence interval, -73% to 110%]; P=0.77 in analysis of covariance model adjusted for baseline proteinuria). Additional adjustment for previous remission and interaction terms (treatment by baseline proteinuria and treatment by previous remission) did not change the results. In conclusion, these data do not support the addition of rituximab to prednisone and calcineurin inhibitors in children with resistant idiopathic nephrotic syndrome.

Publication types

  • Comparative Study
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Albumins / metabolism
  • Antibodies, Monoclonal / therapeutic use
  • Antibodies, Monoclonal, Murine-Derived / therapeutic use*
  • Calcineurin Inhibitors*
  • Child
  • Child, Preschool
  • Confidence Intervals
  • Creatinine / blood
  • Dose-Response Relationship, Drug
  • Drug Administration Schedule
  • Drug Resistance
  • Drug Therapy, Combination
  • Female
  • Follow-Up Studies
  • Humans
  • Kidney Function Tests
  • Male
  • Nephrotic Syndrome / diagnosis*
  • Nephrotic Syndrome / drug therapy*
  • Patient Selection
  • Prednisone / therapeutic use
  • Prospective Studies
  • Proteinuria / physiopathology
  • Reference Values
  • Risk Assessment
  • Rituximab
  • Severity of Illness Index
  • Treatment Outcome


  • Albumins
  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Murine-Derived
  • Calcineurin Inhibitors
  • Rituximab
  • Creatinine
  • Prednisone