Single Dose of Rituximab for Refractory Steroid-Dependent Nephrotic Syndrome in Children

Pediatr Nephrol. 2009 Jul;24(7):1321-8. doi: 10.1007/s00467-009-1191-0. Epub 2009 May 7.

Abstract

We conducted a multicenter prospective trial to evaluate the efficacy, safety and pharmacokinetics of a single dose of rituximab (375 mg/m(2) body surface area) for the treatment of children with refractory steroid-dependent nephrotic syndrome (SDNS). All patients (n = 12) were able to discontinue steroids at a median of 74 days after treatment. The frequency of relapses per 6 months was significantly reduced and the steroid-free period per 6 months was significantly increased after treatment compared with those before treatment. The condition in nine of the patients (75%) relapsed at a median of 129 days after treatment, and seven patients were given additional rituximab due to steroid dependency. Most of the relapses developed simultaneously with recovery of B-cells. However, three patients (25%) did not have a relapse with B-cell recovery and the disease was kept in remission for more than 1 year. None of the patients developed life-threatening adverse events. This is the first report of a prospective study of a single dose of rituximab for refractory SDNS. Treatment with a single dose of rituximab may be effective for refractory SDNS, but its efficacy to prevent relapses was transient in most of the patients.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antibodies, Monoclonal / administration & dosage*
  • Antibodies, Monoclonal / blood
  • Antibodies, Monoclonal / pharmacokinetics
  • Antibodies, Monoclonal, Murine-Derived
  • Area Under Curve
  • B-Lymphocytes / drug effects
  • B-Lymphocytes / immunology
  • Child
  • Child, Preschool
  • Enzyme-Linked Immunosorbent Assay
  • Female
  • Flow Cytometry
  • Half-Life
  • Humans
  • Immunoglobulin A / blood
  • Immunoglobulin A / drug effects
  • Immunoglobulin G / blood
  • Immunoglobulin G / drug effects
  • Immunoglobulin M / blood
  • Immunoglobulin M / drug effects
  • Immunosuppressive Agents / administration & dosage*
  • Immunosuppressive Agents / blood
  • Immunosuppressive Agents / pharmacokinetics
  • Infant
  • Kaplan-Meier Estimate
  • Male
  • Nephrotic Syndrome / drug therapy*
  • Nephrotic Syndrome / immunology
  • Rituximab
  • Steroids / therapeutic use

Substances

  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Murine-Derived
  • Immunoglobulin A
  • Immunoglobulin G
  • Immunoglobulin M
  • Immunosuppressive Agents
  • Steroids
  • Rituximab