Renal transplantation under prophylactic eculizumab in atypical hemolytic uremic syndrome with CFH/CFHR1 hybrid protein

Am J Transplant. 2012 Jul;12(7):1938-44. doi: 10.1111/j.1600-6143.2012.04051.x. Epub 2012 Apr 11.

Abstract

We report the first observation of successful kidney transplantation under pre-emptive eculizumab treatment in a 7-year-old boy with atypical hemolytic uremic syndrome (aHUS) and a known hybrid CFH/CFHR1 gene, who was dependent on plasma therapy during the 3-year dialysis period. The hybrid CFH/CFHR1 protein has an altered C3b/C3d binding, is incapable to protect cells from complement attack and is directly implicated in aHUS pathogenesis. There was no evidence of recurrence during the first 16-month follow-up period. We conclude that eculizumab alone, without plasma therapy (plasma infusion and/or plasma exchange), is sufficient to prevent recurrence of aHUS and to maintain long-term graft function.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antibodies, Monoclonal, Humanized / administration & dosage*
  • Child
  • Child, Preschool
  • Complement C3b Inactivator Proteins / genetics*
  • Complement Factor H / genetics
  • Hemolytic-Uremic Syndrome / genetics
  • Hemolytic-Uremic Syndrome / surgery*
  • Humans
  • Kidney Transplantation*
  • Male

Substances

  • Antibodies, Monoclonal, Humanized
  • CFH protein, human
  • CFHR1 protein, human
  • Complement C3b Inactivator Proteins
  • Complement Factor H
  • eculizumab