New treatment options for atypical hemolytic uremic syndrome with the complement inhibitor eculizumab

Semin Thromb Hemost. 2010 Sep;36(6):669-72. doi: 10.1055/s-0030-1262889. Epub 2010 Sep 23.

Abstract

Hemolytic uremic syndrome (HUS) is a disease characterized by microangiopathic hemolytic anemia, consumptive thrombocytopenia, and renal impairment. Often HUS is triggered by Shiga-like toxin- producing ESCHERICHIA COLI. Less common is atypical HUS (aHUS), which is caused by defective complement control. aHUS is associated with mutations in genes encoding complement regulatory proteins in ~50% of patients with this syndrome. Furthermore, autoantibodies that inactivate to factor H have also been linked to the disease. Initial triggers include infections, use of endothelial-affecting drugs, malignancies, transplantation, and pregnancy. Advances in our understanding of the pathogenesis of atypical HUS suggest that complement inhibition may be used as treatment for the disease. We discuss the potential benefit of the complement inhibitor eculizumab for the treatment of aHUS.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Antibodies, Monoclonal / immunology
  • Antibodies, Monoclonal / therapeutic use*
  • Antibodies, Monoclonal, Humanized
  • Complement C3b Inactivator Proteins / genetics
  • Complement C5 / immunology*
  • Complement Factor H / genetics
  • Complement Pathway, Alternative / drug effects
  • Complement Pathway, Alternative / immunology
  • Female
  • Gene Deletion
  • Hemolytic-Uremic Syndrome / drug therapy*
  • Hemolytic-Uremic Syndrome / genetics
  • Hemolytic-Uremic Syndrome / immunology
  • Humans
  • Mutation
  • Polymorphism, Genetic
  • Treatment Outcome
  • Young Adult

Substances

  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • CFHR1 protein, human
  • Complement C3b Inactivator Proteins
  • Complement C5
  • Complement Factor H
  • eculizumab