Complement therapy in atypical haemolytic uraemic syndrome (aHUS)

Mol Immunol. 2013 Dec 15;56(3):199-212. doi: 10.1016/j.molimm.2013.05.224. Epub 2013 Jun 28.

Abstract

Central to the pathogenesis of atypical haemolytic uraemic syndrome (aHUS) is over-activation of the alternative pathway of complement. Inherited defects in complement genes and autoantibodies against complement regulatory proteins have been described. The use of plasma exchange to replace non-functioning complement regulators and hyper-functional complement components in addition to the removal of CFH-autoantibodies made this the 'gold-standard' for management of aHUS. In the last 4 years the introduction of the complement inhibitor Eculizumab has revolutionised the management of aHUS. In this review we shall discuss the available literature on treatment strategies to date.

Keywords: Complement; Eculizumab; Haemolytic Uraemic Syndrome; Treatment.

Publication types

  • Review

MeSH terms

  • Antibodies, Monoclonal / adverse effects
  • Antibodies, Monoclonal / therapeutic use*
  • Antibodies, Monoclonal, Humanized / adverse effects
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Atypical Hemolytic Uremic Syndrome
  • Complement System Proteins / immunology
  • Diacylglycerol Kinase / genetics
  • Hemolytic-Uremic Syndrome / diagnosis
  • Hemolytic-Uremic Syndrome / genetics
  • Hemolytic-Uremic Syndrome / immunology
  • Hemolytic-Uremic Syndrome / therapy*
  • Humans
  • Penetrance
  • Plasma Exchange*
  • Prognosis

Substances

  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Complement System Proteins
  • eculizumab
  • Diacylglycerol Kinase
  • diacylglycerol kinase epsilon, human