Atypical hemolytic uremic syndrome

Semin Nephrol. 2013 Nov;33(6):508-30. doi: 10.1016/j.semnephrol.2013.08.003.


Hemolytic uremic syndrome (HUS) is a triad of microangiopathic hemolytic anemia, thrombocytopenia, and acute renal failure. The atypical form of HUS is a disease characterized by complement overactivation. Inherited defects in complement genes and acquired autoantibodies against complement regulatory proteins have been described. Incomplete penetrance of mutations in all predisposing genes is reported, suggesting that a precipitating event or trigger is required to unmask the complement regulatory deficiency. The underlying genetic defect predicts the prognosis both in native kidneys and after renal transplantation. The successful trials of the complement inhibitor eculizumab in the treatment of atypical HUS will revolutionize disease management.

Keywords: Complement; eculizumab; factor H; factor I; hemolytic uremic syndrome; membrane cofactor protein; thrombomodulin; transplantation.

Publication types

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

MeSH terms

  • Antibodies, Monoclonal, Humanized / therapeutic use
  • Atypical Hemolytic Uremic Syndrome
  • Autoantibodies
  • Complement Activation / physiology*
  • Complement Factor H / genetics
  • Complement Factor H / immunology
  • Complement System Proteins / genetics*
  • Complement System Proteins / immunology
  • Complement System Proteins / metabolism
  • Hemolytic-Uremic Syndrome* / etiology
  • Hemolytic-Uremic Syndrome* / genetics
  • Hemolytic-Uremic Syndrome* / physiopathology
  • Humans
  • Kidney Transplantation / adverse effects
  • Kidney Transplantation / mortality
  • Precipitating Factors


  • Antibodies, Monoclonal, Humanized
  • Autoantibodies
  • Complement Factor H
  • Complement System Proteins
  • eculizumab