Eculizumab as first-line therapy for atypical hemolytic uremic syndrome

Pediatrics. 2014 Jun;133(6):e1759-63. doi: 10.1542/peds.2013-1787.


Atypical hemolytic uremic syndrome (aHUS) is a genetic, life-threatening, chronic disease that can affect patients of all ages. aHUS is caused by uncontrolled complement activation due to genetic defects of complement regulation. Plasma exchange or infusion has been used to manage aHUS and may transiently maintain hematologic variables in some patients, but as the underlying complement dysregulation persists, end-stage renal disease or death occurs in 33% to 40% of patients during the first clinical manifestation. Here we present a pediatric case showing that first-line eculizumab treatment successfully blocked the progression of thrombotic microangiopathy in aHUS.

Keywords: atypical hemolytic uremic syndrome; complement; eculizumab; infant.

Publication types

  • Case Reports

MeSH terms

  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Child, Preschool
  • Combined Modality Therapy
  • Female
  • Hemolytic-Uremic Syndrome / diagnosis
  • Hemolytic-Uremic Syndrome / drug therapy*
  • Hemolytic-Uremic Syndrome / immunology*
  • Humans
  • Male
  • Renal Dialysis


  • Antibodies, Monoclonal, Humanized
  • eculizumab