Eculizumab for Atypical Hemolytic–Uremic Syndrome

Abstract

Atypical hemolytic–uremic syndrome is a disease of uncontrolled complement activation associated with a high mortality rate, and most cases progress to end-stage renal disease.¹ About 50% of patients with this syndrome carry mutations in genes encoding complement proteins.² Complement inhibition has been suggested for the treatment of atypical hemolytic–uremic syndrome,³ but currently no data on this treatment option are available. We report on a case of atypical hemolytic–uremic syndrome that was successfully treated with eculizumab, a humanized monoclonal antibody that blocks complement activity by cleavage of the complement protein C5, thereby preventing the generation of the inflammatory peptide C5a and the cytotoxic membrane-attack complex, C5b-9. Eculizumab has been approved for the treatment of paroxysmal nocturnal hemoglobinuria.⁴