Eculizumab for Atypical Hemolytic–Uremic Syndrome

Authors:
  • JNJens Nürnberger
    Jens Nürnberger
  • TPThomas Philipp
    Thomas Philipp
  • OWOliver Witzke
    Oliver Witzke
  • ASAnabelle Opazo Saez
    Anabelle Opazo Saez
  • UVUdo Vester
    Udo Vester
  • HBHideo Andreas Baba
    Hideo Andreas Baba
  • AKAndreas Kribben
    Andreas Kribben
  • LZLothar Bernd Zimmerhackl
    Lothar Bernd Zimmerhackl
  • MNMato Nagel
    Mato Nagel
  • MKMichael Kirschfink
    Michael Kirschfink
Publisher website
Google Scholar
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