Semin Thromb Hemost 2010; 36(6): 669-672
DOI: 10.1055/s-0030-1262889
© Thieme Medical Publishers

New Treatment Options for Atypical Hemolytic Uremic Syndrome with the Complement Inhibitor Eculizumab

Özlem Köse1 , Lothar-Bernd Zimmerhackl2 , Therese Jungraithmayr2 , Christoph Mache3 , Jens Nürnberger1
  • 1Department of Nephrology, University Hospital of Essen, University Duisburg-Essen, Essen, Germany
  • 2Department of Pediatrics, Medical University Innsbruck, Innsbruck, Austria
  • 3Department of Pediatrics, Medical University Graz, Graz, Austria
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Publikationsverlauf

Publikationsdatum:
23. September 2010 (online)

ABSTRACT

Hemolytic uremic syndrome (HUS) is a disease characterized by microangiopathic hemolytic anemia, consumptive thrombocytopenia, and renal impairment. Often HUS is triggered by Shiga-like toxin- producing Escherichia coli. Less common is atypical HUS (aHUS), which is caused by defective complement control. aHUS is associated with mutations in genes encoding complement regulatory proteins in ~50% of patients with this syndrome. Furthermore, autoantibodies that inactivate to factor H have also been linked to the disease. Initial triggers include infections, use of endothelial-affecting drugs, malignancies, transplantation, and pregnancy. Advances in our understanding of the pathogenesis of atypical HUS suggest that complement inhibition may be used as treatment for the disease. We discuss the potential benefit of the complement inhibitor eculizumab for the treatment of aHUS.

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PD Dr. med. J. Nürnberger

Department of Nephrology, University Hospital of Essen

University Duisburg-Essen, Hufelandstrasse 55, 45122 Essen, Germany

eMail: jens.nuernberger@uni-due.de