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Atypical hemolytic uremic syndrome

Lookup NU author(s): Professor David KavanaghORCiD, Professor Tim Goodship

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Abstract

Purpose of review The last few years revealed a molecular distinction between thrombotic thrombocytopenic purpura, a disease characterized by a lack of ADAMTS13 activity, and atypical hemolytic uremic syndrome (aHUS), a disease of complement overactivation. Many different predisposing genetic factors resulting in complement overactivation have been described in aHUS. Additionally, autoantibodies against complement regulatory proteins have been reported. Recent findings The last year has seen the description of a new risk factor for aHUS in the form of mutations in thrombomodulin. As with other genetic risk factors seen in aHUS, these mutations result in impaired regulation of complement. It is increasingly recognized that a confluence of risk factors resulting in complement overactivation may be required for the disease to manifest. In the last year the complement inhibitor eculizumab has been used successfully to treat patients with aHUS. Summary The characterization of the molecular defect in aHUS has allowed targeted therapy to be used. Although early reports of the efficacy of the complement inhibitor eculizumab are promising, the outcome of a recent clinical trial is awaited.

Publication metadata

Author(s): Kavanagh D, Goodship THJ

Publication type: Article

Publication status: Published

Journal: Current Opinion in Hematology

Year: 2010

Volume: 17

Issue: 5

Pages: 432-438

Print publication date: 01/09/2010

ISSN (print): 1065-6251

ISSN (electronic): 1531-7048

Publisher: Lippincott Williams & Wilkins

URL: http://dx.doi.org/10.1097/MOH.0b013e32833cae86

DOI: 10.1097/MOH.0b013e32833cae86

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