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Eculizumab therapy for atypical haemolytic uraemic syndrome due to a gain-of-function mutation of complement factor B

Lookup NU author(s): Dr Steven Hardy, Professor Tim Goodship


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Background Atypical haemolytic uraemic syndrome (aHUS) is caused by dysregulated complement activation. A humanised anti-C5 monoclonal antibody has recently become available for treatment of this condition Case-Diagnosis/Treatment We present the first description of an infant with an activating mutation of complement factor B successfully treated with eculizumab. On standard doses she had evidence of ongoing C5 cleavage despite a good clinical response. Conclusions Eculizumab is effective therapy for aHUS associated with factor B mutations, but recommended doses may not be adequate for all patients.

Publication metadata

Author(s): Gilbert RD, Fowler DJ, Angus E, Hardy SA, Stanley L, Goodship TH

Publication type: Article

Publication status: Published

Journal: Pediatric Nephrology

Year: 2013

Volume: 28

Issue: 8

Pages: 1315-1318

Print publication date: 28/04/2013

ISSN (print): 0931-041X

ISSN (electronic): 1432-198X

Publisher: Springer


DOI: 10.1007/s00467-013-2492-x


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