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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.
Author(s): Gilbert RD, Fowler DJ, Angus E, Hardy SA, Stanley L, Goodship TH
Publication type: Article
Publication status: Published
Journal: Pediatric Nephrology
Print publication date: 28/04/2013
ISSN (print): 0931-041X
ISSN (electronic): 1432-198X
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