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Pediatric Nephrology

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01-08-2013 | Brief Report

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

Authors: Rodney D. Gilbert, Darren J. Fowler, Elizabeth Angus, Stephen A. Hardy, Louise Stanley, Timothy H. Goodship

Published in: Pediatric Nephrology | Issue 8/2013

Abstract

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.

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Title: Eculizumab therapy for atypical haemolytic uraemic syndrome due to a gain-of-function mutation of complement factor B
Authors: Rodney D. Gilbert
Darren J. Fowler
Elizabeth Angus
Stephen A. Hardy
Louise Stanley
Timothy H. Goodship
Publication date: 01-08-2013
Publisher: Springer Berlin Heidelberg
Published in: Pediatric Nephrology / Issue 8/2013
Print ISSN: 0931-041X
Electronic ISSN: 1432-198X
DOI: https://doi.org/10.1007/s00467-013-2492-x

At a glance: The ONWARDS insulin icodec trials

Springer Medicine

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

Abstract

Background

Case-Diagnosis/Treatment

Conclusions

At a glance: The ONWARDS insulin icodec trials

Springer Medicine

Abstract

Background

Case-Diagnosis/Treatment

Conclusions

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