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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

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

Erschienen in: Pediatric Nephrology | Ausgabe 8/2013

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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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Titel: Eculizumab therapy for atypical haemolytic uraemic syndrome due to a gain-of-function mutation of complement factor B
verfasst von: Rodney D. Gilbert
Darren J. Fowler
Elizabeth Angus
Stephen A. Hardy
Louise Stanley
Timothy H. Goodship
Publikationsdatum: 01.08.2013
Verlag: Springer Berlin Heidelberg
Erschienen in: Pediatric Nephrology / Ausgabe 8/2013
Print ISSN: 0931-041X
Elektronische ISSN: 1432-198X
DOI: https://doi.org/10.1007/s00467-013-2492-x

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Springer Medizin

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

Abstract

Background

Case-Diagnosis/Treatment

Conclusions

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Springer Medizin

Abstract

Background

Case-Diagnosis/Treatment

Conclusions

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