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Pediatric Nephrology

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Open Access 01-07-2012 | Brief Report

Eculizumab as rescue therapy for atypical hemolytic uremic syndrome with normal platelet count

Authors: Eiske M. Dorresteijn, Nicole C. A. J. van de Kar, Karlien Cransberg

Published in: Pediatric Nephrology | Issue 7/2012

Abstract

Background

Atypical hemolytic uremic syndrome (aHUS) in childhood is a rare disease with frequent progression to end-stage renal disease and a high recurrence after kidney transplantation. Eculizumab, a humanized monoclonal antibody that binds to complement protein C5, may be beneficial in the treatment of aHUS.

Case-diagnosis/treatment

A 6-year-old girl developed aHUS with only slightly elevated C3d (4.4%), no mutations in complement factors, and no antibodies against factor H. Plasma exchange treatment was successful initially, until aHUS recurred. After reinitiating plasma exchange, normalization of the platelet count and improvement of hemolysis occurred, but renal function worsened. Renal function then improved dramatically promptly after the switch to eculizumab.

Conclusions

This case demonstrates that platelet count is not always a reliable marker for improvement of aHUS and that eculizumab can prevent dialysis in plasma-resistant aHUS patients.

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Al-Akash SI, Almond PS, Savell VH Jr, Gharaybeh SI, Hogue C (2011) Eculizumab induces long-term remission in recurrent post-transplant HUS associated with C3 gene mutation. Pediatr Nephrol 26:613–619PubMedCrossRef

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Zimmerhackl LB, Hofer J, Cortina G, Mark W, Wurzner R, Jungraithmayr TC, Khursigara G, Kliche KO, Radauer W (2010) Prophylactic eculizumab after renal transplantation in atypical hemolytic-uremic syndrome. N Engl J Med 362:1746–1748PubMedCrossRef

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Greenbaum L, Babu S, Furman R, Sheerin N, Cohen D, Gaber O, Eitner F, Delmas Y, Loirat C, Bedrosian C, Legendre C (2011) Continued improvements in renal function with sustained eculizumab (ECU) in patients (PTS) with atypical hemolytic uremic syndrome (aHUS) resistant to plasma exchange/infusion (PE/PI). J Am Soc Nephrol 22:197A, TH-PO367CrossRef

Title: Eculizumab as rescue therapy for atypical hemolytic uremic syndrome with normal platelet count
Authors: Eiske M. Dorresteijn
Nicole C. A. J. van de Kar
Karlien Cransberg
Publication date: 01-07-2012
Publisher: Springer-Verlag
Published in: Pediatric Nephrology / Issue 7/2012
Print ISSN: 0931-041X
Electronic ISSN: 1432-198X
DOI: https://doi.org/10.1007/s00467-012-2130-z

At a glance: The ONWARDS insulin icodec trials

Springer Medicine

Eculizumab as rescue therapy for atypical hemolytic uremic syndrome with normal platelet count

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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