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24.11.2015 | Original Article

Liver transplantation for aHUS: still needed in the eculizumab era?

verfasst von: Rosanna Coppo, Roberto Bonaudo, R. Licia Peruzzi, Alessandro Amore, Andrea Brunati, Renato Romagnoli, Mauro Salizzoni, Miriam Galbusera, Eliana Gotti, Erica Daina, Marina Noris, Giuseppe Remuzzi

Erschienen in: Pediatric Nephrology | Ausgabe 5/2016

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Abstract

Background

The risk of disease recurrence after a kidney transplant is high in patients with atypical hemolytic uremic syndrome (aHUS) and mutations in the complement factor H (FH) gene (CFH). Since FH is mostly produced by the liver, a kidney transplant does not correct the genetic defect. The anti-C5 antibody eculizumab prevents post-transplant aHUS recurrence, but it does not cure the disease. Combined liver–kidney transplantation has been performed in few patients with CFH mutations based on the rationale that liver replacement provides a source of normal FH.

Methods

We report the 9-year follow-up of a child with aHUS and a CFH mutation, including clinical data, extensive genetic characterization, and complement profile in the circulation and at endothelial level. The outcome of kidney and liver transplants performed separately 3 years apart are reported.

Results

The patient showed incomplete response to plasma, with relapsing episodes, progression to end-stage renal disease, and endothelial-restricted complement dysregulation. Eculizumab prophylaxis post-kidney transplant did not achieve sustained remission, leaving the child at risk of disease recurrence. A liver graft given 3 years after the kidney transplant completely abrogated endothelial complement activation and allowed eculizumab withdrawal.

Conclusions

Liver transplant may definitely cure aHUS and represents an option for patients with suboptimal response to eculizumab.

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Titel: Liver transplantation for aHUS: still needed in the eculizumab era?
verfasst von: Rosanna Coppo
Roberto Bonaudo
R. Licia Peruzzi
Alessandro Amore
Andrea Brunati
Renato Romagnoli
Mauro Salizzoni
Miriam Galbusera
Eliana Gotti
Erica Daina
Marina Noris
Giuseppe Remuzzi
Publikationsdatum: 24.11.2015
Verlag: Springer Berlin Heidelberg
Erschienen in: Pediatric Nephrology / Ausgabe 5/2016
Print ISSN: 0931-041X
Elektronische ISSN: 1432-198X
DOI: https://doi.org/10.1007/s00467-015-3278-0

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Liver transplantation for aHUS: still needed in the eculizumab era?