Liver transplantation for aHUS: still needed in the eculizumab era?
Pediatr Nephrol
; 31(5): 759-68, 2016 May.
Article
em En
| MEDLINE
| ID: mdl-26604087
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.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Rim
/
Transplante de Fígado
/
Ativação do Complemento
/
Inativadores do Complemento
/
Anticorpos Monoclonais Humanizados
/
Síndrome Hemolítico-Urêmica Atípica
Tipo de estudo:
Diagnostic_studies
Limite:
Humans
/
Infant
/
Male
Idioma:
En
Revista:
Pediatr Nephrol
Assunto da revista:
NEFROLOGIA
/
PEDIATRIA
Ano de publicação:
2016
Tipo de documento:
Article
País de afiliação:
Itália