title: Long-term successful liver-kidney transplantation in a child with atypical hemolytic uremic syndrome caused by homozygous factor H deficiency.
abstract: Rational options for the treatment of end-stage renal disease (ESRD) due to atypical hemolytic uremic syndrome (aHUS) in children are still open to discussion. In the case of human complement factor H (CFH) deficiency, the choice is either kidney transplantation in combination with eculizumab, a humanized anti-C5 monoclonal antibody, or a combined liver-kidney transplantation.
  
  
  
  A child with a homozygous CFH deficiency underwent a successful liver-kidney transplantation. CFH levels normalized within days. After 6 years of follow-up, the graft function (Cockroft clearance 100 ml min-11.73 m-2) and the liver functions were normal.
  
  
  
  The results of this long-term follow-up confirm that combined liver-kidney transplantation remains a reasonable option in patients with ESRD due to aHUS when an identified genetic abnormality of the C3 convertase regulator synthesized in the liver has been identified.
authors.affiliation: Pediatric Hepatology, AP-HP Hôpital Bicêtre, 78 avenue du Général Leclerc, 94270, Le Kremlin-Bicêtre, France.
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Gonzales
authors.affiliation: Pediatric Nephrology, AP-HP Hôpital Armand-Trousseau, 26 avenue du Dr Arnold-Netter, 75012, Paris, France.
authors.forename: Tim
authors.initials: T
authors.lastname: Ulinski
authors.affiliation: Pediatric Hepatology, AP-HP Hôpital Bicêtre, 78 avenue du Général Leclerc, 94270, Le Kremlin-Bicêtre, France.
authors.forename: Dalila
authors.initials: D
authors.lastname: Habes
authors.affiliation: Pediatric Nephrology, AP-HP Hôpital Robert-Debré, 48 Bd Sérurier, 75019, Paris, France.
authors.forename: Georges
authors.initials: G
authors.lastname: Deschênes
authors.affiliation: Laboratory of Immunology, 20 rue Leblanc, 75015, Paris, France.
authors.forename: Véronique
authors.initials: V
authors.lastname: Frémeaux-Bacchi
authors.affiliation: Pediatric Nephrology, AP-HP Hôpital Robert-Debré, 48 Bd Sérurier, 75019, Paris, France. albert.bensman@orange.fr.
authors.forename: Albert
authors.identifier: 0000-0001-9083-7469
authors.initials: A
authors.lastname: Bensman
mesh_terms: D065766:Atypical Hemolytic Uremic Syndrome / Q000209:etiology / Q000235:genetics* / Q000601:surgery*; D017242:Complement Factor H / Q000172:deficiency* / Q000235:genetics; D003241:Consanguinity; D000081208:Hereditary Complement Deficiency Diseases; D006801:Humans; D007223:Infant; D007674:Kidney Diseases / Q000150:complications / Q000235:genetics*; D007676:Kidney Failure, Chronic / Q000209:etiology / Q000601:surgery; D016030:Kidney Transplantation / Q000379:methods*; D016031:Liver Transplantation / Q000379:methods*; D008297:Male; D010951:Plasma Exchange; D016896:Treatment Outcome; D015432:Glomerulonephritis, Membranoproliferative
chemical_list: C493495:CFH protein, human; D017242:Complement Factor H
keywords: Atypical hemolytic uremic syndrome; Combined liver–kidney transplantation; Complement factor H deficiency; End-stage renal disease