Emmanuel Gonzales1, Tim Ulinski2, Dalila Habes1, Georges Deschênes3, Véronique Frémeaux-Bacchi4, Albert Bensman5. 1. Pediatric Hepatology, AP-HP Hôpital Bicêtre, 78 avenue du Général Leclerc, 94270, Le Kremlin-Bicêtre, France. 2. Pediatric Nephrology, AP-HP Hôpital Armand-Trousseau, 26 avenue du Dr Arnold-Netter, 75012, Paris, France. 3. Pediatric Nephrology, AP-HP Hôpital Robert-Debré, 48 Bd Sérurier, 75019, Paris, France. 4. Laboratory of Immunology, 20 rue Leblanc, 75015, Paris, France. 5. Pediatric Nephrology, AP-HP Hôpital Robert-Debré, 48 Bd Sérurier, 75019, Paris, France. albert.bensman@orange.fr.
Abstract
BACKGROUND: 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. CASE-DIAGNOSIS/TREATMENT: 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-1 1.73 m-2) and the liver functions were normal. RESULTS AND CONCLUSIONS: 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.
BACKGROUND: 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 humancomplement 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. CASE-DIAGNOSIS/TREATMENT: 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-1 1.73 m-2) and the liver functions were normal. RESULTS AND CONCLUSIONS: 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.
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