Long-term successful liver-kidney transplantation in a child with atypical hemolytic uremic syndrome caused by homozygous factor H deficiency

Author(s): Emmanuel Gonzales[sup.1] , Tim Ulinski[sup.2] , Dalila Habes[sup.1] , Georges Deschênes[sup.3] , Veronique Fremeaux-Bacchi[sup.4] , Albert Bensman[sup.3] Author Affiliations: (1) Pediatric Hepatology, AP-HP Hôpital Bicêtre, 78 avenue du General [...]
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.sup.-1 1.73 m.sup.-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.