title: Renal transplantation under prophylactic eculizumab in atypical hemolytic uremic syndrome with CFH/CFHR1 hybrid protein.
abstract: We report the first observation of successful kidney transplantation under pre-emptive eculizumab treatment in a 7-year-old boy with atypical hemolytic uremic syndrome (aHUS) and a known hybrid CFH/CFHR1 gene, who was dependent on plasma therapy during the 3-year dialysis period. The hybrid CFH/CFHR1 protein has an altered C3b/C3d binding, is incapable to protect cells from complement attack and is directly implicated in aHUS pathogenesis. There was no evidence of recurrence during the first 16-month follow-up period. We conclude that eculizumab alone, without plasma therapy (plasma infusion and/or plasma exchange), is sufficient to prevent recurrence of aHUS and to maintain long-term graft function.
authors.affiliation: Pediatric Nephrology, Hôpital Necker-Enfants Malades, Paris, France.
authors.forename: S
authors.initials: S
authors.lastname: Krid
authors.forename: L T
authors.initials: LT
authors.lastname: Roumenina
authors.forename: D
authors.initials: D
authors.lastname: Beury
authors.forename: M
authors.initials: M
authors.lastname: Charbit
authors.forename: O
authors.initials: O
authors.lastname: Boyer
authors.forename: V
authors.initials: V
authors.lastname: Frémeaux-Bacchi
authors.forename: P
authors.initials: P
authors.lastname: Niaudet
mesh_terms: D061067:Antibodies, Monoclonal, Humanized / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D003180:Complement C3b Inactivator Proteins / Q000235:genetics*; D017242:Complement Factor H / Q000235:genetics; D006463:Hemolytic-Uremic Syndrome / Q000235:genetics / Q000601:surgery*; D006801:Humans; D016030:Kidney Transplantation; D008297:Male
chemical_list: D061067:Antibodies, Monoclonal, Humanized; C493495:CFH protein, human; C517621:CFHR1 protein, human; D003180:Complement C3b Inactivator Proteins; D017242:Complement Factor H; C481642:eculizumab