title: Changing strategies for organ transplantation in atypical haemolytic uraemic syndrome: a tertiary case series.
abstract: We present three cases of organ transplantation for atypical haemolytic uraemic syndrome secondary to complement factor H mutation: one isolated renal transplant; one previously reported isolated liver transplant; and one combined liver and kidney transplant. All three patients were treated prior to the licensing of eculizumab for this condition, and all have had favourable outcomes with maintenance of graft function for years following transplantation. We discuss the evolution of transplantation therapy for aHUS over the last two decades. Transplantation decision-making in aHUS has evolved over this time with expanding knowledge of pathophysiology and genetics, alongside refined plasma exchange and anticoagulation protocols and improved centre experience. Our cases demonstrate how individual patient factors within this heterogeneous condition also underlie transplantation decisions and outcomes. Whilst our cases demonstrate that transplantation in aHUS can be a successful long-term treatment providing good quality of life, worldwide experience has proven that most curative treatment for aHUS strategies represents significant risks. Whether new pharmacotherapies such as eculizumab will alter this risk is yet to be determined.
authors: Department of Nephrology, Birmingham Children's Hospital NHS Foundation Trust, Birmingham, UK.
authors: Thomas A
authors: TA
authors: Forbes
authors: Mark G
authors: MG
authors: Bradbury
authors: Tim H J
authors: TH
authors: Goodship
authors: Patrick J
authors: PJ
authors: McKiernan
authors: David V
authors: DV
authors: Milford
mesh_terms: D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use; D000925:Anticoagulants / Q000627:therapeutic use; D065766:Atypical Hemolytic Uremic Syndrome; D002648:Child; D002675:Child, Preschool; D017242:Complement Factor H / Q000378:metabolism; D005260:Female; D006463:Hemolytic-Uremic Syndrome / Q000628:therapy*; D006801:Humans; D016030:Kidney Transplantation / Q000379:methods*; D016031:Liver Transplantation / Q000379:methods*; D008297:Male; D010951:Plasma Exchange; D011788:Quality of Life; D051437:Renal Insufficiency / Q000628:therapy; D012306:Risk; D016896:Treatment Outcome
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D000925:Anticoagulants; D017242:Complement Factor H; C481642:eculizumab