title: Complement factor H-antibody-associated hemolytic uremic syndrome: pathogenesis, clinical presentation, and treatment.
abstract: The presence of circulating autoantibodies, primarily to complement factor H antibodies (CFH-Abs) in plasma characterizes the autoimmune form of atypical hemolytic uremic syndrome (aHUS). This acquired form of aHUS defines a distinct subgroup of aHUS patients, which requires diagnostic and treatment approaches in part different from those of the genetically defined forms. The mechanisms leading to CFH-Ab production and disease onset are not completely understood, but CFH-Ab HUS seems to be secondary to a combination of genetic predisposition and environmental factors. Early diagnosis of this specific aHUS entity is important, as prompt induction of plasma exchange and concomitant immunosuppression leads to a favorable outcome. Nevertheless, information on clinical features and outcome in children is limited. Here, we review the literature on the biological and clinical features of CFH-Ab HUS and discuss therapeutic options.
authors.affiliation: Department of Pediatrics I, Innsbruck Medical University, Innsbruck, Austria.
authors.forename: Johannes
authors.initials: J
authors.lastname: Hofer
authors.affiliation: Department of Pediatrics I, Innsbruck Medical University, Innsbruck, Austria.
authors.forename: Thomas
authors.initials: T
authors.lastname: Giner
authors.affiliation: MTA-ELTE "Lendület" Complement Research Group, Department of Immunology, Eötvös Loránd University, Budapest, Hungary.
authors.forename: Mihály
authors.initials: M
authors.lastname: Józsi
mesh_terms: D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use; D065766:Atypical Hemolytic Uremic Syndrome / Q000175:diagnosis* / Q000276:immunology / Q000628:therapy*; D001323:Autoantibodies / Q000097:blood; D001665:Binding Sites; D002986:Clinical Trials as Topic; D003180:Complement C3b Inactivator Proteins / Q000276:immunology; D017242:Complement Factor H / Q000276:immunology*; D020022:Genetic Predisposition to Disease; D006801:Humans; D016030:Kidney Transplantation; D017434:Protein Structure, Tertiary; D016896:Treatment Outcome
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D001323:Autoantibodies; D003180:Complement C3b Inactivator Proteins; D017242:Complement Factor H; C481642:eculizumab