title: Atypical HUS: time to take stock of current guidelines and outcome measures?
abstract: European guidelines for the assessment and management of atypical HUS were written in 2009. Since then our understanding of this group of diseases has advanced. Evidence is emerging that eculizumab, a monoclonal antibody inhibiting C5 activation, is effective, and potentially superior to current treatment with plasmapheresis. The evidence base for the benefits of plasmapheresis consists of case series and small reports. Before we embark on a change of management policy it is vital that we set up a system for genetic diagnosis, standardised protocols and a means to collect predetermined outcome measures, so that we do not make the same mistakes with assessment of the effectiveness of eculizumab as we did for plasmapheresis.
authors: Lesley
authors: L
authors: Rees
mesh_terms: D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D065766:Atypical Hemolytic Uremic Syndrome; D002985:Clinical Protocols; D003167:Complement Activation / Q000187:drug effects; D020022:Genetic Predisposition to Disease; D006463:Hemolytic-Uremic Syndrome / Q000175:diagnosis / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D006801:Humans; D009154:Mutation; D010641:Phenotype; D010956:Plasmapheresis; D017410:Practice Guidelines as Topic; D012307:Risk Factors; D016896:Treatment Outcome
chemical_list: D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab
references: Orphanet J Rare Dis. 2011 Sep 08;6:60
references: 21902819
references: Nephrol Dial Transplant. 2012 Jul;27(7):2673-85
references: 22802583
references: Pediatr Nephrol. 2009 Apr;24(4):687-96
references: 18800230
references: Semin Dial. 2012 Mar-Apr;25(2):214-9
references: 22309967
references: Clin Dev Immunol. 2012;2012:370426
references: 23251215
references: Br J Haematol. 2010 Jan;148(1):37-47
references: 19821824
references: Nat Rev Nephrol. 2012 Nov;8(11):643-57
references: 23026949
references: Pediatr Nephrol. 2013 May;28(5):827-30
references: 23389237