title: The global aHUS registry: methodology and initial patient characteristics.
abstract: BACKGROUND
  Atypical hemolytic uremic syndrome (aHUS) is a rare, genetically-mediated systemic disease most often caused by chronic, uncontrolled complement activation that leads to systemic thrombotic microangiopathy (TMA) and renal and other end-organ damage.
  
  
  METHODS
  The global aHUS Registry, initiated in April 2012, is an observational, noninterventional, multicenter registry designed to collect demographic characteristics, medical and disease history, treatment effectiveness and safety outcomes data for aHUS patients. The global aHUS Registry will operate for a minimum of 5 years of follow-up. Enrollment is open to all patients with a clinical diagnosis of aHUS, with no requirement for identified complement gene mutations, polymorphisms or autoantibodies or particular type of therapy/management.
  
  
  RESULTS
  As of September 30, 2014, 516 patients from 16 countries were enrolled. At enrollment, 315 (61.0 %) were adults (≥18 years) and 201 (39.0 %) were <18 years of age. Mean (standard deviation [SD]) age at diagnosis was 22.7 (20.5) years. Nineteen percent of patients had a family history of aHUS, 60.3 % had received plasma exchange/plasma infusion, 59.5 % had a history of dialysis, and 19.6 % had received ≥1 kidney transplant. Overall, 305 patients (59.1 %) have received eculizumab.
  
  
  CONCLUSIONS
  As enrollment and follow-up proceed, the global aHUS Registry is expected to yield valuable baseline, natural history, medical outcomes, treatment effectiveness and safety data from a diverse population of patients with aHUS.
  
  
  BACKGROUND
  US National Institutes of Health www.ClinicalTrials.gov Identifier NCT01522183 . Registered January 18, 2012.
authors.affiliation: Division of Nephrology and Program in Cell Biology, The Hospital for Sick Children, 555 University Avenue, Toronto, ON, M5G 1X8, Canada. christoph.licht@sickkids.ca.
authors.forename: Christoph
authors.initials: C
authors.lastname: Licht
authors.affiliation: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via della Comenda 9, Milan, 20122, Italy. ardissino@italkid.org.
authors.forename: Gianluigi
authors.initials: G
authors.lastname: Ardissino
authors.affiliation: Pediatric Nephrology, University Hospital Vall d'Hebron, Pg Vall d' Hebron, 119-129, Barcelona, Spain. gariceta@vhebron.net.
authors.forename: Gema
authors.initials: G
authors.lastname: Ariceta
authors.affiliation: Columbia University Medical Center, 622 West 168 Street, Room PH4-124, New York, NY, USA. djc5@cumc.columbia.edu.
authors.forename: David
authors.initials: D
authors.lastname: Cohen
authors.affiliation: Alexion Pharmaceuticals, Inc., 352 Knotter Drive, Cheshire, CT, USA. ColeA@alxn.com.
authors.forename: J Alexander
authors.initials: JA
authors.lastname: Cole
authors.affiliation: Alexion Pharma International, Avenue du Tribunal Fédéral 34, Lausanne, Switzerland. gasteygerc@alxn.com.
authors.forename: Christoph
authors.initials: C
authors.lastname: Gasteyger
authors.affiliation: Emory University and Children's Healthcare of Atlanta, 2015 Uppergate Drive, Atlanta, GA, USA. lgreen6@emory.edu.
authors.forename: Larry A
authors.initials: LA
authors.lastname: Greenbaum
authors.affiliation: Great North Children's Hospital, Sir James Spence Institute, 4th floor, Royal Victoria Infirmary, Newcastle, United Kingdom. sally.johnson@nuth.nhs.uk.
authors.forename: Sally
authors.initials: S
authors.lastname: Johnson
authors.affiliation: Alexion Pharmaceuticals, Inc., 352 Knotter Drive, Cheshire, CT, USA. OgawaM@alxn.com.
authors.forename: Masayo
authors.initials: M
authors.lastname: Ogawa
authors.affiliation: Heidelberg University Medical Center, Im Neuenheimer Feld 672, 69120, Heidelberg, Germany. franz.schaefer@med.uni-heidelberg.de.
authors.forename: Franz
authors.initials: F
authors.lastname: Schaefer
authors.affiliation: Ghent University Hospital, De Pintelaan 185, Ghent, Belgium. Johan.VandeWalle@uzgent.be.
authors.forename: Johan
authors.initials: J
authors.lastname: Vande Walle
authors.affiliation: Assistance Publique-Hôpitaux de Paris, Hôpital Européen Georges Pompidou, 20 rue Leblanc, Paris, France. veronique.fremeaux-bacchi@egp.aphp.fr.
authors.forename: Véronique
authors.initials: V
authors.lastname: Frémeaux-Bacchi
mesh_terms: D000293:Adolescent; D000328:Adult; D065766:Atypical Hemolytic Uremic Syndrome / Q000175:diagnosis / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D012042:Registries