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primary topic: The global aHUS registry: methodology and initial patient characteristics

The global aHUS registry: methodology and initial patient characteristics
type: AcademicArticle
type: Article
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. Trial registration: US National Institutes of Health www.ClinicalTrials.gov Identifier NCT01522183. Registered January 18, 2012.
date: 2015
volume: 16
issue: 207
is part of: BMC Nephrology
author list: authors
type: EPrint
type: ArticleEPrint
is part of: repository
has document: The global aHUS registry: methodology and initial patient characteristics (PDF)
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subject: 610 Medizin

The global aHUS registry: methodology and initial patient characteristics (PDF)
type: Document
has file: 12882_2015_Article_195.pdf
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BioMed Central
type: Organization

BMC Nephrology
type: Collection
issn: 14712369

Christoph Licht
type: Person
given name: Christoph
family name: Licht

Christoph Gasteyger
type: Person
given name: Christoph
family name: Gasteyger

David Cohen
type: Person
given name: David
family name: Cohen

Masayo Ogawa
type: Person
given name: Masayo
family name: Ogawa

Larry A. Greenbaum
type: Person
given name: Larry A.
family name: Greenbaum

Gianluigi Ardissino
type: Person
given name: Gianluigi
family name: Ardissino

Sally Johnson
type: Person
given name: Sally
family name: Johnson

Véronique Frémeaux-Bacchi
type: Person
given name: Véronique
family name: Frémeaux-Bacchi

J. Alexander Cole
type: Person
given name: J. Alexander
family name: Cole

Franz Schaefer
type: Person
given name: Franz
family name: Schaefer

Gema Ariceta
type: Person
given name: Gema
family name: Ariceta

Johan Vande Walle
type: Person
given name: Johan
family name: Vande Walle

The global aHUS registry: methodology and initial patient characteristics (Other)
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primary topic: The global aHUS registry: methodology and initial patient characteristics

610 Medizin
type: Concept
preferred label: 610 Medical sciences Medicine @en