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The global aHUS registry: methodology and initial patient characteristics

Licht, Christoph ; Ardissino, Gianluigi ; Ariceta, Gema ; Cohen, David ; Cole, J. Alexander ; Gasteyger, Christoph ; Greenbaum, Larry A. ; Johnson, Sally ; Ogawa, Masayo ; Schaefer, Franz ; Vande Walle, Johan ; Frémeaux-Bacchi, Véronique

In: BMC Nephrology, 16 (2015), Nr. 207. pp. 1-8. ISSN 1471-2369

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Download (470kB) | Lizenz: Creative Commons LizenzvertragThe global aHUS registry: methodology and initial patient characteristics by Licht, Christoph ; Ardissino, Gianluigi ; Ariceta, Gema ; Cohen, David ; Cole, J. Alexander ; Gasteyger, Christoph ; Greenbaum, Larry A. ; Johnson, Sally ; Ogawa, Masayo ; Schaefer, Franz ; Vande Walle, Johan ; Frémeaux-Bacchi, Véronique underlies the terms of Creative Commons Attribution 3.0 Germany

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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.

Item Type: Article
Journal or Publication Title: BMC Nephrology
Volume: 16
Number: 207
Publisher: BioMed Central
Place of Publication: London
Date Deposited: 09 Feb 2016 12:59
Date: 2015
ISSN: 1471-2369
Page Range: pp. 1-8
Faculties / Institutes: Medizinische Fakultät Heidelberg > Universitätskinderklinik
Subjects: 610 Medical sciences Medicine
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