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The global aHUS registry : methodology and initial patient characteristics

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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.
Keywords
Complement, Hemolytic uremic syndrome, Renal insufficiency, HEMOLYTIC-UREMIC SYNDROME, THROMBOTIC THROMBOCYTOPENIC PURPURA, COMPLEMENT, ECULIZUMAB, MICROANGIOPATHY, MUTATIONS, INSIGHTS

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Chicago
Licht, Christoph, Gianluigi Ardissino, Gema Ariceta, David Cohen, J Alexander Cole, Christoph Gasteyger, Larry A Greenbaum, et al. 2015. “The Global aHUS Registry : Methodology and Initial Patient Characteristics.” Bmc Nephrology 16.
APA
Licht, C., Ardissino, G., Ariceta, G., Cohen, D., Cole, J. A., Gasteyger, C., Greenbaum, L. A., et al. (2015). The global aHUS registry : methodology and initial patient characteristics. BMC NEPHROLOGY, 16.
Vancouver
1.
Licht C, Ardissino G, Ariceta G, Cohen D, Cole JA, Gasteyger C, et al. The global aHUS registry : methodology and initial patient characteristics. BMC NEPHROLOGY. 2015;16.
MLA
Licht, Christoph et al. “The Global aHUS Registry : Methodology and Initial Patient Characteristics.” BMC NEPHROLOGY 16 (2015): n. pag. Print.
@article{8605989,
  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 ({\textrangle}= 18 years) and 201 (39.0 \%) were {\textlangle}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 {\textrangle}= 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.},
  articleno    = {207},
  author       = {Licht, Christoph and Ardissino, Gianluigi and Ariceta, Gema and Cohen, David and Cole, J Alexander and Gasteyger, Christoph and Greenbaum, Larry A and Johnson, Sally and Ogawa, Masayo and Schaefer, Franz and Vande Walle, Johan and Fr{\'e}meaux-Bacchi, V{\'e}ronique},
  issn         = {1471-2369},
  journal      = {BMC NEPHROLOGY},
  pages        = {8},
  title        = {The global aHUS registry : methodology and initial patient characteristics},
  url          = {http://dx.doi.org/10.1186/s12882-015-0195-1},
  volume       = {16},
  year         = {2015},
}

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