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Clinical Characteristics and Outcome of Canadian Patients Diagnosed With Atypical Hemolytic Uremic Syndrome

Published on Jan 24, 2020in Canadian journal of kidney health and disease
路 DOI :10.1177/2054358119897229
Anne-Laure Lapeyraque1
Estimated H-index: 1
(Centre Hospitalier Universitaire Sainte-Justine),
Martin Bitzan14
Estimated H-index: 14
(Montreal Children's Hospital)
+ 9 AuthorsAxel Tosikyan
Abstract
Background:Atypical hemolytic uremic syndrome (aHUS) is an extremely rare, heterogeneous disease of uncontrolled activation of the alternative complement pathway that is difficult to diagnose. We h...
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References19
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#1Jan MenneH-Index: 19
#2Yahsou DelmasH-Index: 24
Last. Larry A. Greenbaum (Emory University)H-Index: 22
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4 CitationsSource
#1Franz Schaefer (Heidelberg University)H-Index: 62
#2Gianluigi ArdissinoH-Index: 24
Last. Lisa SartzH-Index: 9
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Atypical hemolytic uremic syndrome (aHUS) is a rare, genetic, life-threatening disease. The Global aHUS Registry collects real-world data on the natural history of the disease. Here we characterize end-stage renal disease (ESRD)-free survival, the rate of thrombotic microangiopathy, organ involvement and the genetic background of 851 patients in the registry, prior to eculizumab treatment. A sex-specific difference was apparent according to age at initial disease onset as the ratio of males to f...
17 CitationsSource
#1Ghada A Ankawi (London Health Sciences Centre)H-Index: 1
#2William F. Clark (UWO: University of Western Ontario)H-Index: 43
Historically, patients with kidney diseases caused by genetic or acquired dysregulation of the complement alternative pathway have been grouped into clinical syndromes, C3 glomerulopathy (C3GN/DDD) and thrombotic microangiopathy (TMA), specifically atypical haemolytic uremic syndrome (aHUS). Recent data suggested that these diseases share a common pathophysiology and that patients can transition between glomerulopathies in this spectrum. Histopathologically, the main difference cited is the immu...
1 CitationsSource
#1Timothy H.J. Goodship (Newcastle University)H-Index: 55
#2H. Terence Cook (Imperial College London)H-Index: 59
Last. Peter F. ZipfelH-Index: 81
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In both atypical hemolytic uremic syndrome (aHUS) and C3 glomerulopathy (C3G) complement plays a primary role in disease pathogenesis. Herein we report the outcome of a 2015 Kidney Disease: Improving Global Outcomes (KDIGO) Controversies Conference where key issues in the management of these 2 diseases were considered by a global panel of experts. Areas addressed included renal pathology, clinical phenotype and assessment, genetic drivers of disease, acquired drivers of disease, and treatment st...
117 CitationsSource
#1Fadi FakhouriH-Index: 42
#2Maryvonne HourmantH-Index: 35
Last. Christophe Legendre (Paris V: Paris Descartes University)H-Index: 57
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Background Atypical hemolytic uremic syndrome (aHUS) is a rare genetic life-threatening disease of chronic uncontrolled complement activation leading to thrombotic microangiopathy (TMA) and severe end-organ damage. Eculizumab, a terminal complement inhibitor approved for aHUS treatment, was reported to improve hematologic and renal parameters in 2 prior prospective phase 2 studies. This is the largest prospective study of eculizumab in aHUS to date, conducted in an adult population. Study Design...
84 CitationsSource
#1Gianluigi Ardissino (Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico)H-Index: 24
#2Stefania Salardi (Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico)H-Index: 8
Last. Silvana Tedeschi (Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico)H-Index: 18
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Despite the severity of HUS and the fact that it represents a leading cause of acute kidney injury in children, the general epidemiology of HUS is all but well documented. The present study provides updated, population-based, purely epidemiological information on HUS in childhood from a large and densely populated area of northern Italy (9.6 million inhabitants, 1.6 million children). We systematically reviewed the files concerning patients with STEC-HUS and atypical HUS (aHUS) over a 10-year ob...
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#1Larry A. Greenbaum (Emory University)H-Index: 22
#2Marc FilaH-Index: 7
Last. Christoph LichtH-Index: 13
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Atypical hemolytic uremic syndrome (aHUS) is caused by聽alternative complement pathway dysregulation, leading to systemic thrombotic microangiopathy (TMA) and severe聽end-organ damage. Based on 2 prospective studies in mostly adults and retrospective data in children, eculizumab, a terminal complement inhibitor, is approved for aHUS treatment. Here we prospectively evaluated efficacy and safety of weight-based dosing of eculizumab in eligible pediatric patients with aHUS in an open-label phase II ...
90 CitationsSource
#1Christoph LichtH-Index: 13
#2Gianluigi Ardissino (Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico)H-Index: 24
view all 12 authors...
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.
30 CitationsSource
#1Josep M. CampistolH-Index: 63
#2M. AriasH-Index: 20
Last. S. Rodr铆guez de C贸rdoba (CSIC: Spanish National Research Council)H-Index: 57
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Haemolytic uraemic syndrome (HUS) is a clinical entity defined as the triad of nonimmune haemolytic anaemia, thrombocytopenia, and acute renal failure, in which the underlying lesions are mediated by systemic thrombotic microangiopathy (TMA). Atypical HUS (aHUS) is a sub-type of HUS in which the TMA phenomena are the consequence of decreased regulation of the alternative complement pathway on cell surfaces due to a genetic cause. aHUS is an extremely rare disease that, despite the administration...
60 CitationsSource
#1Christoph LichtH-Index: 13
#2Larry A. Greenbaum (Emory University)H-Index: 22
Last. Chantal LoiratH-Index: 59
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Atypical hemolytic uremic syndrome (aHUS) is a rare, possibly life-threatening disease characterized by platelet activation, hemolysis and thrombotic microangiopathy (TMA) leading to renal and other end-organ damage. We originally conducted two phase 2 studies (26 weeks and 1 year) evaluating eculizumab, a terminal complement inhibitor, in patients with progressing TMA (trial 1) and those with long duration of aHUS and chronic kidney disease (trial 2). The current analysis assessed outcomes afte...
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