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Carla M Nester
Licht et al. present the 2-year follow-up data of the landmark trials studying the efficacy of eculizumab in the treatment of atypical hemolytic uremic syndrome (aHUS). They report sustained improvements in hematologic parameters, continued safety, and additional improvements in kidney function with extended treatment. This report adds a layer of comfort to our care of patients with this rare disease; however, it is unlikely to be the final chapter in the treatment of aHUS.
May 2015: Kidney International
Antonio M Risitano
The availability of anticomplement therapies has been a major achievement for medicine in the last decade. Indeed, eculizumab has changed the treatment paradigm of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome and promises to do the same in several other human complement-mediated diseases. Nowadays, a 10-year experience has also taught us that there are some pitfalls that represent a challenge to improve the current anticomplement treatment. Most of these observations come from paroxysmal nocturnal hemoglobinuria, where unmet clinical needs are emerging, triggering the attention of several investigators and pharmaceutical companies...
June 2015: Hematology/oncology Clinics of North America
Marina Noris, Giuseppe Remuzzi
No abstract text is available yet for this article.
August 2015: American Journal of Kidney Diseases: the Official Journal of the National Kidney Foundation
Carla M Nester, Thomas Barbour, Santiago Rodriquez de Cordoba, Marie Agnes Dragon-Durey, Veronique Fremeaux-Bacchi, Tim H J Goodship, David Kavanagh, Marina Noris, Matthew Pickering, Pilar Sanchez-Corral, Christine Skerka, Peter Zipfel, Richard J H Smith
Tremendous advances in our understanding of the thrombotic microangiopathies (TMAs) have revealed distinct disease mechanisms within this heterogeneous group of diseases. As a direct result of this knowledge, both children and adults with complement-mediated TMA now enjoy higher expectations for long-term health. In this update on atypical hemolytic uremic syndrome, we review the clinical characteristics; the genetic and acquired drivers of disease; the broad spectrum of environmental triggers; and current diagnosis and treatment options...
September 2015: Molecular Immunology
Knut Tore Lappegård, Anna Bjerre, Geir Erland Tjønnfjord, Tom Eirik Mollnes
Internationally, the use of the C5-inhibiting monoclonal antibody eculizumab has in the course of just a few years become the first choice of treatment of atypical haemolytic uraemic syndrome and the most severe phenotypes of paroxysmal nocturnal haemoglobinuria. At present eculizumab is the only complement inhibitor in ordinary clinical use. This despite the fact that there only exists one randomised, placebo-controlled trial of eculizumab for paroxysmal nocturnal haemoglobinuria and none for atypical haemolytic uraemic syndrome, and that the therapy is very costly...
October 20, 2015: Tidsskrift for Den Norske Lægeforening: Tidsskrift for Praktisk Medicin, Ny Række
An S De Vriese, Sanjeev Sethi, Jens Van Praet, Karl A Nath, Fernando C Fervenza
Kidney diseases caused by genetic or acquired dysregulation of the complement alternative pathway (AP) are traditionally classified on the basis of clinical presentation (atypical hemolytic uremic syndrome as thrombotic microangiopathy), biopsy appearance (dense deposit disease and C3 GN), or clinical course (atypical postinfectious GN). Each is characterized by an inappropriate activation of the AP, eventuating in renal damage. The clinical diversity of these disorders highlights important differences in the triggers, the sites and intensity of involvement, and the outcome of the AP dysregulation...
December 2015: Journal of the American Society of Nephrology: JASN
Lance A Williams, Marisa B Marques
OBJECTIVES: Pathologists specializing in transfusion medicine, apheresis medicine, and/or coagulation are often consulted by clinicians to reach a diagnosis for patients with thrombotic microangiopathy (TMA), so that disease-specific, often life-saving therapy can be initiated as promptly as possible. METHODS: This article describes how to proceed when treating a patient with TMA. The differential diagnosis is broad and potentially very challenging. Thrombotic thrombocytopenic purpura (TTP), atypical hemolytic uremic syndrome (aHUS), and typical hemolytic uremic syndrome (HUS) are three such TMAs that require timely diagnosis and treatment...
February 2016: American Journal of Clinical Pathology
Andrea Angioi, Fernando C Fervenza, Sanjeev Sethi, Yuzhou Zhang, Richard J Smith, David Murray, Jens Van Praet, Antonello Pani, An S De Vriese
Kidney diseases resulting from abnormal control of the complement alternative pathway include atypical hemolytic uremic syndrome, C3 glomerulonephritis, and dense-deposit disease, as well as atypical postinfectious glomerulonephritis. Although clinically diverse, they all result from loss of surface or fluid-phase complement control, caused by acquired or genetic defects in the complement alternative pathway. As such, the diagnostic approach is similar and includes a comprehensive biochemical, genetic, and pathologic analysis of the complement pathway...
February 2016: Kidney International
Fadi Fakhouri
Pregnancy is a high-risk period for various types of thrombotic microangiopathies (TMA). The improvement of our understanding of the pathophysiology of TMAs has translated into better management of pregnancy-related TMAs. The two main types of TMA, TTP (thrombotic thrombocytopenic purpura) and hemolytic uremic syndrome (HUS), can both occur during pregnancy and postpartum. TTP is related in most cases to acquired or congenital deficiency of ADAMTS13; it tends to develop mainly during the second and third trimesters of pregnancy...
April 2016: Transfusion and Apheresis Science
Chia Wei Teoh, Magdalena Riedl, Christoph Licht
Thrombotic microangiopathies (TMA) are disorders defined by microangiopathic hemolytic anemia, non-immune thrombocytopenia and have multi-organ involvement including the kidneys, brain, gastrointestinal, respiratory tract and skin. Emerging evidence points to the central role of complement dysregulation in leading to microvascular endothelial injury which is crucial for the development of TMAs. This key insight has led to the development of complement-targeted therapy. Eculizumab is an anti-C5 monoclonal antibody, which has revolutionized the treatment of atypical hemolytic uremic syndrome...
April 2016: Transfusion and Apheresis Science
Prabesh Bajracharya, Amrish Jain, Rossana Baracco, Tej K Mattoo, Gaurav Kapur
BACKGROUND: Patients negative for Shiga toxin-producing E. coli (STEC) are categorized as having atypical hemolytic uremic syndrome (HUS) and are associated with an increased risk for complement mutations and poorer prognosis compared with typical HUS. However, STEC identification is limited by the natural history of HUS. METHODS: The current study is aimed at identifying HUS patients with poor outcomes based on the presence or absence of diarrhea (D) or Shiga toxin (S)...
October 2016: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
Carla M Nester, Christie P Thomas
Atypical hemolytic uremic syndrome (aHUS) is a rare syndrome of hemolysis, thrombocytopenia, and renal insufficiency. Genetic mutations in the alternate pathway of complement are well recognized as the cause in more than 60% of patients affected by this thrombotic microangiopathy. The identification of aHUS as a disease of the alternate pathway of complement enables directed therapeutic intervention both in the acute and chronic setting and may include one or all of the following: plasma therapy, complement blockade, and liver transplantation...
2012: Hematology—the Education Program of the American Society of Hematology
Johan Vande Walle, Yahsou Delmas, Gianluigi Ardissino, Jimmy Wang, John F Kincaid, Herman Haller
BACKGROUND: Eculizumab is approved for atypical hemolytic uremic syndrome (aHUS). Guidelines discuss the importance of prompt treatment. We report a post hoc analysis investigating the effect of baseline factors, including patient characteristics and time from the latest aHUS manifestation to eculizumab initiation, on change from baseline in estimated glomerular filtration rate (eGFR) and other outcomes. METHODS: Data were pooled from four phase 2, open-label, single-arm, prospective clinical studies of eculizumab for patients with aHUS...
February 2017: Journal of Nephrology
Chantal Loirat, Fadi Fakhouri, Gema Ariceta, Nesrin Besbas, Martin Bitzan, Anna Bjerre, Rosanna Coppo, Francesco Emma, Sally Johnson, Diana Karpman, Daniel Landau, Craig B Langman, Anne-Laure Lapeyraque, Christoph Licht, Carla Nester, Carmine Pecoraro, Magdalena Riedl, Nicole C A J van de Kar, Johan Van de Walle, Marina Vivarelli, Véronique Frémeaux-Bacchi
Atypical hemolytic uremic syndrome (aHUS) emerged during the last decade as a disease largely of complement dysregulation. This advance facilitated the development of novel, rational treatment options targeting terminal complement activation, e.g., using an anti-C5 antibody (eculizumab). We review treatment and patient management issues related to this therapeutic approach. We present consensus clinical practice recommendations generated by HUS International, an international expert group of clinicians and basic scientists with a focused interest in HUS...
January 2016: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
Tao Zhang, Jianping Lu, Shaoshan Liang, Dachen Chen, Haitao Zhang, Caihong Zeng, Zhihong Liu, Huimei Chen
BACKGROUND: Genetic defects in complement proteins reportedly contribute to the atypical hemolytic uremic syndrome (aHUS). Numerous genetic studies have been published in recent years, but limited data have been gathered from Asian countries. METHODS: Genetic variants of 11 complement genes were analyzed in 23 Chinese patients with aHUS by high-throughput sequencing. The genotype-phenotype relationship in the Han population was evaluated and compared with the relationship that existed in other ethnicities...
2016: American Journal of Nephrology
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