Document Detail


Genetics and genetic testing in hemolytic uremic syndrome/thrombotic thrombocytopenic purpura.
MedLine Citation:
PMID:  20807612     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
The hemolytic uremic syndrome (HUS) and thrombotic thrombocytopenic purpura (TTP) are rare diseases that manifest with thrombocytopenia and microangiopathic hemolytic anemia accompanied by renal and neurologic dysfunction. Most childhood cases of HUS are caused by Shiga-toxin-producing bacteria and have a good prognosis. The other form, atypical HUS (aHUS), accounts for 10% of cases. Prognosis of aHUS and TTP has changed over time from fatal disorders to 60% to 80% survival in the plasma therapy era. In the past 10 years the molecular bases of aHUS and TTP have been discovered that mostly lead to uncontrolled activation of the complement system in aHUS and to abnormal von Willebrand factor processing in TTP. Identification of the underlying abnormality in an individual patient can provide prognostically significant information in predicting long-term outcome, response to therapies, and transplant outcome. It also paves the way for the use of specific new therapies in the near future.
Authors:
Marina Noris; Giuseppe Remuzzi
Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  Seminars in nephrology     Volume:  30     ISSN:  1558-4488     ISO Abbreviation:  Semin. Nephrol.     Publication Date:  2010 Jul 
Date Detail:
Created Date:  2010-09-02     Completed Date:  2011-01-18     Revised Date:  2011-11-24    
Medline Journal Info:
Nlm Unique ID:  8110298     Medline TA:  Semin Nephrol     Country:  United States    
Other Details:
Languages:  eng     Pagination:  395-408     Citation Subset:  IM    
Copyright Information:
Copyright 2010 Elsevier Inc. All rights reserved.
Affiliation:
Mario Negri Institute for Pharmacological Research, Clinical Research Center for Rare Diseases, Aldo e Cele Daccò, Villa Camozzi, Ranica, Bergamo, Italy. marina.noris@marionegri.it
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MeSH Terms
Descriptor/Qualifier:
ADAM Proteins / genetics*
Complement Activation / genetics*
Complement System Proteins / genetics
Female
Hemolytic-Uremic Syndrome / diagnosis,  genetics*,  physiopathology
Humans
Male
Mutation / genetics
Purpura, Thrombotic Thrombocytopenic / diagnosis,  genetics*,  physiopathology
Grant Support
ID/Acronym/Agency:
GGP09075//Telethon
Chemical
Reg. No./Substance:
9007-36-7/Complement System Proteins; EC 3.4.24.-/ADAM Proteins; EC 3.4.24.-/ADAMTS13 protein, human

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