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Thrombotic thrombocytopenic purpura.
Journal article

Thrombotic thrombocytopenic purpura.

  • Kremer Hovinga JA Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Freiburgstrasse, Bern, Switzerland.
  • Coppo P Centre de Référence des Microangiopathies Thrombotiques, Service d' Hématologie, Hôpitaux Universitaires de l'Est Parisien et Université Pierre et Marie Curie (Paris 6), Paris, France.
  • Lämmle B Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Freiburgstrasse, Bern, Switzerland.
  • Moake JL Department of Bioengineering, Rice University, Houston, Texas, USA.
  • Miyata T Department of Molecular Pathogenesis, National Cerebral and Cardiovascular Center, Suita, Japan.
  • Vanhoorelbeke K Laboratory for Thrombosis Research, Interdisciplinary Research Facility Life Sciences, Katholieke Universiteit Leuven Campus Kulak Kortrijk, Kortrijk, Belgium.
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  • 2017-04-07
Published in:
  • Nature reviews. Disease primers. - 2017
ADAMTS13 Protein
Acetylcysteine
Antiviral Agents
Female
Glucocorticoids
HIV Infections
Humans
Immunologic Factors
Immunomodulation
Pregnancy
Pregnancy Complications
Purpura, Thrombotic Thrombocytopenic
Rituximab
Shiga Toxins
Splenectomy
English Thrombotic thrombocytopenic purpura (TTP; also known as Moschcowitz disease) is characterized by the concomitant occurrence of often severe thrombocytopenia, microangiopathic haemolytic anaemia and a variable degree of ischaemic organ damage, particularly affecting the brain, heart and kidneys. Acute TTP was almost universally fatal until the introduction of plasma therapy, which improved survival from <10% to 80-90%. However, patients who survive an acute episode are at high risk of relapse and of long-term morbidity. A timely diagnosis is vital but challenging, as TTP shares symptoms and clinical presentation with numerous conditions, including, for example, haemolytic uraemic syndrome and other thrombotic microangiopathies. The underlying pathophysiology is a severe deficiency of the activity of a disintegrin and metalloproteinase with thrombospondin motifs 13 (ADAMTS13), the protease that cleaves von Willebrand factor (vWF) multimeric strings. Ultra-large vWF strings remain uncleaved after endothelial cell secretion and anchorage, bind to platelets and form microthrombi, leading to the clinical manifestations of TTP. Congenital TTP (Upshaw-Schulman syndrome) is the result of homozygous or compound heterozygous mutations in ADAMTS13, whereas acquired TTP is an autoimmune disorder caused by circulating anti-ADAMTS13 autoantibodies, which inhibit the enzyme or increase its clearance. Consequently, immunosuppressive drugs, such as corticosteroids and often rituximab, supplement plasma exchange therapy in patients with acquired TTP.
Language
  • English
Open access status
closed
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Persistent URL
https://sonar.ch/global/documents/43924
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