Kremer Hovinga JA Coppo P Lämmle B Moake JL Miyata T Vanhoorelbeke K 2017 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. https://sonar.ch/global/documents/43924 eng info:eu-repo/semantics/altIdentifier/doi/10.1038/nrdp.2017.20 info:eu-repo/semantics/altIdentifier/pmid/28382967 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 Thrombotic thrombocytopenic purpura. http://purl.org/coar/resource_type/c_6501