2. Academic Validation
  3. An update on pathogenesis and diagnosis of thrombotic thrombocytopenic purpura

An update on pathogenesis and diagnosis of thrombotic thrombocytopenic purpura

  • Expert Rev Hematol. 2019 Jun;12(6):383-395. doi: 10.1080/17474086.2019.1611423.
Bérangère S Joly 1,  Paul Coppo 2,  Agnès Veyradier 1
Affiliations

Affiliations

  • 1 a Service d'hématologie biologique and EA3518 Centre Hayem, Institut Universitaire d'Hématologie, Groupe Hospitalier Saint-Louis - Lariboisière, Assistance Publique - Hôpitaux de Paris, Université Paris Dider , Paris , France.
  • 2 b Service d'hématologie, Hôpital Saint-Antoine, Assistance Publique - Hôpitaux de Paris , Université Sorbonne Paris Cité , Paris , France.
PMID: 31107120 DOI: 10.1080/17474086.2019.1611423
Abstract

Introduction: Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy characterized by microangiopathic hemolytic anemia, consumption thrombocytopenia, and organ injury. TTP pathophysiology is based on a severe ADAMTS13 deficiency, the specific von Willebrand factor (VWF)-cleaving protease. Areas covered: VWF/ADAMTS13 interaction is specific, and ADAMTS13 conformation has been elucidated recently. ADAMTS13 deficiency is congenital or acquired. Several targets are used in TTP treatment. Expert opinion: Differential diagnosis of TTP may be challenging and ADAMTS13 investigations are needed. ADAMTS13 conformation could be a promising parameter for TTP diagnosis and prognosis in the coming years. Plasma therapy remains the first-line treatment of the acute phase of TTP. Additional curative and preemptive rituximab are used in acquired TTP. Clinical trials dedicated to innovative drugs are promising.

Keywords

ADAMTS13; Thrombotic microangiopathy; autoimmunity; rare disease; thrombotic thrombocytopenic purpura; von Willebrand factor.

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