An update on pathogenesis and diagnosis of thrombotic thrombocytopenic purpura

被引：82

作者：

Joly, Berangere S. ^{[1
,2
]}

Coppo, Paul ^{[3
]}

Veyradier, Agnes ^{[1
,2
]}

机构：

[1] Univ Paris Dider, Grp Hosp St Louis Lariboisiere, AP HP, Serv Hematol Biol,Inst Univ Hematol, Paris, France

[2] Univ Paris Dider, Grp Hosp St Louis Lariboisiere, AP HP, Ctr Hayem EA3518,Inst Univ Hematol, Paris, France

[3] Univ Sorbonne Paris Cite, Hop St Antoine, AP HP, Serv Hematol, Paris, France

来源：

EXPERT REVIEW OF HEMATOLOGY | 2019年 / 12卷 / 06期

关键词：

Thrombotic microangiopathy; thrombotic thrombocytopenic purpura; ADAMTS13; von Willebrand factor; autoimmunity; rare disease; VON-WILLEBRAND-FACTOR; FACTOR-CLEAVING PROTEASE; SEVERE ADAMTS13 DEFICIENCY; FRENCH NATIONAL REGISTRY; UPSHAW-SCHULMAN SYNDROME; MOLECULAR CHARACTERIZATION; CONFORMATIONAL ACTIVATION; ANTI-ADAMTS13; ANTIBODIES; PLASMA-EXCHANGE; FACTOR-VIII;

D O I：

10.1080/17474086.2019.1611423

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：383 / 395

页数：13

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