Expert review of hematology
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Introduction: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a life-threatening disease characterized by a severe functional deficit in the von-Willebrand cleaving protease ADAMTS13, due to autoantibody production. The once-dismal prognosis of the disease has been changed by the discovery of the dramatic efficiency of therapeutic plasma exchange (TPE). Areas covered: This review focuses on the history and recent developments in the use of TPE for iTTP with a special emphasis on the consequences for TPE practice of the recent introduction of new highly effective immunosuppressive strategies and anti-von Willebrand factor (vWF) therapies. Expert opinion: Although TPE still represents the cornerstone, emergency treatment of iTTP, their duration, and associated complications could be dramatically reduced in the future by the systematic addition of early immunosuppression using corticosteroids and rituximab as well as an anti-vWF therapy with caplacizumab.
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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. ⋯ 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.