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Abstract
Acquired thrombotic thrombocytopenic purpura (TTP) is a rare and severe disease due to autoimmune ADAMTS13 deficiency. Plasma exchange therapy is the standard of care associated with immunosuppression. In recent years, caplacizumab, a new monoclonal antibody, was approved in Europe and the USA in combination with standard therapy. Caplacizumab blocks the binding of von Willebrand factor to the platelet IB-IX-V receptor, decreasing platelet adhesion and aggregation and consequently the risk of developing microthrombi. Phase II and III studies have shown that caplacizumab reduces the time required for platelet count normalization and the risk of disease relapse but conversely, the risk of bleeding increases. It was not yet approved in our country. Real-life and cost-effectiveness studies are required to increase our knowledge regarding this drug.
References
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