How targeted therapy disrupts the treatment paradigm for acquired TTP: the risks, benefits, and unknowns

Insights into immune-mediated thrombotic thrombocytopenic purpura (iTTP) pathophysiology have led to novel targeted therapies. Immunomodulatory strategies target anti-ADAMTS13 antibodies: rituximab is effective in inducing responses in refractory/relapsed TTP and increasing relapse-free survival; caplacizumab targets the von Willebrand factor–platelet interaction to hasten platelet count recovery and reduce mortality and TTP-related ischemic events. Bortezomib and recombinant ADAMTS13 are under investigation. This review examines how targeted therapies are disrupting current treatment paradigms to improve outcomes of iTTP.
Source: Blood - Category: Hematology Authors: Tags: Thrombocytopenia, Platelets and Thrombopoiesis, Thrombosis and Hemostasis, Blood Spotlight, Clinical Trials and Observations Source Type: research