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

Marshall A. Mazepa, Camila Masias and Shruti Chaturvedi


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.

  • Submitted February 12, 2019.
  • Accepted June 13, 2019.
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