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 TTP (iTTP) pathophysiology have led to novel targeted therapies. Immunomodulatory strategies target anti-ADAMTS13 antibodies. Of these, rituximab is effective in inducing responses in refractory/relapsed TTP, and increasing relapse free survival. Caplacizumab targets the VWF-platelet interaction to hasten platelet count recovery and reduces mortality and TTP-related ischemic events. Other promising therapies including bortezomib and recombinant ADAMTS13 are under investigation. This Spotlight review examines how targeted therapies are disrupting current treatment paradigms to improve outcomes of iTTP.

  • Submitted February 12, 2019.
  • Revision received June 18, 2019.
  • Accepted June 13, 2019.