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.
Bibliographical noteFunding Information:
This work was supported by a Mentored Research Award from the Hemostasis and Thrombosis Research Society (S.C.). The funders had no role in preparation of the manuscript, or decision to publish.
PubMed: MeSH publication types
- Journal Article
- Research Support, Non-U.S. Gov't