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Review
. 2019 Aug 1;134(5):415-420.
doi: 10.1182/blood.2019000954. Epub 2019 Jun 19.

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

Marshall A Mazepa  1 , Camila Masias  2 , Shruti Chaturvedi  3
Affiliations
Free article
Review

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

Marshall A Mazepa et al. Blood. .
Free article

Abstract

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.

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