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Thrombotic Thrombocytopenic Purpura: Pathophysiology, Diagnosis, and Management

Division of Hematology, Department of Medicine, The Ohio State University, Columbus, OH 43210, USA
Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, University of Bern, CH 3010 Bern, Switzerland
Center for Thrombosis and Hemostasis, University Medical Center, Johannes Gutenberg University, 55131 Mainz, Germany
Haemostasis Research Unit, University College London, London WC1E 6BT, UK
Author to whom correspondence should be addressed.
Academic Editor: Michael Callaghan
J. Clin. Med. 2021, 10(3), 536;
Received: 30 December 2020 / Revised: 24 January 2021 / Accepted: 27 January 2021 / Published: 2 February 2021
(This article belongs to the Special Issue The Latest Clinical Advances in Thrombocytopenia)
Thrombotic thrombocytopenic purpura (TTP) is a rare thrombotic microangiopathy characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and ischemic end organ injury due to microvascular platelet-rich thrombi. TTP results from a severe deficiency of the specific von Willebrand factor (VWF)-cleaving protease, ADAMTS13 (a disintegrin and metalloprotease with thrombospondin type 1 repeats, member 13). ADAMTS13 deficiency is most commonly acquired due to anti-ADAMTS13 autoantibodies. It can also be inherited in the congenital form as a result of biallelic mutations in the ADAMTS13 gene. In adults, the condition is most often immune-mediated (iTTP) whereas congenital TTP (cTTP) is often detected in childhood or during pregnancy. iTTP occurs more often in women and is potentially lethal without prompt recognition and treatment. Front-line therapy includes daily plasma exchange with fresh frozen plasma replacement and immunosuppression with corticosteroids. Immunosuppression targeting ADAMTS13 autoantibodies with the humanized anti-CD20 monoclonal antibody rituximab is frequently added to the initial therapy. If available, anti-VWF therapy with caplacizumab is also added to the front-line setting. While it is hypothesized that refractory TTP will be less common in the era of caplacizumab, in relapsed or refractory cases cyclosporine A, N-acetylcysteine, bortezomib, cyclophosphamide, vincristine, or splenectomy can be considered. Novel agents, such as recombinant ADAMTS13, are also currently under investigation and show promise for the treatment of TTP. Long-term follow-up after the acute episode is critical to monitor for relapse and to diagnose and manage chronic sequelae of this disease. View Full-Text
Keywords: thrombotic thrombocytopenic purpura; TTP; ADAMTS13; treatment; diagnosis; follow-up; review; caplacizumab thrombotic thrombocytopenic purpura; TTP; ADAMTS13; treatment; diagnosis; follow-up; review; caplacizumab
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MDPI and ACS Style

Sukumar, S.; Lämmle, B.; Cataland, S.R. Thrombotic Thrombocytopenic Purpura: Pathophysiology, Diagnosis, and Management. J. Clin. Med. 2021, 10, 536.

AMA Style

Sukumar S, Lämmle B, Cataland SR. Thrombotic Thrombocytopenic Purpura: Pathophysiology, Diagnosis, and Management. Journal of Clinical Medicine. 2021; 10(3):536.

Chicago/Turabian Style

Sukumar, Senthil, Bernhard Lämmle, and Spero R. Cataland. 2021. "Thrombotic Thrombocytopenic Purpura: Pathophysiology, Diagnosis, and Management" Journal of Clinical Medicine 10, no. 3: 536.

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