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Open Access 10-09-2023 | Rituximab | Guideline

Diagnostic and treatment guidelines for thrombotic thrombocytopenic purpura (TTP) in Japan 2023

Authors: Masanori Matsumoto, Yoshitaka Miyakawa, Koichi Kokame, Yasunori Ueda, Hideo Wada, Satoshi Higasa, Hideo Yagi, Yoshiyuki Ogawa, Kazuya Sakai, Toshiyuki Miyata, Eriko Morishita, Yoshihiro Fujimura, For TTP group of Blood Coagulation Abnormalities Research Study Team, Research on Rare and Intractable diseases, Health and Labour Sciences Research Grants from the Ministry of Health, Labour and Welfare of Japan

Published in: International Journal of Hematology | Issue 5/2023

Abstract

Thrombotic thrombocytopenic purpura (TTP) can rapidly become a life-threatening condition, and the importance of its appropriate diagnosis and treatment cannot be overstated. Until recently, TTP has mainly been diagnosed by clinical findings such as thrombocytopenia and hemolytic anemia. In addition to these clinical findings, however, reduced activity of a disintegrin-like and metalloprotease with thrombospondin type 1 motif 13 (ADAMTS13) below 10% has become internationally accepted as a diagnostic criterion for TTP. TTP is classified as immune-mediated TTP (iTTP) if the patient is positive for anti-ADAMTS13 autoantibodies, and as congenital TTP (cTTP) if ADAMTS13 gene abnormalities are detected. Fresh frozen plasma (FFP) transfusion is performed in patients with cTTP to supplement ADAMTS13. Plasma exchange therapy using FFP is conducted in patients with iTTP to supplement ADAMTS13 and to remove both anti-ADAMTS13 autoantibodies and unusually large von Willebrand factor (VWF) multimers. To suppress autoantibody production, corticosteroid therapy is administered in conjunction with plasma exchange. The monoclonal anti-CD-20 antibody rituximab is effective in patients with iTTP. In addition, caplacizumab, an anti-VWF A1 domain nanobody, has a novel mechanism of action, involving direct inhibition of platelet glycoprotein Ib–VWF binding. The recommended first-line treatments of iTTP in Japan are plasma exchange and corticosteroids, as well as caplacizumab.

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Title: Diagnostic and treatment guidelines for thrombotic thrombocytopenic purpura (TTP) in Japan 2023
Authors: Masanori Matsumoto
Yoshitaka Miyakawa
Koichi Kokame
Yasunori Ueda
Hideo Wada
Satoshi Higasa
Hideo Yagi
Yoshiyuki Ogawa
Kazuya Sakai
Toshiyuki Miyata
Eriko Morishita
Yoshihiro Fujimura
For TTP group of Blood Coagulation Abnormalities Research Study Team, Research on Rare and Intractable diseases, Health and Labour Sciences Research Grants from the Ministry of Health, Labour and Welfare of Japan
Publication date: 10-09-2023
Publisher: Springer Nature Singapore
Keywords: Rituximab
Rituximab
Thrombotic Microangiopathy
Thrombotic Thrombocytopenic Purpura
Hemolytic Uremic Syndrome
Published in: International Journal of Hematology / Issue 5/2023
Print ISSN: 0925-5710
Electronic ISSN: 1865-3774
DOI: https://doi.org/10.1007/s12185-023-03657-0

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Prediction of major bleeding events in 1381 patients with essential thrombocythemia

COVID-19 omicron variant outbreak in a hematopoietic stem cell transplant unit

Laryngeal edema as a symptom of local cytokine release syndrome after BCMA-targeting CAR-T therapy for relapsed and refractory multiple myeloma

Keynote webinar | Spotlight on antibody–drug conjugates in cancer