{"title":"Impact of new medications on the treatment of Immune TTP.","authors":"Marie Scully, Lara Howells, William A Lester","doi":"10.1182/blood.2024026390","DOIUrl":null,"url":null,"abstract":"<p><p>The last decade has seen the introduction of two new licensed therapies for thrombotic thrombocytopenic purpura (TTP)-caplacizumab and recombinant ADAMTS 13 (rADAMTS 13), for immune (iTTP) and congenital TTP (cTTP) respectively. They improve acute TTP outcomes, reduce the need for plasma therapy, time to clinical response and treatment burden. Future pathways need to replace plasma exchange in acute TTP and optimise/personalise rADAMTS 13 in cTTP. Future emphasis should focus on additional monoclonals/treatments to tackle ADAMTS 13 antibodies.</p>","PeriodicalId":9102,"journal":{"name":"Blood","volume":" ","pages":""},"PeriodicalIF":21.0000,"publicationDate":"2025-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1182/blood.2024026390","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
The last decade has seen the introduction of two new licensed therapies for thrombotic thrombocytopenic purpura (TTP)-caplacizumab and recombinant ADAMTS 13 (rADAMTS 13), for immune (iTTP) and congenital TTP (cTTP) respectively. They improve acute TTP outcomes, reduce the need for plasma therapy, time to clinical response and treatment burden. Future pathways need to replace plasma exchange in acute TTP and optimise/personalise rADAMTS 13 in cTTP. Future emphasis should focus on additional monoclonals/treatments to tackle ADAMTS 13 antibodies.
期刊介绍：
Blood, the official journal of the American Society of Hematology, published online and in print, provides an international forum for the publication of original articles describing basic laboratory, translational, and clinical investigations in hematology. Primary research articles will be published under the following scientific categories: Clinical Trials and Observations; Gene Therapy; Hematopoiesis and Stem Cells; Immunobiology and Immunotherapy scope; Myeloid Neoplasia; Lymphoid Neoplasia; Phagocytes, Granulocytes and Myelopoiesis; Platelets and Thrombopoiesis; Red Cells, Iron and Erythropoiesis; Thrombosis and Hemostasis; Transfusion Medicine; Transplantation; and Vascular Biology. Papers can be listed under more than one category as appropriate.
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