{"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><strong>Abstract: </strong>The last decade has seen the introduction of 2 new licensed therapies for thrombotic thrombocytopenic purpura (TTP), caplacizumab and recombinant ADAMTS13 (rADAMTS13), for immune and congenital TTP (cTTP), respectively. They improve acute TTP outcomes, and reduce the need for plasma therapy, time to clinical response, and treatment burden. Future pathways need to replace plasma exchange in acute TTP and optimize/personalize rADAMTS13 in cTTP. Future emphasis should focus on additional monoclonals/treatments to tackle ADAMTS13 antibodies.</p>","PeriodicalId":9102,"journal":{"name":"Blood","volume":" ","pages":"1353-1357"},"PeriodicalIF":23.1000,"publicationDate":"2025-03-27","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
Abstract: The last decade has seen the introduction of 2 new licensed therapies for thrombotic thrombocytopenic purpura (TTP), caplacizumab and recombinant ADAMTS13 (rADAMTS13), for immune and congenital TTP (cTTP), respectively. They improve acute TTP outcomes, and reduce the need for plasma therapy, time to clinical response, and treatment burden. Future pathways need to replace plasma exchange in acute TTP and optimize/personalize rADAMTS13 in cTTP. Future emphasis should focus on additional monoclonals/treatments to tackle ADAMTS13 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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