Thrombotic Thrombocytopenic Purpura: From the Bench to the Bedside, but Not Yet to the Community

Editorials21 January 2003Thrombotic Thrombocytopenic Purpura: From the Bench to the Bedside, but Not Yet to the CommunityJames N. George, MD and Sara K. Vesely, PhDJames N. George, MDFrom The University of Oklahoma Health Sciences Center; Oklahoma City, OK 73190Search for more papers by this author and Sara K. Vesely, PhDFrom The University of Oklahoma Health Sciences Center; Oklahoma City, OK 73190Search for more papers by this authorAuthor, Article, and Disclosure Informationhttps://doi.org/10.7326/0003-4819-138-2-200301210-00018 SectionsAboutFull TextPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareFacebookTwitterLinkedInRedditEmail In the past 4 years, remarkable advances have been made in our understanding of the pathogenesis of thrombotic thrombocytopenic purpura (TTP). This condition is associated with a severe deficiency of von Willebrand factorcleaving protease (1-4), now known as ADAMTS13 (5). This deficiency prevents normal processing of large von Willebrand factor multimers that are secreted from endothelial cells (6). Persistence of the large von Willebrand factor multimers in the circulation helps form microvascular platelet thrombi, the pathologic hallmark of TTP (6). Mutations of the ADAMTS13 gene may result in life-long recurrent episodes of TTP due to congenital deficiency of ADAMTS13 (7). ...References1. Furlan M, Robles R, Galbusera M, Remuzzi G, Kyrle PA, Brenner B, . von Willebrand factor-cleaving protease in thrombotic thrombocytopenic purpura and the hemolytic-uremic syndrome. N Engl J Med. 1998;339:1578-84. [PMID: 9828245] CrossrefMedlineGoogle Scholar2. Tsai HM, Lian EC. Antibodies to von Willebrand factor-cleaving protease in acute thrombotic thrombocytopenic purpura. N Engl J Med. 1998;339:1585-94. [PMID: 9828246] CrossrefMedlineGoogle Scholar3. Furlan M, Lmmle B. 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Kidney Int. 2001;60:831-46. [PMID: 11532079] CrossrefMedlineGoogle Scholar17. Veyradier A, Obert B, Houllier A, Meyer D, Girma JP. Specific von Willebrand factor-cleaving protease in thrombotic microangiopathies: a study of 111 cases. Blood. 2001;98:1765-72. [PMID: 11535510] CrossrefMedlineGoogle Scholar18. Barbot J, Costa E, Guerra M, Barreirinho MS, Isvarlal P, Robles R, . Ten years of prophylactic treatment with fresh-frozen plasma in a child with chronic relapsing thrombotic thrombocytopenic purpura as a result of a congenital deficiency of von Willebrand factor-cleaving protease. Br J Haematol. 2001;113:649-51. [PMID: 11380451] CrossrefMedlineGoogle Scholar19. Rock G, Shumak K, Kelton J, Blanchette VS, Buskard N, Nair R, . Thrombotic thrombocytopenic purpura: outcome in 24 patients with renal impairment treated with plasma exchange. Canadian Apheresis Study Group. Transfusion. 1992;32:710-4. [PMID: 1412677] CrossrefMedlineGoogle Scholar Author, Article, and Disclosure InformationAffiliations: From The University of Oklahoma Health Sciences Center; Oklahoma City, OK 73190Corresponding Author: James N. George, MD, Hematology-Oncology Section, The University of Oklahoma Health Sciences Center, 1100 N. Lindsay, Oklahoma City, OK 73104; e-mail, [email protected]edu.Current Author Addresses: Drs. George and Vesely: Hematology-Oncology Section, The University of Oklahoma Health Sciences Center, PO Box 26901, Oklahoma City, OK 73190. PreviousarticleNextarticle Advertisement FiguresReferencesRelatedDetailsSee AlsoRemission of Chronic Thrombotic Thrombocytopenic Purpura after Treatment with Cyclophosphamide and Rituximab Xinglong Zheng , Arnel M. Pallera , Lawrence T. Goodnough , J. Evan Sadler , and Morey A. Blinder Metrics Cited ByTherapeutic ApheresisTherapeutic ApheresisTherapeutic ApheresisPurpura and Microvascular OcclusionProspective study on the behaviour of the metalloprotease ADAMTS13 and of von Willebrand factor after bone marrow transplantationPúrpura trombocitopénica trombótica, lupus eritematoso sistémico y tratamiento de rescate con ciclofosfamidaThe role of ADAMTS13 in the new pathogenesis of TTPThe Oklahoma thrombotic thrombocytopenic Purpura-Hemolytic uremic syndrome (TTP-HUS) registry: a community perspective of patients with clinically diagnosed TTP-HUSThrombotic thrombocytopenic purpura: advances in pathophysiology, diagnosis, and treatment—introductionADAMTS13 activity in thrombotic thrombocytopenic purpura–hemolytic uremic syndrome: relation to presenting features and clinical outcomes in a prospective cohort of 142 patients 21 January 2003Volume 138, Issue 2Page: 152-153KeywordsBlood plasmaChildrenCyclophosphamideHemolytic anemiaHemolytic uremic syndromePathogenesisPatient advocacyPatientsRenal failureThrombotic thrombocytopenic purpura ePublished: 21 January 2003 Issue Published: 21 January 2003 CopyrightCopyright © 2003 by American College of Physicians. 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