title: Acquired thrombotic thrombocytopenic purpura: new therapeutic options and their optimal use.
abstract: Advances in our understanding of the pathophysiology of both congenital and acquired thrombotic thrombocytopenic purpura (TTP) have led to both an increased understanding of the disease and novel approaches to therapy. The efficacy of rituximab in acquired TTP has led to consideration of rituximab as a prophylactic therapy to prevent relapse of TTP. Novel therapies that target the A1 domain of von Willebrand factor (VWF) to block the formation of microthrombotic disease have also entered clinical study and have demonstrated promise as potential therapeutic options. Additionally, a recombinant ADAMTS13 protease has been developed which may be an important therapeutic option for both congenital and acquired TTP. The development of these new therapeutic options for patients diagnosed with TTP has increased the importance of conducting prospective, randomized studies with these agents to both confirm their efficacy and more importantly understand their most appropriate role in the treatment of patients with TTP.
authors.affiliation: Department of Medicine, Ohio State University, Columbus, OH, USA.
authors.forename: S R
authors.initials: SR
authors.lastname: Cataland
authors.affiliation: Department of Pathology, Ohio State University, Columbus, OH, USA.
authors.forename: H M
authors.initials: HM
authors.lastname: Wu
mesh_terms: D051722:ADAM Proteins / Q000009:adverse effects / Q000097:blood / Q000172:deficiency / Q000627:therapeutic use*; D000071120:ADAMTS13 Protein; D000818:Animals; D001777:Blood Coagulation / Q000187:drug effects*; D055808:Drug Discovery; D056947:Enzyme Replacement Therapy / Q000009:adverse effects; D005343:Fibrinolytic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D058990:Molecular Targeted Therapy; D011697:Purpura, Thrombotic Thrombocytopenic / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy* / Q000201:enzymology; D011994:Recombinant Proteins / Q000627:therapeutic use; D012008:Recurrence; D012074:Remission Induction; D000069283:Rituximab / Q000627:therapeutic use; D013156:Splenectomy; D016896:Treatment Outcome; D014841:von Willebrand Factor / Q000037:antagonists & inhibitors
chemical_list: D005343:Fibrinolytic Agents; D011994:Recombinant Proteins; D014841:von Willebrand Factor; D000069283:Rituximab; D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human
keywords: ADAMTS13 protein, human; relapse; rituximab; thrombotic thrombocytopenic purpura; von Willebrand factor