title: Thrombotic thrombocytopenic purpura-what is new?
abstract: A functional deficiency of ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif), a von-Willebrand factor (VWF) cleaving protease, is central to the pathogenesis of congenital and acquired thrombotic thrombocytopenic purpura (TTP). ADAMTS13 testing has evolved from assays that required long denaturation and incubation times to ones that employ a modified recombinant VWF with improved standardization and turn around times. While plasma exchange is a mainstay in the treatment of TTP, increased use of rituximab, an antibody against CD20, has proved helpful in the treatment of patients with exacerbations and relapses. The next generation of drugs focuses on using recombinant ADAMTS13 and molecules that block the interaction of VWF and platelets to prevent thrombotic microangiopathy. The increased awareness and availability of ADAMTS13 testing has also made it possible to detect atypical presentations of TTP such as patients with macrovascular neurological symptoms without accompanying hematological findings as well as help diagnose other causes of thrombotic microangiopathies e.g. atypical hemolytic uremic syndrome. The use of ADAMTS13 testing in the management of TTP should continue to grow especially with newer assays with greater sensitivity, reproducibility, and timelier availability.
authors: Division of Transfusion Medicine and Hemostasis, Department of Pathology, UT Southwestern Medical Center, Dallas, TX 75390, USA.
authors: Neil
authors: N
authors: Shah
authors: Ravi
authors: R
authors: Sarode
mesh_terms: D051722:ADAM Proteins / Q000032:analysis* / Q000172:deficiency / Q000235:genetics / Q000276:immunology; D000071120:ADAMTS13 Protein; D058846:Antibodies, Monoclonal, Murine-Derived / Q000627:therapeutic use; D016477:Artifacts; D001323:Autoantibodies / Q000097:blood; D002986:Clinical Trials as Topic; D003131:Combined Modality Therapy; D006463:Hemolytic-Uremic Syndrome / Q000175:diagnosis; D006801:Humans; D010446:Peptide Fragments / Q000138:chemical synthesis / Q000378:metabolism; D010951:Plasma Exchange; D015539:Platelet Activation; D011499:Protein Processing, Post-Translational; D011697:Purpura, Thrombotic Thrombocytopenic / Q000175:diagnosis / Q000188:drug therapy / Q000235:genetics / Q000628:therapy*; D011994:Recombinant Proteins / Q000627:therapeutic use; D015203:Reproducibility of Results; D000069283:Rituximab; D012680:Sensitivity and Specificity; D014841:von Willebrand Factor / Q000378:metabolism
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D001323:Autoantibodies; D010446:Peptide Fragments; D011994:Recombinant Proteins; D014841:von Willebrand Factor; D000069283:Rituximab; D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human