title: ADAMTS13 and anti-ADAMTS13 autoantibodies in thrombotic thrombocytopenic purpura - current perspectives and new treatment strategies.
abstract: A deficiency in ADAMTS13 (A Disintegrin And Metalloprotease with ThromboSpondin type-1 repeats, member 13) is associated with thrombotic thrombocytopenic purpura (TTP). Congenital TTP is caused by a defect in the ADAMTS13 gene resulting in decreased or absent enzyme activity; acquired TTP results from autoantibodies that either inhibit the activity or increase the clearance of ADAMTS13. Despite major progress in recent years in our understanding of the disease, many aspects around the pathophysiology of TTP are still unclear. Newer studies expanded the TTP field from ADAMTS13 and inhibitory antibodies to immune complexes, cloned autoantibodies, and a possible involvement of other proteases. Additionally, several new treatment strategies supplementing plasma-exchange and infusion are under investigation for a better and more specific treatment of TTP patients. In this review, we discuss the recent insights in TTP pathophysiology and describe upcoming therapeutic opportunities.
authors.affiliation: a Laboratory for Thrombosis Research, IRF Life Sciences , KU Leuven Campus Kulak Kortrijk , Kortrijk , Belgium.
authors.forename: Claudia
authors.initials: C
authors.lastname: Tersteeg
authors.affiliation: a Laboratory for Thrombosis Research, IRF Life Sciences , KU Leuven Campus Kulak Kortrijk , Kortrijk , Belgium.
authors.forename: Sebastien
authors.initials: S
authors.lastname: Verhenne
authors.affiliation: a Laboratory for Thrombosis Research, IRF Life Sciences , KU Leuven Campus Kulak Kortrijk , Kortrijk , Belgium.
authors.forename: Elien
authors.initials: E
authors.lastname: Roose
authors.affiliation: a Laboratory for Thrombosis Research, IRF Life Sciences , KU Leuven Campus Kulak Kortrijk , Kortrijk , Belgium.
authors.forename: An-Sofie
authors.initials: AS
authors.lastname: Schelpe
authors.affiliation: a Laboratory for Thrombosis Research, IRF Life Sciences , KU Leuven Campus Kulak Kortrijk , Kortrijk , Belgium.
authors.forename: Hans
authors.initials: H
authors.lastname: Deckmyn
authors.affiliation: a Laboratory for Thrombosis Research, IRF Life Sciences , KU Leuven Campus Kulak Kortrijk , Kortrijk , Belgium.
authors.forename: Simon F
authors.initials: SF
authors.lastname: De Meyer
authors.affiliation: a Laboratory for Thrombosis Research, IRF Life Sciences , KU Leuven Campus Kulak Kortrijk , Kortrijk , Belgium.
authors.forename: Karen
authors.initials: K
authors.lastname: Vanhoorelbeke
mesh_terms: D051722:ADAM Proteins / Q000737:chemistry / Q000235:genetics / Q000276:immunology* / Q000378:metabolism; D000071120:ADAMTS13 Protein; D000818:Animals; D000936:Antigen-Antibody Complex / Q000097:blood / Q000276:immunology; D001323:Autoantibodies / Q000145:classification / Q000235:genetics / Q000276:immunology*; D003001:Cloning, Molecular; D003131:Combined Modality Therapy; D018604:Epitope Mapping; D000939:Epitopes / Q000276:immunology; D006801:Humans; D009154:Mutation; D011485:Protein Binding / Q000187:drug effects; D011697:Purpura, Thrombotic Thrombocytopenic / Q000151:congenital / Q000276:immunology* / Q000628:therapy; D014841:von Willebrand Factor / Q000737:chemistry / Q000378:metabolism
chemical_list: D000936:Antigen-Antibody Complex; D001323:Autoantibodies; D000939:Epitopes; D014841:von Willebrand Factor; D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human
keywords: ADAMTS13; acquired TTP; autoantibodies; congenital TTP; immune complexes; proteases; thrombotic thrombocytopenic purpura (TTP); von Willebrand factor