1. Scully M, Yarranton H, Liesner R, et al. Regional UK TTP registry: correlation with laboratory ADAMTS 13 analysis and clinical features. Br J Haematol. 2008;142(5):819–826. [PubMed] [Google Scholar] 2. Furlan M, Robles R, Galbusera M, et al. von Willebrand factor-cleaving protease in thrombotic thrombocytopenic purpura and the hemolytic-uremic syndrome. N Engl J Med. 1998;339(22):1578–1584. [PubMed] [Google Scholar] 3. Fujikawa K, Suzuki H, McMullen B, Chung D. Purification of human von Willebrand factor-cleaving protease and its identification as a new member of the metalloproteinase family. Blood. 2001;98(6):1662–1666. [PubMed] [Google Scholar] 4. Ferrari S, Mudde GC, Rieger M, Veyradier A, Kremer Hovinga JA, Scheiflinger F. IgG subclass distribution of anti-ADAMTS13 antibodies in patients with acquired thrombotic thrombocytopenic purpura. J Thromb Haemost. 2009;7(10):1703–1710. [PubMed] [Google Scholar] 5. Yamaguchi Y, Moriki T, Igari A, et al. Epitope analysis of autoantibodies to ADAMTS13 in patients with acquired thrombotic thrombocytopenic purpura. Thromb Res. 2011;128(2):169–173. [PubMed] [Google Scholar] 6. Klaus C, Plaimauer B, Studt JD, et al. Epitope mapping of ADAMTS13 autoantibodies in acquired thrombotic thrombocytopenic purpura. Blood. 2004;103(12):4514–4519. [PubMed] [Google Scholar] 7. Pos W, Crawley JT, Fijnheer R, Voorberg J, Lane DA, Luken BM. An autoantibody epitope comprising residues R660, Y661, and Y665 in the ADAMTS13 spacer domain identifies a binding site for the A2 domain of VWF. Blood. 2010;115(8):1640–1649. [PMC free article] [PubMed] [Google Scholar] 8. Lotta LA, Garagiola I, Palla R, Cairo A, Peyvandi F. ADAMTS13 mutations and polymorphisms in congenital thrombotic thrombocytopenic purpura. Hum Mutat. 2010;31(1):11–19. [PubMed] [Google Scholar] 9. Moschcowitz E. An Acute Febrile Pleiochromic Anemia with Hyaline Thrombosis of the Terminal Arterioles and Capillaries – an undescribed disease. Arch Int Med. 1925;36:89–93. [Google Scholar] 10. Mackie I, Langley K, Chitolie A, et al. Discrepancies between ADAMTS13 activity assays in patients with thrombotic microangiopathies. Thromb Haemost. 2013;109(3):488–496. [PubMed] [Google Scholar] 11. Starke R, Machin S, Scully M, Purdy G, Mackie I. The clinical utility of ADAMTS13 activity, antigen and autoantibody assays in thrombotic thrombocytopenic purpura. Br J Haematol. 2007;136(4):649–655. [PubMed] [Google Scholar] 12. Pereira A, Mazzara R, Monteagudo J, et al. Thrombotic thrombocytopenic purpura/hemolytic uremic syndrome: a multivariate analysis of factors predicting the response to plasma exchange. Ann Hematol. 1995;70(6):319–323. [PubMed] [Google Scholar] 13. Rock GA, Shumak KH, Buskard NA, et al. Comparison of plasma exchange with plasma infusion in the treatment of thrombotic thrombocytopenic purpura. Canadian Apheresis Study Group. N Engl J Med. 1991;325(6):393–397. [PubMed] [Google Scholar] 14. Bandarenko N, Brecher ME. United States Thrombotic Thrombocytopenic Purpura Apheresis Study Group (US TTP ASG): multicenter survey and retrospective analysis of current efficacy of therapeutic plasma exchange. J Clin Apher. 1998;13(3):133–141. [PubMed] [Google Scholar] 15. Rock G, Shumak KH, Sutton DM, Buskard NA, Nair RC. Cryosupernatant as replacement fluid for plasma exchange in thrombotic thrombocytopenic purpura. Members of the Canadian Apheresis Group. Br J Haematol. 1996;94(2):383–386. [PubMed] [Google Scholar] 16. Zeigler ZR, Shadduck RK, Gryn JF, et al.North American TTP Group Cryoprecipitate poor plasma does not improve early response in primary adult thrombotic thrombocytopenic purpura (TTP) J Clin Apher. 2001;16(1):19–22. [PubMed] [Google Scholar] 17. Rock G, Anderson D, Clark W, et al.Canadian Apheresis Group. Canadian Association of Apheresis Nurses Does cryosupernatant plasma improve outcome in thrombotic thrombocytopenic purpura? No answer yet. Br J Haematol. 2005;129(1):79–86. [PubMed] [Google Scholar] 18. Hellstern P, Solheim BG. The Use of Solvent/Detergent Treatment in Pathogen Reduction of Plasma. Transfus Med Hemother. 2011;38(1):65–70. [PMC free article] [PubMed] [Google Scholar] 19. Scully M, Longair I, Flynn M, Berryman J, Machin SJ. Cryosupernatant and solvent detergent fresh-frozen plasma (Octaplas) usage at a single centre in acute thrombotic thrombocytopenic purpura. Vox Sang. 2007;93(2):154–158. [PubMed] [Google Scholar] 20. Lawrie AS, Green L, Canciani MT, et al. The effect of prion reduction in solvent/detergent-treated plasma on haemostatic variables. Vox Sang. 2010;99(3):232–238. [PubMed] [Google Scholar] 21. Jilma-Stohlawetz P, Kursten FW, Horvath M, et al. Recovery, safety, and tolerability of a solvent/detergent-treated and prion-safeguarded transfusion plasma in a randomized, crossover, clinical trial in healthy volunteers. Transfusion. 2013;53(9):1906–1917. [PubMed] [Google Scholar] 22. Scully M, McDonald V, Cavenagh J, et al. A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura. Blood. 2011;118(7):1746–1753. [PubMed] [Google Scholar] 23. McDonald V, Manns K, Mackie IJ, Machin SJ, Scully MA. Rituximab pharmacokinetics during the management of acute idiopathic thrombotic thrombocytopenic purpura. J Thromb Haemost. 2010;8(6):1201–1208. [PubMed] [Google Scholar] 24. Froissart A, Buffet M, Veyradier A, et al.French Thrombotic Microangiopathies Reference Center Efficacy and safety of first-line rituximab in severe, acquired thrombotic thrombocytopenic purpura with a suboptimal response to plasma exchange. Experience of the French Thrombotic Microangiopathies Reference Center. Crit Care Med. 2012;40(1):104–111. [PubMed] [Google Scholar] 25. Westwood JP, Webster H, McGuckin S, McDonald V, Machin SJ, Scully M. Rituximab for thrombotic thrombocytopenic purpura: benefit of early administration during acute episodes and use of prophylaxis to prevent relapse. J Thromb Haemost. 2013;11(3):481–490. [PubMed] [Google Scholar] 26. Bresin E, Gastoldi S, Daina E, et al. Rituximab as pre-emptive treatment in patients with thrombotic thrombocytopenic purpura and evidence of anti-ADAMTS13 autoantibodies. Thromb Haemost. 2009;101(2):233–238. [PubMed] [Google Scholar] 27. Balduini CL, Gugliotta L, Luppi M, et al.Italian TTP Study Group High versus standard dose methylprednisolone in the acute phase of idiopathic thrombotic thrombocytopenic purpura: a randomized study. Ann Hematol. 2010;89(6):591–596. [PubMed] [Google Scholar] 28. Bobbio-Pallavicini E, Gugliotta L, Centurioni R, et al. Antiplatelet agents in thrombotic thrombocytopenic purpura (TTP). Results of a randomized multicenter trial by the Italian Cooperative Group for TTP. Haematologica. 1997;82(4):429–435. [PubMed] [Google Scholar] 29. Hart D, Sayer R, Miller R, et al. Human immunodeficiency virus associated thrombotic thrombocytopenic purpura – favourable outcome with plasma exchange and prompt initiation of highly active antiretroviral therapy. Br J Haematol. 2011;153(4):515–519. [PubMed] [Google Scholar] 30. Plaimauer B, Kremer Hovinga JA, Juno C, et al. Recombinant ADAMTS13 normalizes von Willebrand factor-cleaving activity in plasma of acquired TTP patients by overriding inhibitory antibodies. J Thromb Haemost. 2011;9(5):936–944. [PubMed] [Google Scholar] 31. Jian C, Xiao J, Gong L, et al. Gain-of-function ADAMTS13 variants that are resistant to autoantibodies against ADAMTS13 in patients with acquired thrombotic thrombocytopenic purpura. Blood. 2012;119(16):3836–3843. [PMC free article] [PubMed] [Google Scholar] 32. Ulrichts H, Silence K, Schoolmeester A, et al. Antithrombotic drug candidate ALX-0081 shows superior preclinical efficacy and safety compared with currently marketed antiplatelet drugs. Blood. 2011;118(3):757–765. [PubMed] [Google Scholar] 33. Callewaert F, Roodt J, Ulrichts H, et al. Evaluation of efficacy and safety of the anti-VWF Nanobody ALX-0681 in a preclinical baboon model of acquired thrombotic thrombocytopenic purpura. Blood. 2012;120(17):3603–3610. [PubMed] [Google Scholar] 34. Cataland SR, Peyvandi F, Mannucci PM, et al. Initial experience from a double-blind, placebo-controlled, clinical outcome study of ARC1779 in patients with thrombotic thrombocytopenic purpura. Am J Hematol. 2012;87(4):430–432. [PubMed] [Google Scholar] 35. Chen J, Reheman A, Gushiken FC, et al. N-acetylcysteine reduces the size and activity of von Willebrand factor in human plasma and mice. J Clin Invest. 2011;121(2):593–603. [PMC free article] [PubMed] [Google Scholar] 36. Chang BY, Huang MM, Francesco M, et al. The Bruton tyrosine kinase inhibitor PCI-32765 ameliorates autoimmune arthritis by inhibition of multiple effector cells. Arthritis Res Ther. 2011;13(4):R115. [PMC free article] [PubMed] [Google Scholar] 37. Honigberg LA, Smith AM, Sirisawad M, et al. The Bruton tyrosine kinase inhibitor PCI-32765 blocks B-cell activation and is efficacious in models of autoimmune disease and B-cell malignancy. Proc Natl Acad Sci U S A. 2010;107(29):13075–13080. [PMC free article] [PubMed] [Google Scholar] 38. Shortt J, Oh DH, Opat SS. ADAMTS13 antibody depletion by bortezomib in thrombotic thrombocytopenic purpura. N Engl J Med. 2013;368(1):90–92. [PubMed] [Google Scholar] 39. van Balen T, Schreuder MF, de Jong H, van de Kar NC. Refractory thrombotic thrombocytopenic purpura in a 16-year-old girl: successful treatment with bortezomib. Eur J Haematol. 2014;92(1):80–82. [PubMed] [Google Scholar] Page 2Genetic mutations and sites of autoantibody specificity in congenital and acquired TTP. Abbreviations: TTP, thrombotic thrombocytopenic purpura; TSP, thrombospondin-type 1 repeats; CUB, complement c1r/c1s, sea Urchin epidermal growth factor, and Bone morphogenetic protein. |