Pathogenesis and treatment of acquired idiopathic thrombotic thrombocytopenic purpura

Haematologica

Volumn 95, Issue 9, 2010, Pages 1444-1447

  Peyvandi, Flora ^a   Palla, Roberta ^a   Lotta, Luca A ^a  

^a UNIVERSITY OF MILAN   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ACR 1779; AZATHIOPRINE; BLOOD CLOTTING INHIBITOR; CORTICOSTEROID; CYCLOSPORIN; RITUXIMAB; UNCLASSIFIED DRUG; VON WILLEBRAND FACTOR CLEAVING PROTEINASE;

ANEMIA; AUTOIMMUNE DISEASE; CLINICAL TRIAL; DISEASE COURSE; EDITORIAL; FEVER; HEMATURIA; HEMIPARESIS; HISTOPATHOLOGY; HUMAN; IDIOPATHIC THROMBOCYTOPENIC PURPURA; INCIDENCE; MORTALITY; PATHOGENESIS; PATHOPHYSIOLOGY; PLASMAPHERESIS; PROGNOSIS; THROMBOCYTOPENIA; THROMBOSIS; THROMBOTIC THROMBOCYTOPENIC PURPURA;

ADAM PROTEINS; AUTOANTIBODIES; EPITOPES; FEMALE; HUMANS; MALE; PURPURA, THROMBOTIC THROMBOCYTOPENIC;

EID: 77956791768     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2010.027169     Document Type: Editorial

References (42)

1. Moschcowitz E. Hyaline thrombosis of the terminal arterioles and capillaries: a hitherto undescribed disease. Proc N Y Pathol Soc. 1924;24:21-4.
2. Moake JL, Rudy CK, Troll JH, Weinstein MJ, Colannino NM, Azocar J, et al. Unusually large plasma factor VIII: von Willebrand factor multimers in chronic relapsing thrombotic thrombocytopenic purpura. N Engl J Med. 1982;307(23):1432-5.
3. Moake JL. Thrombotic microangiopathies. N Engl J Med. 2002; 347 (8):589-600.
4. Furlan M, Robles R, Solenthaler M, Wassmer M, Sandoz P, Lammle B. Deficient activity of von Willebrand factor-cleaving protease in chronic relapsing thrombotic thrombocytopenic purpura. Blood 1997;89:3097-103.
5. Gerritsen HE, Robles R, Lammle B, Furlan M. Partial amino acid sequence of purified von Willebrand factor-cleaving protease. Blood. 2001;98(9):1654-61.
6. 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):1662-6.
7. Zheng X, Chung D, Takayama TK, Majerus EM, Sadler JE, Fujikawa K. Structure of von Willebrand factor-cleaving protease (ADAMTS13), a metalloprotease involved in thrombotic thrombocytopenic purpura. J Biol Chem. 2001;276(44):41059-63.
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):11-9.
9. Furlan M, Robles R, Galbusera M, Remuzzi G, Kyrle PA, Brenner B, et al. von Willebrand factor-cleaving protease in thrombotic thrombocytopenic purpura and the hemolytic-uremic syndrome. N Engl J Med. 1998;339(22):1578-84.
10. Tsai HM, Lian EC. Antibodies to von Willebrand factor cleaving protease in acute thrombotic thrombocytopenic purpura. N Engl J Med. 1998;339:1585-94.
11. Tsai HM, Li A, Rock G. Inhibitors of von Willebrand factor-cleaving protease in thrombotic thrombocytopenic purpura. Clin Lab. 2001;47(7-8):387-92.
12. Shelat SG, Ai J, Zheng XL. Molecular biology of ADAMTS13 and diagnostic utility of ADAMTS13 proteolytic activity and inhibitor assays. Semin Thromb Hemost. 2005;31(6):659-72.
13. 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(6):1765-72.
14. Terrell DR, Williams LA, Vesely SK, Lämmle B, Hovinga JA, George JN. The incidence of thrombotic thrombocytopenic purpurahemolytic uremic syndrome: all patients, idiopathic patients, and patients with severe ADAMTS-13 deficiency. J Thromb Haemost. 2005;3(7):1432-6.
15. Zheng XL, Kaufman RM, Goodnough LT, Sadler JE. Effect of plasma exchange on plasma ADAMTS13 metalloprotease activity, inhibitor level, and clinical outcome in patients with idiopathic and nonidiopathic thrombotic thrombocytopenic purpura. Blood. 2004;103(11): 4043-9.
16. Hovinga JA, Vesely SK, Terrell DR, Lammle B, George JN. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood 2010;115(8):1500-11.
17. Shepard KV, Bukowski RM. The treatment of thrombotic thrombocytopenic purpura with exchange transfusions, plasma infusions, and plasma exchange. Semin Hematol. 1987;24(3):178-93.
18. Rock GA, Shumak KH, Buskard NA, Blanchette VS, Kelton JG, Nair RC, 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):393-7.
19. Bell WR, Braine HG, Ness PM, Kickler TS. Improved survival in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome. Clinical experience in 108 patients. N Engl J Med. 1991;325(6):398-403.
20. 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):133-41.
21. George JN. The thrombotic thrombocytopenic purpura and hemolytic uremic syndromes: evaluation, management, and long-term outcomes experience of the Oklahoma TTP-HUS Registry, 1989-2007. Kidney International. 2009; 5(Suppl 112): S52-S54.
22. Vesely SK, George JN, Lämmle B, Studt JD, Alberio L, El-Harake MA, et al. ADAMTS13 activity in thrombotic thrombocytopenic purpurahemolytic uremic syndrome: relation to presenting features and clinical outcomes in a prospective cohort of 142 patients. Blood. 2003;102(1):60-8.
23. Raife T, Atkinson B, Montgomery R, Vesely S, Friedman K. Severe deficiency of VWF-cleaving protease (ADAMTS13) activity defines a distinct population of thrombotic microangiopathy patients. Transfusion. 2004;44(2):146-50.
24. Coppo P, Bengoufa D, Veyradier A, Wolf M, Bussel A, Millot GA, et al. Severe ADAMTS13 deficiency in adult idiopathic thrombotic microangiopathies defines a subset of patients characterized by various autoimmune manifestations, lower platelet count, and mild renal involvement. Medicine. 2004;83(4):233-44.
25. Ferrari S, Scheiflinger F, Rieger M, Mudde G, Wolf M, Coppo P, et al. Prognostic value of anti-ADAMTS13 antibody features (Ig isotype, titer, and inhibitory effect) in a cohort of 35 adult French patients undergoing a first episode of thrombotic microangiopathy with undetectable ADAMTS13 activity. Blood. 2007;109(7):2815-22.
26. Peyvandi F, Lavoretano S, Palla R, Feys HB, Vanhoorelbeke K, Battaglioli T, et al. ADAMTS13 and anti-ADAMTS13 antibodies as markers for recurrence of acquired thrombotic thrombocytopenic purpura during remission. Haematologica. 2008;93(2):232-9.
27. Tsai HM, Raoufi M, Zhou W, Guinto E, Grafos N, Ranzurmal S, et al. ADAMTS13-binding IgG are present in patients with thrombotic thrombocytopenic purpura. Thromb Haemost. 2006;95(5):886-92.
28. Rieger M, Mannucci PM, Kremer Hovinga JA, Herzog A, Gerstenbauer G, Konetschny C, et al. ADAMTS13 autoantibodies in patients with thrombotic microangiopathies and other immunomediated diseases. Blood. 2005;106(4):1262-7.
29. Luken BM, Turenhout EA, Kaijen PH, Greuter MJ, Pos W, Van Mourik JA, et al. Amino acid regions 572-579 and 657-666 of the spacer domain of ADAMTS13 provide a common antigenic core required for binding of antibodies in patients with acquired TTP. Thromb Haemost. 2006;96(3):295-301.
30. Jin SY, Skipwith CG., Zheng XL. Amino acid residues Arg659, Arg660 and Tyr661 in the ADAMTS13 spacer domain are critical for recognition of von Willebrand factor. Blood. 2010;115(11):2300-10.
31. 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):1703-10.
32. Scheiflinger F, Knoebl P, Trattner B, Plaimauer B, Mohr G, Dockal M, et al. Nonneutralizing IgM and IgG antibodies to von Willebrand factor-cleaving protease (ADAMTS-13) in a patient with thrombotic thrombocytopenic purpura (TTP). Blood. 2003;102(9):3241-3.
33. Klaus C, Plaimauer B, Studt JD, Dorner F, Lammle B, Mannucci PM, et al. Epitope mapping of ADAMTS13 autoantibodies in acquired thrombotic thrombocytopenic purpura. Blood. 2004;103(12):4514-9.
34. Luken BM, Turenhout EA, Hulstein JJ, Van Mourik JA, Fijnheer R, Voorberg J. The spacer domain of ADAMTS13 contains a major binding site for antibodies in patients with thrombotic thrombocytopenic purpura. Thromb Haemost. 2005;93(2):267-74.
35. Zheng XL, Wu HM, Shang D, Falls E, Skipwith CG, Cataland SR, et al. Multiple domains of ADAMTS13 are targeted by autoantibodies against ADAMTS13 in patients with acquired idiopathic thrombotic thrombocytopenic purpura. Haematologica. 2010;95(9):1555-62.
36. Hughes C, McEwan JR, Longair I, Hughes S, Cohen H, Machin S, Scully M. Cardiac involvement in acute thrombotic thrombocytopenic purpura: association with troponin T and IgG antibodies to ADAMTS 13. J Thromb Haemost. 2009;7(4):529-36.
37. Studt JD, Kremer Hovinga JA, Radonic R, Gasparovic V, Ivanovic D, Merkler M, et al. Familial acquired thrombotic thrombocytopenic purpura: ADAMTS13 inhibitory autoantibodies in identical twins. Blood. 2004;103(11):4195-7.
38. Coppo P, Busson M, Veyradier A, Wynckel A, Poullin P, Azoulay E, et al. HLA-DRB1*11: a strong risk factor for acquired severe ADAMTS13 deficiency-related idiopathic thrombotic thrombocytopenic purpura in Caucasians. J Thromb Haemost. 2010;8(4):856-9.
39. Scully M, Brown J, Patel R, McDonald V, Brown CJ, Machin S. Human leukocyte antigen association in idiopathic thrombotic thrombocytopenic purpura: evidence for an immunogenetic link. J Thromb Haemost. 2010;8(2):257-62.
40. George JN. How I treat patients with thrombotic thrombocytopenic purpura-hemolytic uremic syndrome. Blood. 2000;96(4):1223-9.
41. Fakhouri F, Vernant JP, Veyradier A, Wolf M, Kaplanski G, Binaut R, et al. Efficiency of curative and prophylactic treatment with rituximab in ADAMTS13-deficient thrombotic thrombocytopenic purpura: a study of 11 cases. Blood. 2005;106(6):1932-7.
42. Scully M, Cohen H, Cavenagh J, Benjamin S, Starke R, Killick S, et al. Remission in acute refractory and relapsing thrombotic thrombocytopenic purpura following rituximab is associated with a reduction in IgG antibodies to ADAMTS-13. Br J Haematol. 2007;136(3):451-61.