Thrombotic thrombocytopenic purpura - Then and now

Seminars in Thrombosis and Hemostasis

Volumn 32, Issue 2, 2006, Pages 81-89

  Galbusera, Miriam ^a   Noris, Marina ^a   Remuzzi, Giuseppe ^a,b  

^a MARIO NEGRI INSTITUTE FOR PHARMACOLOGICAL RESEARCH   (Italy)

^b OSPEDALI RIUNITI   (Italy)

Author keywords

ADAMTS13; Endothelial damage; Hemolytic uremic syndrome; Thrombotic thrombocytopenic purpura; Von Willebrand factor

Indexed keywords

ANTINEOPLASTIC AGENT; BLEOMYCIN; CISPLATIN; CLOPIDOGREL; CYTARABINE; DAUNORUBICIN; IMMUNOGLOBULIN G; MITOMYCIN; NITRIC OXIDE; ORAL CONTRACEPTIVE AGENT; PLASMINOGEN ACTIVATOR INHIBITOR; PROSTACYCLIN; PROTEIN S; SIMVASTATIN; THROMBOMODULIN; TICLOPIDINE; VINBLASTINE; VON WILLEBRAND FACTOR; VON WILLEBRAND FACTOR CLEAVING PROTEINASE; ZOLEDRONIC ACID;

ARTICLE; BLEEDING; COMPLEMENT ACTIVATION; ENDOTHELIUM CELL; GENE MUTATION; HEMATURIA; HEMOLYTIC UREMIC SYNDROME; HUMAN; MEGAKARYOCYTE; MICROTHROMBUS; NEUROLOGIC DISEASE; PATHOGENESIS; PLASMA; PLASMAPHERESIS; PRIORITY JOURNAL; PROTEINURIA; RELAPSE; SHEAR STRESS; THROMBOCYTE ACTIVATION; THROMBOCYTE ADHESION; THROMBOCYTE AGGREGATION; THROMBOCYTOPENIA; THROMBOTIC THROMBOCYTOPENIC PURPURA;

ADAM PROTEINS; COMPLEMENT ACTIVATION; ENDOTHELIUM, VASCULAR; HISTORY, 20TH CENTURY; HISTORY, 21ST CENTURY; HUMANS; MOLECULAR WEIGHT; PLATELET ACTIVATION; PURPURA, THROMBOTIC THROMBOCYTOPENIC; VON WILLEBRAND FACTOR;

EID: 33645574296     PISSN: 00946176     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (66)

1. Moschcowitz E. Acute febrile pleiochromic anemia with hyaline thrombosis of a terminal arterioles and capillaries: an undescribed disease. Arch Intern Med 1925;36:89-93
2. Kennedy SS, Zacharski LR, Beck JR. Thrombotic thrombocytopenic purpura: analysis of 48 unselected cases. Semin Thromb Hemost 1980;6:341-349
3. Amorosi EL, Ultmann JE. Thrombotic thrombocytopenic purpura: report of 16 cases and review of the literature. Medicine 1966;45:139-159
4. Cuttner J. Thrombotic thrombocytopenic purpura: a ten-year experience. Blood 1980;56:302-306
5. Bukowski RM. Thrombotic thrombocytopenic purpura: a review. In: Spaet TH, ed. Progress in Hemostasis and Thrombosis. New York: Grune & Stratton; 1982:287-337
6. Ruggenenti P, Remuzzi G. Thrombotic thrombocytopenic purpura and related disorders. Hematol Oncol Clin North Am 1990;4:219-241
7. Silverstein A. Thrombotic thrombocytopenic purpura: the initial neurologic manifestations. Arch Neurol 1968;18:358-362
8. Percival SPB. Ocular findings in thrombotic thrombocytopenic purpura (Moschowitz's disease). Br J Ophthalmol 1970;54:73-78
9. Chen YC, McLeod B, Hall ER, Wu KK. Accelerated prostacyclin degradation in thrombotic thrombocytopenic purpura. Lancet 1981;2:267-269
10. Cuttner J. Chronic thrombotic thrombocytopenic purpura: report of a case with five relapses and review of the literature. Mt Sinai J Med 1978;45:418-425
11. Meacham GC, Orbison JL, Heinle RW, Steele HJ, Schaefer JA. Thrombotic thrombocytopenic purpura a disseminated disease of arterioles. Blood 1952;6:706-719
12. Fuchs WE, George JN, Dotin LN, Sears DA. Thrombotic thrombocytopenic purpura-occurrence two years apart during late pregnancy in two sisters. JAMA 1976;235:2126-2127
13. Hellman RM, Jackson DV, Buss DH. Thrombotic thrombocytopenic purpura and hemolytic uremic syndrome in HLA-identical siblings. Ann Intern Med 1980;93:283-284
14. Altschule MD. A rare type of acute thrombocytopenic purpura: widespread formation of platelet thrombi in capillaries. N Engl J Med 1942;227:477-479
15. Remuzzi G, Ruggenenti P, Bertani T. Thrombotic microangiopathies. In: Tisher CC, Brenner BM, eds. Renal Pathology. With Clinical and Functional Correlations. Philadelphia, PA: J.B. Lippincott; 1994:1154-1184
16. Ruiz-Torres MP, Casiraghi F, Galbusera M, et al. Complement activation: the missing link between ADAMTS-13 deficiency and microvascular thrombosis of thrombotic microangiopathies. Thromb Haemost 2005;93:443-452
17. Mitra D, Jaffe EA, Weksler B, Hajjar KA, Soderland C, Laurence J. Thrombotic thrombocytopenic purpura and sporadic hemolytic-uremic syndrome plasmas induce apoptosis in restricted lineages of human microvascular endothelial cells. Blood 1997;89:1224-1234
18. Neild GH. Mechanisms and models of endothelial injury. In: Bertani T, Remuzzi G, eds. Glomerular Injury 300 Years After Morgagni. Milan, Italy: Wichtig; 1983:139-162
19. Kniker WT, Cochrane CG. Pathogenetic factors in vascular lesions of experimental serum sickness. J Exp Med 1965;122:83-98
20. Kniker WT, Cochrane CG. The localization of circulating immune complexes in experimental serum sickness. The role of vasoactive amines and hydrodinamic forces. J Exp Med 1968;127:119-136
21. Lowenhaupt R, Nathan P. The participation of platelets in the rejection of dog kidney allotransplants: hematologic and electron microscopic studies. Transplant Proc 1969;1:305-310
22. Sharma HM, Moore S, Merrick HW, Smith MR. Platelet in early hyperacute allograft rejection in kidneys and their modification by sulfinpyrazone (Anturan) therapy: an experimental study. Am J Pathol 1972;66:445-460
23. Wall RT, Harker LA. The endothelium and thrombosis. Annu Rev Med 1980;31:361-371
24. Foster PA, Anderson JC. Effects of plasma from patients with thrombotic thrombocytopenic purpura (TTP) on cultured human endothelial cells (abst). Blood 1979;54:240a
25. Bennett CL, Weinberg PD, Rozenberg-Ben-Dror K, Yarnold PR, Kwaan HC, Green D. Thrombotic thrombocytopenic purpura associated with ticlopidine: a review of 60 cases. Ann Intern Med 1998;128:541-544
26. Bennett CL, Connors JM, Carwile JM, et al. Thrombotic thrombocytopenic purpura associated with clopidogrel. N Engl J Med 2000;342:1773-1777
27. Ferretti G, Petti MC, Carlini P, et al. Zoledronic acid-associated thrombotic thrombocytopenic purpura. Ann Oncol 2004;15:1847-1848
28. Sundram F, Roberts P, Kennedy B, Pavord S. Thrombotic thrombocytopenic purpura associated with statin treatment. Postgrad Med J 2004;80:551-552
29. Neame PD. Immunologic and other factors in thrombotic thrombocytopenic purpura (TTP). Semin Thromb Hemost 1980;6:416-429
30. Leaf AN, Laubenstein LJ, Raphael B, Hochster H, Baez L, Karpatkin S. Thrombotic thrombocytopenic purpura associated with human immunodeficiency virus type 1 (HIV-1) infection. Ann Intern Med 1988;109:194-197
31. Ruggenenti P, Remuzzi G. The pathophysiology and management of thrombotic thrombocytopenic purpura. Eur J Haematol 1996;56:191-207
32. Lian ECY, Harkness DR, Byrnes JJ, Wallach H, Nunez R. Presence of a platelet aggregating factor in the plasma of patients with thrombotic thrombocytopenic purpura (TTP) and its inhibition by normal plasma. Blood 1979;53:333-338
33. Lian ECY, Siddiqui FA, Chen SH, Feng LM. Platelet-agglutinating/ aggregating proteins from the plasma of patients with thrombotic thrombocytopenic purpura. In: Kaplan BS, Trompeter RS, Moake JL, eds. HUS and TTP. New York: Marcel Dekker; 1992:473-481
34. Moake JL, Rudy CK, Troll JH, et al. Unusually large plasma factor VIII: von Willebrand factor multimers in chronic relapsing thrombotic thrombocytopenic purpura. N Engl J Med 1982;307:1432-1435
35. Dent JA, Galbusera M, Ruggeri ZM. Heterogeneity of plasma von Willebrand factor multimers resulting from proteolysis of constituent subunit. J Clin Invest 1991;88:774-782
36. Moake JL, McPherson PD. Abnormalities of von Willebrand factor multimers in thrombotic thrombocytopenic purpura and the hemolytic-uremic syndrome. Am J Med 1989;87:9N-15N
37. Moake JL. Moschcowitz, multimers, and metalloprotease. N Engl J Med 1998;339:1629-1631
38. Moake JL. Hemolytic-uremic syndrome: basic science. Lancet 1994;343:393-397
39. Tsai HM, Sussman II, Nagel RL. Shear stress enhances the proteolysis of von Willebrand factor in normal plasma. Blood 1994;83:2171-2179
40. 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;339:1578-1584
41. Tsai HM, Lia ECY. Antibodies to von Willebrand factor-cleaving protease in acute thrombotic thrombocytopenic purpura. N Engl J Med 1998;339:1585-1594
42. Soejima K, Nakagaki T. Interplay between ADAMTS13 and von Willebrand factor in inherited and acquired thrombotic microangiopathies. Semin Hematol 2005;42:56-62
43. Levy GG, Nichols WC, Lian EC, et al. Mutations in a member of the ADAMTS gene family cause thrombotic thrombocytopenic purpura. Nature 2001;413:488-494
44. Bork P, Beckmann G. The CUB domain. A widespread module in developmentally regulated proteins. J Mol Biol 1993;231:539-545
45. Bornstein P. Thrombospondin as matricellular modulators of cell function. J Clin Invest 2001;107:929-934
46. Ruoslahti E, Pierschbacher MD. Arg-Gly-Asp: a versatile cell recognition signal. Cell 1986;44:517-518
47. Upshaw JD. Congenital deficiency of a factor in normal plasma that reverses microangiopathic hemolysis and thrombocytopenia. N Engl J Med 1978;298:1350-1352
48. Moake JL. Thrombotic microangiopathies. N Engl J Med 2002;347:589-600
49. Veyradier A, Obert B, Houiller A, Meyer D, Girma JP. Specific von Willebrand factor-cleaving protease in thrombotic microangiopathies: a study of 111 cases. Blood 2001;98:1765-1772
50. Furlan M, Robles R, Solenthaler M, Lämmle B. Acquired deficiency of von Willebrand factor-cleaving protease in a patient with thrombotic thrombocytopenic purpura. Blood 1998;91:2839-2846
51. Kokame K, Matsumoto M, Soejima K, et al. Mutations and common polymorphisms in ADAMTS13 gene responsible for von Willebrand factor-cleaving protease activity. Proc Natl Acad Sci USA 2002;99:11902-11907
52. Matsumoto M, Kokame K, Soejima K, et al. Molecular characterization of ADAMTS13 gene mutations in Japanese patients with Upshaw-Schulman syndrome. Blood 2004;103:1305-1310
53. Uchida T, Wada H, Mizutani M, et al. Identification of novel mutations in ADAMTS13 in an adult patient with congenital thrombotic thrombocytopenic purpura. Blood 2004;104:2081-2083
54. Klaus C, Plaimauer B, Studt JD, et al. Epitope mapping of ADAMTS13 autoantibodies in acquired thrombotic thrombocytopenic purpura. Blood 2004;103:4514-4519
55. Remuzzi G, Galbusera M, Noris M, et al. von Willebrand factor cleaving protease (ADAMTS13) is deficient in recurrent and familial thrombotic thrombocytopenic purpura and haemolytic uremic syndrome. Blood 2002;100:778-785
56. Soejima K, Matsumoto M, Kokame K, et al. ADAMTS-13 cysteine-rich/spacer domains are functionally essential for von Willebrand factor cleavage. Blood 2003;102:3232-3237
57. Galbusera M, Noris M, Rossi C, et al. Increased fragmentation of von Willebrand factor, due to abnormal cleavage of the subunit, parallels disease activity in recurrent hemolytic uremic syndrome and thrombotic thrombocytopenic purpura and discloses predisposition in families. The Italian Registry of Familial and Recurrent HUS/TTP. Blood 1999;94:610-620
58. Vesely SK, George JN, Lämmle B, et al. ADAAdTS13 activity in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome: relation to presenting features and clinical outcomes in a prospective cohort of 142 patients. Blood 2003;102:60-68
59. Tsai HM. Is severe deficiency of ADAMTS-13 specific for thrombotic thrombocytopenic purpura? Yes. J Thromb Haemost 2003;1:625-631
60. Remuzzi G. Is ADAMTS-13 deficiency specific for thrombotic thrombocytopenic purpura? No. J Thromb Haemost 2003;1:632-634
61. Noris M, Bucchioni S, Galbusera M, et al. Complement factor H mutation in familial thrombotic thrombocytopenic purpura with ADAMTS13 deficiency and renal involvement. J Am Soc Nephrol 2005;16:1177-1183
62. Dong JF, Moake JL, Nolasco L, et al. ADAMTS-13 rapidly cleaves newly secreted ultralarge von Willebrand factor multimers on the endothelial surface under flowing conditions. Blood 2002;100:4033-4039
63. Del Conde I, Cruz MA, Zhang H, Lopez JA, Afshar-Kharghan V. Platelet activation leads to activation and propagation of the complement system. J Exp Med 2005;201:871-879
64. Wright JF, Wang H, Horsnstein A, et al. Characterization of platelet glycoproteins and platelet/endothelial cell antibodies in patients with thrombotic thrombocytopenic purpura. Br J Haematol 1999;107:546-555
65. Yomtovian R, Niklinski W, Silver B, Sarode R, Tsai H-M. Rituximab for chronic recurring thrombotic thrombocytopenic purpura: a case report and review of the literature. Br J Haematol 2004;124:787-795
66. Galbusera M, Moris M, Gastoldi S, et al. Rituximab prevents recurrence of thrombotic thrombocytopenic purpura: a case report. Blood 2005;106:925-928