Erratum: Sayani FA, Abrams CS. How I treat refractory thrombotic thrombocytopenic purpura (Blood (2015) 125:25 (3860-3867) DOI: 10.1182/blood-2014-11-551580);How I treat refractory thrombotic thrombocytopenic purpura

Blood

Volumn 125, Issue 25, 2015, Pages 3860-3867

  Sayani, Farzana A ^a   Abrams, Charles S ^a,b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACETYLCYSTEINE; ALX 0681; BORTEZOMIB; CORTICOSTEROID; CREATININE; CYCLOPHOSPHAMIDE; CYCLOSPORIN; DISINTEGRIN; ECULIZUMAB; EGAPTIVON PEGOL; GBR 600; HEMOGLOBIN; IMMUNOSUPPRESSIVE AGENT; LACTATE DEHYDROGENASE; METALLOPROTEINASE; METHYLPREDNISOLONE; MONOCLONAL ANTIBODY; PREDNISONE; RECOMBINANT ENZYME; RECOMBINANT VON WILLEBRAND FACTOR CLEAVING PROTEINASE; RITUXIMAB; THROMBOSPONDIN 1; UNCLASSIFIED DRUG; VINCRISTINE; VON WILLEBRAND FACTOR ANTIBODY; VON WILLEBRAND FACTOR CLEAVING PROTEINASE;

ABDOMINAL PAIN; ABDOMINAL TENDERNESS; ACQUIRED THROMBOTIC THROMBOCYTOPENIC PURPURA; ADULT; ARTICLE; BLOOD SAMPLING; BLOOD SMEAR; CASE REPORT; CORTICOSTEROID THERAPY; DRUG MEGADOSE; ERYTHROCYTE; FATIGUE; FEMALE; FEVER; HUMAN; HUMAN CELL; IMMUNOSUPPRESSIVE TREATMENT; NAUSEA; NONHUMAN; PLASMAPHERESIS; PRIORITY JOURNAL; PURPURA; REFRACTORY THROMBOTIC THROMBOCYTOPENIC PURPURA; RETICULOCYTE; SKIN BRUISING; THROMBOCYTE; THROMBOCYTOPENIA; THROMBOTIC THROMBOCYTOPENIC PURPURA; DRUG RESISTANCE; PURPURA, THROMBOTIC THROMBOCYTOPENIC;

ADULT; DRUG RESISTANCE; FEMALE; HUMANS; PURPURA, THROMBOTIC THROMBOCYTOPENIC;

EID: 84929994371     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2017-08-803171     Document Type: Erratum

References (87)

1. Rock GA, Shumak KH, Buskard NA, et al Canadian Apheresis Study Group. Comparison of plasma exchange with plasma infusion in the treatment of thrombotic thrombocytopenic purpura. N Engl J Med. 1991;325(6):393-397.
2. 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.
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.
4. Levy GG, Nichols WC, Lian EC, et al. Mutations in a member of the ADAMTS gene family cause thrombotic thrombocytopenic purpura. Nature. 2001;413(6855):488-494.
5. Tsai HM, Lian EC. Antibodies to von Willebrand factor-cleaving protease in acute thrombotic thrombocytopenic purpura. N Engl J Med. 1998;339(22):1585-1594.
6. Scheiflinger F, Knöbl P, Trattner B, et al. Nonneutralizing IgM and IgG antibodies to von Willebrand factor-cleaving protease (ADAMTS-13) in a patient with thrombotic thrombocytopenic purpura. Blood. 2003;102(9):3241-3243.
7. 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(8):2839-2846.
8. Furlan M, Lämmle B. Deficiency of von Willebrand factor-cleaving protease in familial and acquired thrombotic thrombocytopenic purpura. Baillieres Clin Haematol. 1998;11(2):509-514.
9. Scully M, Goodship T. How I treat thrombotic thrombocytopenic purpura and atypical haemolytic uraemic syndrome. Br J Haematol. 2014;164(6):759-766.
10. George JN. How I treat patients with thrombotic thrombocytopenic purpura: 2010. Blood. 2010;116(20):4060-4069.
11. Hughes C, McEwan JR, Longair I, et al. Cardiac involvement in acute thrombotic thrombocytopenic purpura: association with troponin T and IgG antibodies to ADAMTS 13. J Thromb Haemost. 2009;7(4):529-536.
12. Coppo P, Veyradier A. Thrombotic microangiopathies: towards a pathophysiology-based classification. Cardiovasc Hematol Disord Drug Targets. 2009;9(1):36-50.
13. Scully M, Hunt BJ, Benjamin S, et al British Committee for Standards in Haematology. Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies. Br J Haematol. 2012;158(3):323-335.
14. Peyvandi F, Ferrari S, Lavoretano S, Canciani MT, Mannucci PM. von Willebrand factor cleaving protease (ADAMTS-13) and ADAMTS-13 neutralizing autoantibodies in 100 patients with thrombotic thrombocytopenic purpura. Br J Haematol. 2004;127(4):433-439.
15. Coppo P, Wolf M, Veyradier A, et al; Réseau d'Etude des Microangiopathies Thrombotiques de l'Adulte. Prognostic value of inhibitory anti-ADAMTS13 antibodies in adult-acquired thrombotic thrombocytopenic purpura. Br J Haematol. 2006;132(1):66-74.
16. Bianchi V, Robles R, Alberio L, Furlan M, Lämmle B. Von Willebrand factor-cleaving protease (ADAMTS13) in thrombocytopenic disorders: a severely deficient activity is specific for thrombotic thrombocytopenic purpura. Blood. 2002;100(2):710-713.
17. Wu N, Liu J, Yang S, et al. Diagnostic and prognostic values of ADAMTS13 activity measured during daily plasma exchange therapy in patients with acquired thrombotic thrombocytopenic purpura. Transfusion. 2015;55(1):18-24.
18. Loof AH, van Vliet HH, Kappers-Klunne MC. Low activity of von Willebrand factor-cleaving protease is not restricted to patients suffering from thrombotic thrombocytopenic purpura. Br J Haematol. 2001;112(4):1087-1088.
19. Moore JC, Hayward CP, Warkentin TE, Kelton JG. Decreased von Willebrand factor protease activity associated with thrombocytopenic disorders. Blood. 2001;98(6):1842-1846.
20. Kremer Hovinga JA, Vesely SK, Terrell DR, Lämmle B, George JN. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2010;115(8):1500-1511; quiz 1662.
21. Sarode R, Bandarenko N, Brecher ME, et al. Thrombotic thrombocytopenic purpura: 2012 American Society for Apheresis (ASFA) consensus conference on classification, diagnosis, management, and future research. J Clin Apher. 2014;29(3):148-167.
22. Coppo P, Bussel A, Charrier S, et al. High-dose plasma infusion versus plasma exchange as early treatment of thrombotic thrombocytopenic purpura/hemolytic-uremic syndrome. Medicine (Baltimore). 2003;82(1):27-38.
23. 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):4043-4049.
24. Nguyen L, Li X, Duvall D, Terrell DR, Vesely SK, George JN. Twice-daily plasma exchange for patients with refractory thrombotic thrombocytopenic purpura: the experience of the Oklahoma Registry, 1989 through 2006. Transfusion. 2008;48(2):349-357.
25. 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.
26. Blitzer JB, Granfortuna JM, Gottlieb AJ, et al. Thrombotic thrombocytopenic purpura: treatment with plasmapheresis. Am J Hematol. 1987;24(4):329-339.
27. Roberts AW, Gillett EA, Fleming SJ. Hemolytic uremic syndrome/thrombotic thrombocytopenic purpura: outcome with plasma exchange. J Clin Apher. 1991;6(3):150-154.
28. Onundarson PT, Rowe JM, Heal JM, Francis CW. Response to plasma exchange and splenectomy in thrombotic thrombocytopenic purpura. A 10-year experience at a single institution. Arch Intern Med. 1992;152(4):791-796.
29. Shah N, Rutherford C, Matevosyan K, Shen Y-M, Sarode R. Role of ADAMTS13 in the management of thrombotic microangiopathies including thrombotic thrombocytopenic purpura (TTP). Br J Haematol. 2013;163(4):514-519.
30. Toyoshige M, Zaitsu Y, Okafuji K, et al. Successful treatment of thrombotic thrombocytopenic purpura with high-dose corticosteroid. Am J Hematol. 1992;41(1):69.
31. 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.
32. Franchin G, Diamond B. Pulse steroids: how much is enough? Autoimmun Rev. 2006;5(2):111-113.
33. Rock G, Shumak KH, Sutton DM, Buskard NA, Nair RC; Members of the Canadian Apheresis Group. Cryosupernatant as replacement fluid for plasma exchange in thrombotic thrombocytopenic purpura. Br J Haematol. 1996;94(2):383-386.
34. Fakhouri F, Vernant J-P, Veyradier A, 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):1932-1937.
35. Caramazza D, Quintini G, Abbene I, et al. Rituximab for managing relapsing or refractory patients with idiopathic thrombotic thrombocytopenic purpura - haemolytic uraemic syndrome. Blood Transfus. 2010;8(3):203-210.
36. Scully M, Cohen H, Cavenagh J, 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):451-461.
37. Chemnitz J, Draube A, Scheid C, et al. Successful treatment of severe thrombotic thrombocytopenic purpura with the monoclonal antibody rituximab. Am J Hematol. 2002;71(2):105-108.
38. Ahmad A, Aggarwal A, Sharma D, et al. Rituximab for treatment of refractory/relapsing thrombotic thrombocytopenic purpura (TTP). Am J Hematol. 2004;77(2):171-176.
39. Ling HT, Field JJ, Blinder MA. Sustained response with rituximab in patients with thrombotic thrombocytopenic purpura: a report of 13 cases and review of the literature. Am J Hematol. 2009;84(7):418-421.
40. de la Rubia J, Moscardó F, Gómez MJ, et al Grupo Español de Aféresis. Efficacy and safety of rituximab in adult patients with idiopathic relapsing or refractory thrombotic thrombocytopenic purpura: results of a Spanish multicenter study. Transfus Apheresis Sci. 2010;43(3):299-303.
41. Chemnitz JM, Uener J, Hallek M, Scheid C. Long-term follow-up of idiopathic thrombotic thrombocytopenic purpura treated with rituximab. Ann Hematol. 2010;89(10):1029-1033.
42. Jasti S, Coyle T, Gentile T, Rosales L, Poiesz B. Rituximab as an adjunct to plasma exchange in TTP: a report of 12 cases and review of literature. J Clin Apher. 2008;23(5):151-156.
43. Lim W, Vesely SK, George JN. The role of rituximab in the management of patients with acquired thrombotic thrombocytopenic purpura. Blood. 2015;125(10):1526-1531.
44. Froissart A, Buffet M, Veyradier A, et al French Thrombotic Microangiopathies Reference Center; Experience of the 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. Crit Care Med. 2012;40(1):104-111.
45. 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.
46. Aqui NA, Stein SH, Konkle BA, Abrams CS, Strobl FJ. Role of splenectomy in patients with refractory or relapsed thrombotic thrombocytopenic purpura. J Clin Apher. 2003;18(2):51-54.
47. Kappers-Klunne MC, Wijermans P, Fijnheer R, et al. Splenectomy for the treatment of thrombotic thrombocytopenic purpura. Br J Haematol. 2005;130(5):768-776.
48. Dubois L, Gray DK. Case series: splenectomy: does it still play a role in the management of thrombotic thrombocytopenic purpura? Can J Surg. 2010;53(5):349-355.
49. Kierdorf H, Maurin N, Heintz B. Cyclosporine for thrombotic thrombocytopenic purpura. Ann Intern Med. 1993;118(12):987-988.
50. Bachman WR, Brennan JK. Refractory thrombotic thrombocytopenic purpura treated with cyclosporine. Am J Hematol. 1996;51(1):93-94.
51. Pasquale D, Vidhya R, DaSilva K, Tsan MF, Lansing L, Chikkappa G. Chronic relapsing thrombotic thrombocytopenic purpura: role of therapy with cyclosporine. Am J Hematol. 1998; 57(1):57-61.
52. Jhaveri KD, Scheuer A, Cohen J, Gordon B. Treatment of refractory thrombotic thrombocytopenic purpura using multimodality therapy including splenectomy and cyclosporine. Transfus Apheresis Sci. 2009;41(1):19-22.
53. Cataland SR, Jin M, Lin S, et al. Cyclosporin and plasma exchange in thrombotic thrombocytopenic purpura: long-term follow-up with serial analysis of ADAMTS13 activity. Br J Haematol. 2007;139(3):486-493.
54. Hertzberg MS, Koutts J. Oral cyclophosphamide for refractory or relapsing thrombotic thrombocytopenic purpura (TTP). Aust N Z J Med. 1997;27(4):439.
55. Zappasodi P, Corso A, Castagnola C, Tajana M, Lunghi M, Bernasconi C. A successful combination of plasma exchange and intravenous cyclophosphamide in a patient with a refractory thrombotic thrombocytopenic purpura. Eur J Haematol. 1999;63(4):278-279.
56. Beloncle F, Buffet M, Coindre J-P, et al Thrombotic Microangiopathies Reference Center. Splenectomy and/or cyclophosphamide as salvage therapies in thrombotic thrombocytopenic purpura: the French TMA Reference Center experience. Transfusion. 2012;52(11):2436-2444.
57. Stein GY, Zeidman A, Fradin Z, Varon M, Cohen A, Mittelman M. Treatment of resistant thrombotic thrombocytopenic purpura with rituximab and cyclophosphamide. Int J Hematol. 2004;80(1):94-96.
58. Zheng X, Pallera AM, Goodnough LT, Sadler JE, Blinder MA. Remission of chronic thrombotic thrombocytopenic purpura after treatment with cyclophosphamide and rituximab. Ann Intern Med. 2003;138(2):105-108.
59. Bobbio-Pallavicini E, Porta C, Centurioni R, et al The Italian Cooperative Group for TTP. Vincristine sulfate for the treatment of thrombotic thrombocytopenic purpura refractory to plasma-exchange. Eur J Haematol. 1994;52(4):222-226.
60. Mazzei C, Pepkowitz S, Klapper E, Goldfinger D. Treatment of thrombotic thrombocytopenic purpura: a role for early vincristine administration. J Clin Apher. 1998;13(1):20-22.
61. Ferrara F, Copia C, Annunziata M, et al. Vincristine as salvage treatment for refractory thrombotic thrombocytopenic purpura. Ann Hematol. 1999;78(11):521-523.
62. Ziman A, Mitri M, Klapper E, Pepkowitz SH, Goldfinger D. Combination vincristine and plasma exchange as initial therapy in patients with thrombotic thrombocytopenic purpura: one institution's experience and review of the literature. Transfusion. 2005;45(1):41-49.
63. Yang C-W, Chen Y-C, Dunn P, Chang MY, Fang JT, Huang CC. Thrombotic thrombocytopenic purpura (TTP): initial treatment with plasma exchange plus steroids and immunosuppressive agents for relapsing cases. Ren Fail. 2003;25(1):21-30.
64. Spiekermann K, Wörmann B, Rumpf KW, Hiddemann W. Combination chemotherapy with CHOP for recurrent thrombotic thrombocytopenic purpura. Br J Haematol. 1997;97(3):544-546.
65. Shortt J, Oh DH, Opat SS. ADAMTS13 antibody depletion by bortezomib in thrombotic thrombocytopenic purpura. N Engl J Med. 2013;368(1):90-92.
66. 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.
67. Mazepa MA, Raval JS, Moll S, Ma A, Park YA. Bortezomib induces clinical remission and reduction of ADAMTS13 inhibitory antibodies in relapsed refractory idiopathic thrombotic thrombocytopenic purpura. Br J Haematol. 2014;164(6):900-902.
68. Yates S, Matevosyan K, Rutherford C, Shen Y-M, Sarode R. Bortezomib for chronic relapsing thrombotic thrombocytopenic purpura: a case report. Transfusion. 2014;54(8):2064-2067.
69. Everly JJ, Walsh RC, Alloway RR, Woodle ES. Proteasome inhibition for antibody-mediated rejection. Curr Opin Organ Transplant. 2009;14(6):662-666.
70. George JN, López JA, Konkle BA. N-Acetylcysteine: an old drug, a new insight, a potentially effective treatment for thrombotic thrombocytopenic purpura. Transfusion. 2014;54(5):1205-1207.
71. 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.
72. Li GW, Rambally S, Kamboj J, et al. Treatment of refractory thrombotic thrombocytopenic purpura with N-acetylcysteine: a case report. Transfusion. 2014;54(5):1221-1224.
73. Chapin J, Weksler B, Magro C, Laurence J. Eculizumab in the treatment of refractory idiopathic thrombotic thrombocytopenic purpura. Br J Haematol. 2012;157(6):772-774.
74. Shortt J, Opat SS, Wood EM. N-Acetylcysteine for thrombotic thrombocytopenic purpura: is a von Willebrand factor-inhibitory dose feasible in vivo? Transfusion. 2014;54(9):2362-2363.
75. Waters AM, Licht C. aHUS caused by complement dysregulation: new therapies on the horizon. Pediatr Nephrol. 2011;26(1):41-57.
76. Tsai E, Chapin J, Laurence JC, Tsai H-M. Use of eculizumab in the treatment of a case of refractory, ADAMTS13-deficient thrombotic thrombocytopenic purpura: additional data and clinical follow-up. Br J Haematol. 2013;162(4):558-559.
77. Plaimauer B, Scheiflinger F. Expression and characterization of recombinant human ADAMTS-13. Semin Hematol. 2004;41(1):24-33.
78. Antoine G, Zimmermann K, Plaimauer B, et al. ADAMTS13 gene defects in two brothers with constitutional thrombotic thrombocytopenic purpura and normalization of von Willebrand factor-cleaving protease activity by recombinant human ADAMTS13. Br J Haematol. 2003;120(5):821-824.
79. Schiviz A, Wuersch K, Piskernik C, et al. A new mouse model mimicking thrombotic thrombocytopenic purpura: correction of symptoms by recombinant human ADAMTS13. Blood. 2012;119(25):6128-6135.
80. 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.
81. 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.
82. Crawley JT, Scully MA. Thrombotic thrombocytopenic purpura: basic pathophysiology and therapeutic strategies. Hematology (Am Soc Hematol Educ Program). 2013;2013:292-299.
83. 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.
84. 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.
85. Feys HB, Roodt J, Vandeputte N, et al. Inhibition of von Willebrand factor-platelet glycoprotein Ib interaction prevents and reverses symptoms of acute acquired thrombotic thrombocytopenic purpura in baboons. Blood. 2012;120(17):3611-3614.
86. Knöbl P, Jilma B, Gilbert JC, Hutabarat RM, Wagner PG, Jilma-Stohlawetz P. Anti-von Willebrand factor aptamer ARC1779 for refractory thrombotic thrombocytopenic purpura. Transfusion. 2009;49(10):2181-2185.
87. Jilma-Stohlawetz P, Gorczyca ME, Jilma B, Siller-Matula J, Gilbert JC, Knöbl P. Inhibition of von Willebrand factor by ARC1779 in patients with acute thrombotic thrombocytopenic purpura. Thromb Haemost. 2011;105(3):545-552.