Rituximab and intermediate-purity plasma-derived factor VIII concentrate (Koate®) as adjuncts to therapeutic plasma exchange for thrombotic thrombocytopenic purpura in patients with an ADAMTS13 inhibitor
Soumya Pandey
Department of Pathology, University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorMayumi Nakagawa
Department of Pathology, University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorEric R. Rosenbaum
Department of Pathology, University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorKonstantinos Arnaoutakis
Department of Medicine (Division of Hematology/Oncology), University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorLaura F. Hutchins
Department of Medicine (Division of Hematology/Oncology), University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorIssam Makhoul
Department of Medicine (Division of Hematology/Oncology), University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorNatasha Milojkovic
Department of Medicine (Division of Hematology/Oncology), University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorCorresponding Author
Michele Cottler-Fox
Department of Pathology, University of Arkansas for Medical Sciences, Little Rock, Arkansas
Correspondence to: Michele Cottler-Fox, Department of Pathology, 4301 W Markham Street, Slot 823, Little Rock, AR 72205. E-mail: [email protected]Search for more papers by this authorSoumya Pandey
Department of Pathology, University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorMayumi Nakagawa
Department of Pathology, University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorEric R. Rosenbaum
Department of Pathology, University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorKonstantinos Arnaoutakis
Department of Medicine (Division of Hematology/Oncology), University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorLaura F. Hutchins
Department of Medicine (Division of Hematology/Oncology), University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorIssam Makhoul
Department of Medicine (Division of Hematology/Oncology), University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorNatasha Milojkovic
Department of Medicine (Division of Hematology/Oncology), University of Arkansas for Medical Sciences, Little Rock, Arkansas
Search for more papers by this authorCorresponding Author
Michele Cottler-Fox
Department of Pathology, University of Arkansas for Medical Sciences, Little Rock, Arkansas
Correspondence to: Michele Cottler-Fox, Department of Pathology, 4301 W Markham Street, Slot 823, Little Rock, AR 72205. E-mail: [email protected]Search for more papers by this authorAbstract
Thrombotic thrombocytopenic purpura (TTP) results from a congenital or acquired deficiency of the von Willebrand factor (vWF)-cleaving protease ADAMTS13. The disease can be fatal and hence treatment should be initiated promptly. Therapeutic plasma exchange (TPE) remains the standard treatment along with adjunct therapies including steroids and immunosuppressive drugs. Addition of rituximab to TPE has been shown to be beneficial in refractory/relapsing TTP; however, TPE results in removal of rituximab from the circulation requiring more frequent dosing of rituximab to achieve a favorable outcome. The intermediate-purity plasma-derived Factor VIII concentrate (FVIII) Koate® contains the highest amount of ADAMTS13 activity yet reported and has been used successfully in treating congenital TTP. Here we report our experience with addition of this FVIII concentrate to rituximab, corticosteroids and TPE in three TTP patients with an ADAMTS13 inhibitor to permit withholding TPE for 48 h after rituximab infusion. J. Clin. Apheresis 30:50–54, 2015. © 2014 Wiley Periodicals, Inc.
REFERENCES
- 1 Levy GG, Nichols WC, Lian EC, Foroud T, McClintick JN, McGee BM, Yang AY, Siemieniak DR, Stark KR, Gruppo R, Sarode R, Shurin SB, Chandrasekaran V, Stabler SP, Sabio H, Bouhassira EE, Upshaw JD Jr, Ginsburg D, Tsai HM, et al. Mutations in a member of the ADAMTS gene family cause thrombotic thrombocytopenic purpura. Nature 2001; 413: 488–494.
- 2 Veyradier A, Obert B, Houllier A, et al. Specific von Willebrand factor-cleaving protease in thrombotic microangiopathies: a study of 111 cases. Blood 2001; 98: 1765–1772.
- 3 Peyvandi F, Ferrari S, Lavoretano S, et al. von Willebrand factor cleaving protease (ADAMTS-13) and ADAMTS-13 neutralizing autoantibodies in 100 patients with thrombotic thrombocytopenic purpura. Br J Haematol 2004; 127: 433–439.
- 4 Rieger M, Mannucci PM, Kremer H, et al. ADAMTS13 autoantibodies in patients with thrombotic microangiopathies and other immunomediated diseases. Blood 2005; 106: 1262–1267.
- 5 Balduini CL, Gugliotta L, Luppi M, et al. High versus standard dose methylprednisolone in the acute phase of idiopathic thrombotic thrombocytopenic purpura: a randomized study. Ann Hematol 2010; 89: 591–596.
- 6 Sadler JE. Von Willebrand factor, ADAMTS13, and thrombotic thrombocytopenic purpura. Blood 2008; 112: 11–18.
- 7 Bhagirath V, Kelton J, Moore J, Arnold D. Rituximab maintenance for relapsed refractory thrombotic thrombocytopenic purpura. Transfusion 2012; 52: 2517–2523.
- 8 Plaimauer B, Hoving J, Scheiflinger F, et al. Recombinant ADAMTS13 normalizes von Williebrand factor-cleaving activity in plasma of acquired TTP patients by overriding inhibitory antibodies. J Thrombosis Hemostasis 2011; 9: 936–944.
- 9 Allford A, Harrison P, Machin S, et al. Von Williebrand factor-cleaving protease activity in congenital thrombotic thrombocytopenic purpura. Br J Haematol 2000; 111: 1215–1222.
- 10 Peyvandi F, Mannucci PM, Valsecchi C, et al. ADAMTS13 content in plasma-derived factor VIII/von Willebrand factor concentrates. Am J Hematol 2013; 88: 895–898.
- 11 Naik S, Mahoney MD. Successful treatment of congenital TTP with a novel approach using plasma-derived factor VIII. J Pediatr Hematol Oncol 2013; 35: 551–553.
- 12 Lester WA, Williams MD, Allford SL, et al. Successful treatment of congenital thrombocytopenic purpura using the intermediate purity factor VIII concentrate BPL 8Y. Br J Hematol 2002; 119: 176–179.
- 13 Scully M, Michael G, Khair K, et al. The use of intermediate purity factor VIII concentrate BPL 8Y as prophylaxis and treatment in congenital thrombotic thrombocytopenic purpura. Br J Hematol 2006; 135: 101–104.
- 14 Kokame K, Nobe Y, Kokubo Y, et al. FRETSVWF73, a first fluorogenic substrate for ADAMTS13 assay. Br J Haematol 2005; 129: 93–100.
- 15 Kasper CK, Aledort LM, Counts RB, et al. A more uniform measurement of factor VIII inhibitors. Thromb Diath Haemorrh 1975; 34: 869–872.
- 16 Schwartz J, Winters J, Padmanabhan, et al. Guidelines on the use of therapeutic apheresis in clinical practice—evidence-based approach from the writing committee of the American Society for Apheresis: the sixth special issue. J Clin Apher 2013; 28: 145–284.
- 17 Furlan M, Robles R, Solenthaler M, et al. Deficient activity of von Willebrand factor-cleaving protease in chronic relapsing thrombotic thrombocytopenic purpura. Blood 1997: 89: 3097–3103.
- 18 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.
- 19 Tsai HM, Lian EC. Antibodies to von Willebrand factor-cleaving protease in acute thrombotic thrombocytopenic purpura. N Engl J Med 1998; 339: 1585–1594.
- 20 Mackie I, Langley K, Chitolie A, et al. Discrepancies between ADAMTS13 activity assays in patients with thrombotic microangiopathies. Thromb Haemost 2013; 109: 488–496.
- 21 Shah N, Sarode R. Thrombotic thrombocytopenic purpura—what is new? J Clin Apher 2013; 28: 30–35.
- 22 Sarode R, Bandarenko N, Brecher M, 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: 148–167. DOI: 10.1002/jca.21302. Epub 2013 Oct 17.
- 23 Shumak KH, Rock GA, Nair RC. Late relapses in patients successfully treated for thrombotic thrombocytopenic purpura. Canadian Apheresis Group. Ann Int Med 1995; 122: 569–572.
- 24
Bandarenko N and
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: 133–141.
10.1002/(SICI)1098-1101(1998)13:3<133::AID-JCA7>3.0.CO;2-Z CAS PubMed Web of Science® Google Scholar
- 25 Byrnes JJ, Khurana M. Treatment of thrombotic thrombocytopenic purpura with plasma. N Engl J Med 1977; 297: 1386–1389.
- 26 Byrnes JJ, Lian ECY. Recent therapeutic advances in thrombotic thrombocytopenic purpura. Semin Hematol 1979; 5: 199–215.
- 27 Rock GA, Shuman KH, Buskard N, et al. Comparison of plasma exchange with plasma infusion in the treatment of thrombotic thrombocytopenic purpura. N Engl J Med 1991; 325: 393–397.
- 28 Henon P. Treatment of thrombotic thrombopenic purpura. Results of a multicenter randomized clinical study. Presse Med 1991; 20: 1761–1767.
- 29 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: 486–493.
- 30 Böhm M, Betz C, Miesbach W, et al. The course of ADAMTS-13 activity and inhibitor titre in the treatment of thrombotic thrombocytopenic purpura with plasma exchange and vincristine. Br J Haematol 2005; 129: 644–652.
- 31 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: 451–461.
- 32 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: 1746–1753.
- 33 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: 1201–1208.
- 34 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: 104–111.
- 35 Puisset F, White-Koning M, Kamar N, et al. Population pharmacokinetics of rituximab with or without plasmapheresis in kidney patients with antibody-mediated disease. Br J Clin Pharmacol 2013; 76: 734–740.
- 36 Furlan M, Robles R, Morselli B, et al. Recovery and half-life of von Willebrand Factor-cleaving protease after plasma therapy in patients with thrombotic thrombocytopenic purpura. Thromb Haemost 1999; 81: 8–13.
- 37 Cao W, Krishnaswamy S, Camire R, et al. Factor VIII accelerates proteolytic cleavage of von Willebrand factor by ADAMTS13. Proc Natl Acad Sci USA 2008; 105: 7416–7421.
Citing Literature
