Inhibition of von Willebrand factor by ARC1779 in patients with acute thrombotic thrombocytopenic purpura

Thrombosis and Haemostasis

Volumn 105, Issue 3, 2011, Pages 545-552

  Jilma Stohlawetz, Petra ^a   Gorczyca, Monika E ^a   Jilma, Bernd ^a   Siller Matula, Jolanta ^a   Gilbert, James C ^b   Knöbl, Paul ^a  

^a MEDICAL UNIVERSITY OF VIENNA   (Austria)

^b Archemix Corporation   (United States)

Author keywords

Aptamer; ARC1779; Platelets; Thrombotic thrombocytopenic purpura (TTP); Von Willebrand factor (VWF)

Indexed keywords

APTAMER; ARC 1779; STEROID; UNCLASSIFIED DRUG; VON WILLEBRAND FACTOR;

ACUTE DISEASE; ADULT; ARTICLE; BLEEDING; CLINICAL ARTICLE; CONSCIOUSNESS DISORDER; CONTINUOUS INFUSION; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG BLOOD LEVEL; DRUG DISTRIBUTION; DRUG EFFICACY; DRUG ELIMINATION; DRUG HALF LIFE; DRUG SAFETY; DRUG TOLERABILITY; FEMALE; HUMAN; LOADING DRUG DOSE; MALE; PHARMACODYNAMICS; PHASE 2 CLINICAL TRIAL; PLASMAPHERESIS; PRIORITY JOURNAL; PROSPECTIVE STUDY; SEIZURE; STEADY STATE; THROMBOCYTE COUNT; THROMBOTIC THROMBOCYTOPENIC PURPURA;

ADOLESCENT; ADULT; APTAMERS, NUCLEOTIDE; BLOOD PLATELETS; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; HUMANS; MALE; MIDDLE AGED; PILOT PROJECTS; PLASMA EXCHANGE; PURPURA, THROMBOTIC THROMBOCYTOPENIC; REMISSION INDUCTION; REPRODUCIBILITY OF RESULTS; TIME FACTORS; TREATMENT OUTCOME; VON WILLEBRAND FACTOR;

EID: 79952505304     PISSN: 03406245     EISSN: None     Source Type: Journal    
DOI: 10.1160/TH10-08-0520     Document Type: Article

References (52)

1. Sadler JE. Von Willebrand factor, ADAMTS13, and thrombotic thrombocytopenic purpura. Blood 2008; 112: 11-18.
2. Zheng XL, Sadler JE. Pathogenesis of thrombotic microangiopathies. Annu Rev Pathol 2008; 3: 249-277. (Pubitemid 351488282)
3. Moake JL. Thrombotic microangiopathies. N Engl J Med. 2002; 347: 589-600.
4. Benz K, Amann K. Pathological aspects of membranoproliferative glomerulonephritis (MPGN) and haemolytic uraemic syndrome (HUS) / thrombocytic thrombopenic purpura (TTP). Thromb Haemost 2009; 101: 265-270.
5. Terrell DR, Williams LA, Vesely SK, et al. The incidence of thrombotic thrombocytopenic purpura-hemolytic uremic syndrome: all patients, idiopathic patients, and patients with severe ADAMTS-13 deficiency. J Thromb Haemost 2005; 3: 1432-1436. (Pubitemid 41725200)
6. Wahl PM, Terrell DR, George JN, et al. Validation of claims-based diagnostic codes for idiopathic thrombotic thrombocytopenic purpura in a commercially-insured population. Thromb Haemost 2010; 103: 1203-1209.
7. Franchini M, Lippi G. The role of von Willebrand factor in hemorrhagic and thrombotic disorders. Crit Rev Clin Lab Sci 2007; 44: 115-149.
8. Lammle B, Kremer Hovinga JA, Alberio L. Thrombotic thrombocytopenic purpura. J Thromb Haemost 2005; 3: 1663-1675. (Pubitemid 41716552)
9. Raife TJ, Montgomery RR. von Willebrand factor and thrombotic thrombocytopenic purpura. Curr Opin Hematol 2000; 7: 278-283. (Pubitemid 30637666)
10. Furlan M, Lammle B. von Willebrand factor in thrombotic thrombocytopenic purpura. Thromb Haemost 1999; 82: 592-600.
11. Lenting PJ, Pegon JN, Groot E, et al. Regulation of von Willebrand factor-platelet interactions. Thromb Haemost 2010; 104: 449-455.
12. 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. (Pubitemid 32938741)
13. Cataland SR, Wu HM. Targeting the inhibitor of ADAMTS13 in thrombotic thrombocytopenic purpura. Expert Opin Pharmacother 2007; 8: 437-444. (Pubitemid 46416412)
14. Asada Y, Sumiyoshi A, Hayashi T, et al. Immunohistochemistry of vascular lesion in thrombotic thrombocytopenic purpura, with special reference to factor VIII related antigen. Thromb Res 1985; 38: 469-479. (Pubitemid 15041946)
15. Kremer Hovinga JA, Meyer SC. Current management of thrombotic thrombocytopenic purpura. Curr Opin Hematol 2008; 15: 445-450.
16. McLeod BC. Therapeutic apheresis: history, clinical application, and lingering uncertainties. Transfusion 2010; 50: 1413-1426.
17. Nguyen L, Terrell DR, Duvall D, et al. Complications of plasma exchange in patients treated for thrombotic thrombocytopenic purpura. IV. An additional study of 43 consecutive patients, 2005 to 2008. Transfusion 2009; 49: 392-394.
18. Norda R, Stegmayr BG. Therapeutic apheresis in Sweden: update of epidemiology and adverse events. Transfus Apher Sci 2003; 29: 159-166. (Pubitemid 36993783)
19. Bramlage CP, Schroder K, Bramlage P, et al. Predictors of complications in therapeutic plasma exchange. J Clin Apher 2009; 24: 225-231.
20. Hovinga JA, Vesely SK, Terrell DR, et al. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood 2010; 115: 1500-11; quiz 1662.
21. Zhan H, Streiff MB, King KE, et al. Thrombotic thrombocytopenic purpura at the Johns Hopkins Hospital from 1992 to 2008: clinical outcomes and risk factors for relapse. Transfusion 2010; 50: 868-874.
22. Heidel F, Lipka DB, von Auer C, et al. Addition of rituximab to standard therapy improves response rate and progression-free survival in relapsed or refractory thrombotic thrombocytopenic purpura and autoimmune haemolytic anaemia. Thromb Haemost 2007; 97: 228-233. (Pubitemid 46592142)
23. Cataland SR, Wu HM. Immunotherapy for thrombotic thrombocytopenic purpura. Curr Opin Hematol 2005; 12: 359-363. (Pubitemid 41284969)
24. 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: 233-238.
25. Ojeda-Uribe M, Federici L, Wolf M, et al. Successful long-term rituximab maintenance for a relapsing patient with idiopathic thrombotic thrombocytopenic purpura. Transfusion 2010; 50: 733-735.
26. Caramazza D, Quintini G, Abbene I, et al. Relapsing or refractory idiopathic thrombotic thrombocytopenic purpura-hemolytic uremic syndrome: the role of rituximab. Transfusion 2010; 50: 2753-2760.
27. Becker RC, Povsic T, Cohen MG, et al. Nucleic acid aptamers as antithrombotic agents: Opportunities in extracellular therapeutics. Thromb Haemost 2010; 103: 586-595.
28. Firbas C, Siller-Matula JM, Jilma B. Targeting von Willebrand Factor and platelet glycoprotein Ib (GpIb) receptor. Expert Rev Cardiovasc Ther 2010; 8: 1689-1701.
29. Bouchard PR, Hutabarat RM, Thompson KM. Discovery and development of therapeutic aptamers. Annu Rev Pharmacol Toxicol 2010; 50: 237-257.
30. Gilbert JC, DeFeo-Fraulini T, Hutabarat RM, et al. First-in-human evaluation of anti von Willebrand factor therapeutic aptamer ARC1779 in healthy volunteers. Circulation 2007; 116: 2678-2686. (Pubitemid 350291240)
31. Diener JL, Daniel Lagasse HA, Duerschmied D, et al. Inhibition of von Willebrand factor-mediated platelet activation and thrombosis by the anti-von Willebrand factor A1-domain aptamer ARC1779. J Thromb Haemost 2009; 7: 1155-1162.
32. Jilma B, Paulinska P, Jilma-Stohlawetz P, et al. A randomised pilot trial of the antivon Willebrand factor aptamer ARC1779 in patients with type 2b von Willebrand disease. Thromb Haemost 2010; 104: 563-570.
33. Mayr FB, Knobl P, Jilma B, et al. The aptamer ARC1779 blocks von Willebrand factor-dependent platelet function in patients with thrombotic thrombocytopenic purpura ex vivo. Transfusion 2010; 50: 1079-1087.
34. Knobl P, Jilma B, Gilbert JC, et al. Anti-von Willebrand factor aptamer ARC1779 for refractory thrombotic thrombocytopenic purpura. Transfusion 2009; 49: 2181-2185.
35. Cosmi B. ARC-1779, a PEGylated aptamer antagonist of von Willebrand factor for potential use as an anticoagulant or antithrombotic agent. Curr Opin Mol Ther 2009; 11: 322-328.
36. Spiel AO, Mayr FB, Ladani N, et al. The aptamer ARC1779 is a potent and specific inhibitor of von Willebrand Factor mediated ex vivo platelet function in acute myocardial infarction. Platelets 2009; 20: 334-340.
37. Reiter RA, Mayr F, Blazicek H, et al. Desmopressin antagonizes the in vitro platelet dysfunction induced by GPIIb/IIIa inhibitors and aspirin. Blood 2003; 102: 4594-4599. (Pubitemid 37494129)
38. Derhaschnig U, Schweeger-Exeli I, Marsik C, et al. Effects of aspirin and NO-aspirin (NCX 4016) on platelet function and coagulation in human endotoxemia. Platelets 2010; 21: 320-328.
39. Kato S, Matsumoto M, Matsuyama T, et al. Novel monoclonal antibody-based enzyme immunoassay for determining plasma levels of ADAMTS13 activity. Transfusion 2006; 46: 1444-1452. (Pubitemid 44117975)
40. Gerritsen HE, Turecek PL, Schwarz HP, et al. Assay of von Willebrand factor (vWF)-cleaving protease based on decreased collagen binding affinity of degraded vWF: a tool for the diagnosis of thrombotic thrombocytopenic purpura (TTP). Thromb Haemost 1999; 82: 1386-1389.
41. Leitner JM, Firbas C, Mayr FB, et al. Recombinant human antithrombin inhibits thrombin formation and interleukin 6 release in human endotoxemia. Clin Pharmacol Ther 2006; 79: 23-34. (Pubitemid 43069576)
42. Patschan D, Witzke O, Duhrsen U, et al. Acute myocardial infarction in thrombotic microangiopathies - clinical characteristics, risk factors and outcome. Nephrol Dial Transplant 2006; 21: 1549-1554. (Pubitemid 44063126)
43. 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: 529-536.
44. Pene F, Vigneau C, Auburtin M, et al. Outcome of severe adult thrombotic microangiopathies in the intensive care unit. Intensive Care Med 2005; 31: 71-78. (Pubitemid 40174284)
45. Hawkins BM, Abu-Fadel M, Vesely SK, et al. Clinical cardiac involvement in thrombotic thrombocytopenic purpura: a systematic review. Transfusion 2008; 48: 382-392.
46. Wahla AS, Ruiz J, Noureddine N, et al. Myocardial infarction in thrombotic thrombocytopenic purpura: a single-center experience and literature review. Eur J Haematol 2008; 81: 311-316.
47. Perez L, Ramappa P, Guzman JA. Myocardial injury in thrombotic thrombocytopenic purpura: a frequent, perplexing complication. Int J Cardiol 2008; 128: 257-260. (Pubitemid 352033585)
48. McCarthy LJ, Danielson CF, Skipworth EM, et al. Myocardial infarction/injury is relatively common at presentation of acute thrombotic thrombocytopenic purpura: the Indiana University experience. Ther Apher 2002; 6: 2-4. (Pubitemid 34218721)
49. Viswanathan S, Rovin BH, Shidham GB, et al. Long-term, sub-clinical cardiac and renal complications in patients with multiple relapses of thrombotic thrombocytopenic purpura. Br J Haematol 2010; 149: 623-625.
50. Kennedy AS, Lewis QF, Scott JG, et al. Cognitive deficits after recovery from thrombotic thrombocytopenic purpura. Transfusion 2009; 49: 1092-1101.
51. Lewis QF, Lanneau MS, Mathias SD, et al. Long-term deficits in health-related quality of life after recovery from thrombotic thrombocytopenic purpura. Transfusion 2009; 49: 118-124.
52. 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: 429-435.