PDF herunterladen
Thromb Haemost 2001; 86(01): 149-153
DOI: 10.1055/s-0037-1616212
Research Article
Schattauer GmbH

Treatment of Von Willebrand Disease

Pier Mannuccio Mannucci
1   Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Department of Internal Medicine, IRCCS Maggiore Hospital, University of Milan, Italy
› Institutsangaben
Weitere Informationen

Publikationsverlauf

Publikationsdatum:
12. Dezember 2017 (online)

Auch verfügbar auf
    Lizenzen und Reprints

Summary

In von Willebrand disease, there are two main options for the treatment of spontaneous bleeding episodes and for bleeding prophylaxis: desmopressin and transfusional therapy with plasma products. Desmopressin is the treatment of choice for most patients with type 1, who account for approximately 70 to 80 per cent of all cases with the disease. This non-transfusional hemostatic agent raises endogenous factor VIII and von Willebrand factor three- to fivefold and thereby transiently corrects both the intrinsic coagulation and primary hemostasis defects. In patients with the more severe type 3 and in the majority of those with type 2 desmopressin is not effective or is contraindicated, so that it is usually necessary to resort to plasma concentrates containing factor VIII and von Willebrand factor. Concentrates treated with virus inactivation methods should be preferred to cryoprecipitate because they are equally effective and perceived as safer.

Key words

Desmopressin - factor VIII - von Willebrand disease - von Willebrand factor
 

  • References

  • 1 Sadler JE, Mannucci PM, Berntorp E, Bochkov N, Boulyjenkov V, Ginsburg D, Meyer D, Peake I, Rodeghiero F, Srivastava A. Impact, diagnosis and treatment of von Willebrand disease. Thromb Haemost 2000; 84: 160-74.
    Artikel in Thieme ConnectPubMedGoogle Scholar
  • 2 Sadler JE. A revised classification of von Willebrand disease. Thromb Haemost 1994; 71: 520-5.
    Artikel in Thieme ConnectPubMedGoogle Scholar
  • 3 Federici AB. Diagnosis of von Willebrand disease. Haemophilia 1998; 4: 654-60.
    CrossrefPubMedGoogle Scholar
  • 4 Mannucci PM, Ruggeri ZM, Pareti FI, Capitanio A. Deamino–8-D-arginine vasopressin: a new pharmacological approach to the management of hemophilia and von Willebrand disease. Lancet 1977; 1: 869-72.
    PubMedGoogle Scholar
  • 5 Mannucci PM. Desmopressin (DDAVP) in the treatment of bleeding disorders: the first 20 years. Blood 1997; 90: 2515-21.
    PubMedGoogle Scholar
  • 6 Kaufmann JE, Oksche A, Wollheim CB, Gunther G, Rosenthal W, Vischer UM. Vasopressin-induced von Willebrand factor secretion from endothelial cells involves V2 receptors and cAMP. J Clin Invest 2000; 106: 107-16.
    CrossrefPubMedGoogle Scholar
  • 7 Mannucci PM, Bettega D, Cattaneo M. Consistency of responses to repeated DDAVP infusions in patients with von Willebrand disease and haemophilia A. Br J Haematol 1992; 82: 87-93.
    CrossrefPubMedGoogle Scholar
  • 8 Mannucci PM, Canciani MT, Rota L, Donovan BS. Response of factor VIII/ von Willebrand factor to DDAVP in healthy subjects and patients with hemophilia A and von Willebrand disease. Br J Haematol 1981; 47: 283-93.
    CrossrefPubMedGoogle Scholar
  • 9 Smith TJ, Gill JC, Ambroso DR, Hathaway WE. Hyponatremia and seizures in young children given DDAVP. Am J Hematol 1989; 31: 199-202.
    CrossrefPubMedGoogle Scholar
  • 10 Bond L, Bevin D. Myocardial infarction in a patient with hemophilia A treated with DDAVP. N Engl J Med 1988; 318: 121.
    PubMedGoogle Scholar
  • 11 Byrnes JJ, Larcada A, Moake JL. Thrombosis following desmopressin for uremic bleeding. Am J Hematol 1988; 28: 63-5.
    CrossrefPubMedGoogle Scholar
  • 12 Mannucci PM, Lombardi R, Bader R, Vianello L, Federici AB, Solinas S, Mazzucconi MG, Mariani G. Heterogeneity of type I von Willebrand disease: evidence for a subgroup with an abnormal von Willebrand factor. Blood 1985; 66: 796-802.
    PubMedGoogle Scholar
  • 13 Holmberg L, Nilsson IM, Borge L, Gunnarsson M, Sjorin E. Platelet aggregation induced by 1-desamino-8-D-arginine vasopressin (DDAVP) in type II B von Willebrand disease. N Engl J Med 1983; 309: 816-21.
    CrossrefPubMedGoogle Scholar
  • 14 Mazurier C, Gaucher C, Jorieux S, Goudemand M. and the Collaborative Group.. Biological effect of desmopressin in eight patients with type 2 N (‘Normandy’) von Willebrand disease. Br J Haematol 1994; 88: 849-54.
    CrossrefPubMedGoogle Scholar
  • 15 Mannucci PM. Hemostatic drugs. N Engl J Med 1998; 339: 245-53.
    CrossrefPubMedGoogle Scholar
  • 16 Perkins HA. Correction of the hemostatic defects in von Willebrand disease. Blood 1967; 30: 375-80.
    PubMedGoogle Scholar
  • 17 Rodeghiero F, Castaman G, Meyer D, Mannucci PM. Replacement therapy with virus-inactivated plasma concentrates in von Willebrand disease. Vox Sang 1992; 62: 193-9.
    PubMedGoogle Scholar
  • 18 Dobrokovska A, Krzensk U, Chediak JR. Pharmacokinetics, efficacy and safety of Humate-P® in von Willebrand disease. Haemophilia 1998; 4: 33-9.
    PubMedGoogle Scholar
  • 19 Mannucci PM, Chediak J, Hanna W, Byrnes JJ, Kessler CM, Ledford M, Retzios AD, Kapelan BA, Gallagher P, Schwartz RS. and the Alphanate Study Group.. Treatment of von Willebrand’s disease (vWD) with a high purity factor VIII concentrate: dissociation between correction of the bleeding time (BT), vWF multimer pattern, and treatment efficacy. Blood. 1999 94: Suppl. 1 (Part 2 of 2): 98b.
    PubMedGoogle Scholar
  • 20 Kohler M, Hellstern P, Wenzel E. The use of heat-treated factor VIII concentrates in von Willebrand’s disease. Blut 1985; 50: 25-7.
    CrossrefPubMedGoogle Scholar
  • 21 Mazurier C, De Romeuf C, Parquet-Gernez A, Goudemand M. In vitro and in vivo characterization of a high-purity, solvent detergent treated factor VIII concentrate: evidence of its therapeutic efficacy in von Willebrand disease. Eur J Haematol 1990; 75: 228-33.
    CrossrefPubMedGoogle Scholar
  • 22 Pasi KJ, William MD, Enayat MS, Hill FGH. Clinical and laboratory evaluation of the treatment of von Willebrand’s disease patients with heat-treated factor VIII concentrate (BPL 8Y). Br J Haematol 1990; 75: 228-33.
    CrossrefPubMedGoogle Scholar
  • 23 Lethagen S, Berntorp E, Nilsson IM. Pharmacokinetics and hemostatic effect of different factor VIII/von Willebrand factor concentrates in von Willebrand’s disease type III. Ann Hematol 1992; 65: 253-9.
    CrossrefPubMedGoogle Scholar
  • 24 Hanna WT, Bona RD, Zimmerman CE, Carta CA, Hebert GZ, Rickles FR. The use of intermediate and high purity factor VIII products in the treatment of von Willebrand disease. Thromb Haemost 1994; 71: 173-9.
    CrossrefPubMedGoogle Scholar
  • 25 Morfini M, Mannucci PM, Tenconi PM, Longo G, Mazzucconi MG, Rodeghiero F, Ciavarella N, De Rosa V, Arter A. Pharmacokinetics of monoclonally purified and recombinant factor VIII in patients with severe von Willebrand disease. Thromb Haemost 1993; 70: 270-2.
    Artikel in Thieme ConnectPubMedGoogle Scholar
  • 26 Menache D, Aronson DL, Darr F, Montgomery RR. and the Cooperative Study Group.. Pharmacokinetics of von Willebrand factor and factor VIIIC in patients with severe von Willebrand disease (type 3 vWD): estimation of the rate of factor VIIIC synthesis. Br J Haematol 1996; 94: 740-5.
    CrossrefPubMedGoogle Scholar
  • 27 Goudemand J, Negrier C, Ounnoughene O, Sultan Y. Clinical management of patients with von Willebrand disease with a VHP VWF concentrate: the French experience. Haemophilia 1998; 4: 48-53.
    CrossrefPubMedGoogle Scholar
  • 28 Chang AC, Rick ME, Pierce LR, Weinstein MJ. Summary of a workshop on potency and dosage of von Willebrand factor concentrates. Haemophilia 1998; 4: 1-6.
    CrossrefPubMedGoogle Scholar
  • 29 Favaloro EJ, Dean MI, Grispo L, Exner T, Koutts J. Von Willebrand disease: use of collagen binding assay provides potential improvement in laboratory monitoring of desmopressin (DDAVP) therapy. Am J Hematol 1994; 45: 205-11.
    CrossrefPubMedGoogle Scholar
  • 30 Siekmann J, Turecek PL, Schwartz HP. The determination of von Willebrand factor activity by collagen binding assay. Haemophilia 1998; 4: 15-24.
    CrossrefPubMedGoogle Scholar
  • 31 Mannucci PM, Tenconi PM, Castaman G, Rodeghiero F. Comparison of four virus-inactivated plasma concentrates for treatment of severe von Willebrand disease: A cross-over randomized trial. Blood 1992; 79: 3130-7.
    PubMedGoogle Scholar
  • 32 Mannucci PM, Lattuada A, Ruggeri ZM. Proteolysis of von Willebrand factor in therapeutic plasma concentrates. Blood 1994; 83: 3018-27.
    PubMedGoogle Scholar
  • 33 Castillo R, Monteagudo J, Escolar G, Ordinas A, Magallon M, Martin Villar J. Hemostatic effect of normal platelet transfusion in severe von Willebrand disease. Blood 1991; 77: 1901-5.
    PubMedGoogle Scholar
  • 34 Castillo R, Escolar G, Monteagudo J, Aznar-Salatti J, Reverter JC, Ordinas A. Hemostasis in patients with severe von Willebrand disease improves after normal platelet transfusion and normalizes with further correction of the plasma defect. Transfusion 1987; 37: 785-90.
    PubMedGoogle Scholar
  • 35 Lak M, Peyvandi F, Mannucci PM. Clinical manifestations and complications of childbirth and replacement therapy in 385 Iranian patients with type 3 von Willebrand disease. Br J Haematol 2000; III: 1236-9.
    PubMedGoogle Scholar
  • 36 Conti M, Mari D, Conti E, Muggiasca ML, Mannucci PM. Pregnancy in women with different types of von Willebrand disease. Obstet Gynecol 1986; 68: 282-5.
    PubMedGoogle Scholar
  • 37 Pareti FI, Federici AB, Cattaneo M, Mannucci PM. Spontaneous platelet aggregation during pregnancy in a patient with vWD type II B can be blocked by monoclonal antibodies to both platelet glycoproteins Ib and IIb/IIIa. Br J Haematol 1990; 75: 86-91.
    CrossrefPubMedGoogle Scholar
  • 38 Mannucci PM, Federici AB. Antibodies to von Willebrand factor in von Willebrand disease. In: Aledort LM, Hoyer LW, Reisner JM, White II GC. eds. Inhibitors to coagulation factor in the 1990s. New York, NY: Plenum Press; 1995: 87-92.
    Google Scholar
  • 39 Lusher JM. Factor VIII inhibitors: etiology, characterization, natural history, and management. Ann NY Acad Sci 1987; 509: 89-102.
    CrossrefPubMedGoogle Scholar
  • 40 McMillan CW, Shapiro SS, Whitehurst D, Hoyer LW, Rao AV, Lazerson J. The natural history of factor VIII:C inhibitors in patients with hemophilia A: a national cooperative study. II. Observations on the initial development of factor VIII:C inhibitors. Blood 1988; 71: 344-8.
    PubMedGoogle Scholar
  • 41 Mannucci PM, Ruggeri ZM, Ciavarella N, Kazatchkine MD, Mowbray JF. Precipitating antibodies to FVIII/von Willebrand factor in von Willebrand disease: effects on replacement therapy. Blood 1981; 57: 25-31.
    PubMedGoogle Scholar
  • 42 Mannucci PM, Tamaro G, Narchi G, Condotti G, Federici A, Altieri D, Tedesco F. Life-threatening reaction to FVIII concentrate in a patient with severe vWD and alloantibodies to vWF. Eur J Haematol 1987; 39: 467-70.
    PubMedGoogle Scholar
  • 43 Bergamaschini L, Mannucci PM, Federici AB, Coppola R, Guzzoni S, Agostoni A. Postransfusion anaphylactic reaction in a patient with severe von Willebrand disease: role of complement and alloantibodies to von Willebrand factor. J Lab Clin Med 1995; 125: 348-55.
    PubMedGoogle Scholar