Seminars in Hematology
Volume 42, Issue 1 , Pages 29-35 , January 2005

Treatment of von Willebrand disease

  • Francesco Rodeghiero

      Affiliations

    • Department of Hematology and Hemophilia and Thrombosis Center, San Bortolo Hospital, Vicenza, Italy
    • Corresponding Author InformationAddress correspondence to Francesco Rodeghiero, MD, Department of Hematology and Hemophilia and Thrombosis Center, San Bortolo Hospital, I-36100 Vicenza, Italy
    • ,
  • Giancarlo Castaman

      Affiliations

    • Department of Hematology and Hemophilia and Thrombosis Center, San Bortolo Hospital, Vicenza, Italy

References 

  1. Rodeghiero F . von Willebrand disease (Still an intriguing disorder in the era of molecular medicine) . Haemophilia . 2002;8:292–300
  2. Mannucci PM . Desmopressin (DDAVP) in the treatment of bleeding disorders (The first 20 years) . Blood . 1997;90:2515–2521
  3. Mannucci PM . Treatment of von Willebrand’s disease . N Engl J Med . 2004;351:47–58
  4. Ruggeri ZM . Structure of von Willebrand factor and its function in platelet adhesion and thrombus formation . Clin Haematol . 2001;14:257–279
  5. Biggs R , Matthews JM . The treatment of haemorrhage in von Willebrand’s disease and the blood levels of factor VIII (AHG) . Br J Haematol . 1963;9:203–214
  6. Larrieu MJ , Caen JP , Meyer DO , Vainer H , Sultan Y , Bernard J . Congenital bleeding disorders with long bleeding time and normal platelet count. II. Von Willebrand’s disease (report of thirty-seven patients) . Am J Med . 1968;45:354–372
  7. Green D , Potter EV . Failure of AHF concentration to control bleeding in von Willebrand’s disease . Am J Med . 1976;60:357–360
  8. Blatt PM , Brinkhous KM , Culp HR , Krauss JS , Roberts HR . Antihemophilic factor concentrate therapy in von Willebrand’s disease (Effects on replacement therapy) . JAMA . 1976;236:2770–2772
  9. Cash JD , Garder AMA , Da Costa J . The release of plasminogen activator and factor VIII by LVP, AVP, DDAVP, ATIII and OT in man . Br J Haematol . 1974;27:363–364
  10. Mannucci PM , Ruggeri ZM , Pareti FI , Capitanio A . A new pharmacological approach to the management of hemophilia and von Willebrand disease . Lancet . 1977;1:869–872
  11. Mannucci PM , Aberg M , Nilsson IM , Robertson B . Mechanism of plasminogen activator and factor VIII increase after vasoactive drugs . Br J Haematol . 1975;30:81–93
  12. 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;106:107–116
  13. Rodeghiero F , Castaman G , Mannucci PM . Clinical indications for desmopressin (DDAVP) in congenital and acquired von Willebrand disease . Blood Rev . 1991;5:155–161
  14. Mannucci PM , Bettega D , Cattaneo M . Patterns of development of tachyphylaxis in patients with haemophilia and von Willebrand disease after repeated doses of desmopressin . Br J Haematol . 1992;82:87–93
  15. Rose EH , Aledort LM . Nasal spray desmopressin (DDAVP) for mild hemophilia A and von Willebrand disease . Ann Intern Med . 1991;114:563–568
  16. Rodeghiero F , Castaman G , Mannucci PM . Prospective multicenter study on subcutaneous concentrated desmopressin for home treatment of patients with von Willebrand disease and mild or moderate hemophilia A . Thromb Haemost . 1996;76:692–696
  17. Smith TJ , Gill JC , Ambroso DR , Hathaway WE . Hyponatremia and seizures in young children given DDAVP . Am J Hematol . 1989;31:199–202
  18. Bond L , Bevin D . Myocardial infarction in a patient with hemophilia A treated with DDAVP . N Engl J Med . 1988;318:121
  19. Byrnes JJ , Larcada A , Moake JL . Thrombosis following desmopressin for uremic bleeding . Am J Hematol . 1988;28:63–65
  20. Mannucci PM , Lombardi R , Bader R , Vianello L , Federici AB , Solinas S , et al.   Heterogeneity of type I von Willebrand disease (Evidence for a subgroup with an abnormal von Willebrand factor) . Blood . 1985;66:796–802
  21. Rodeghiero F , Castaman G , Di Bona E , Ruggeri M , Lombardi R , Mannucci PM . Hyper-responsiveness to DDAVP for patients with type I von Willebrand’s disease and normal intraplatelet von Willebrand factor . Eur J Haematol . 1988;40:163–167
  22. Federici AB , Mazurier C , Berntorp E , Lee CA , Scharrer I , Goudemand J , et al.   Biologic response to desmopressin in patients with severe type 1 and type 2 von Willebrand disease (Results of a multicenter European study) . Blood . 2004;103:2032–2038
  23. Gralnick HR , Williams SB , McKeown LP , Rick ME , Maisonneuve P , Jenneau C , et al.   DDAVP in type IIa von Willebrand’s disease . Blood . 1986;67:465–468
  24. 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;309:816–821
  25. Castaman G , Rodeghiero F . Desmopressin and type II B von Willebrand disease . Hemophilia . 1996;2:73–76
  26. Castaman G , Rodeghiero F , Lattuada A , Mannucci PM . Desmopressin-induced thrombocytopenia in type I platelet discordant von Willebrand disease . Am J Hematol . 1993;43:5–9
  27. Castaman G , Federici AB , Rodeghiero F , Mannucci PM . Von Willebrand’s disease in the year 2003 (Towards the complete identification of gene defects for correct diagnosis and treatment) . Haematologica . 2003;88:94–108
  28. Casonato A , Pontara E , Sartorello F , Cattini MG , Sartori MT , Padrini R , et al.   Reduced von Willebrand factor survival in type Vicenza von Willebrand disease . Blood . 2002;99:180–184
  29. Mazurier C , Gaucher C , Jorieux S , Goudemand M . Biological effect of desmopressin in eight patients with type 2N (Normandy) von Willebrand disease . Br J Haematol . 1994;88:849–854
  30. Castaman G , Lattuada A , Mannucci PM , Rodeghiero F . Factor VIII:C increases after desmopressin in a subgroup of patients with autosomal recessive von Willebrand disease . Br J Haematol . 1995;89:147–151
  31. Ruggeri ZM , Mannucci PM , Lombardi R , Federici AB, Zimmerman TS . Multimeric composition of factor VIII/von Willebrand factor following administration of DDAVP (Implications for pathophysiology and therapy of von Willebrand’s disease subtypes) . Blood . 1982;59:1272–1278
  32. Rodeghiero F , Castaman G , Di Bona E , Ruggeri M . Consistency of responses to repeated DDAVP infusions in patients with von Willebrand’s disease and hemophilia A . Blood . 1989;74:1997–2000
  33. Castaman G , Rodeghiero F . Current management of von Willebrand’s disease . Drugs . 1995;50:602–614
  34. Rodeghiero F , Castaman G , Meyer D , Mannucci PM . Replacement therapy with virus-inactivated plasma concentrates in von Willebrand disease . Vox Sang . 1992;62:193–199
  35. Berntorp E , Nilsson IM . Use of a high-purity factor VIII concentrate (Hemate P) in von Willebrand’s disease . Vox Sang . 1989;56:212–217
  36. 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;71:173–179
  37. Kreuz W , Mentzer D , Becker S , Scharrer T , Kornhuber B . Haemate P in children with von Willebrand disease . Haemostasis . 1994;24:304–310
  38. Cornu P , Larrieu MJ , Caen J , Bernard J . Transfusion studies in von Willebrand disease (Effect on bleeding time and factor VIII) . Br J Haematol . 1963;9:189–202
  39. Koster T , Blann AD , Briet E , Vandenbroucke JP , Rosendaal FR . Role of clotting factor VIII in effect of von Willebrand factor on occurrence of deep vein thrombosis . Lancet . 1995;345:152–155
  40. Ktraaijenhagen RA , Anker PS , Koopman MMW , Reitsma PH , Prins MH , van den Ende A , et al.   High plasma concentration of facor VIIIc is a major risk factor for venous thromboembolism . Thromb Haemost . 2000;83:5–9
  41. Kyrle PA , Minar E , Hirschl M , Bialonczyk C , Stain M , Schneider B , et al.   High plasma levels of factor VIII and the risk of recurrent venous thromboembolism . N Engl J Med . 2000;343:457–462
  42. Mannucci PM . Venous thromboembolism in von Willebrand disease . Thromb Haemost . 2002;88:378–379
  43. Makris M , Colvin B , Gupta V , Shields ML , Smith MP . Venous thrombosis following the use of intermediate purity FVIII concentrate to treat patients with von Willebrand’s disease . Thromb Haemost . 2002;88:387–388
  44. Burnouf-Radosevich M , Burnouf T . Chromatographic preparation of a therapeutic highly purified von Willebrand factor concentrate from human cryoprecipitate . Vox Sang . 1992;62:1–11
  45. Meriane F , Zerhouni L , Djeha N , Goudemand M , Mazurier C . Biological effects of a S/D-treated, very high purity, von Willebrand factor concentrate in five patients with severe von Willebrand disease . Blood Coagul Fibrinolysis . 1993;4:1023–1029
  46. Goudemand J , Negrier C , Ounnoughene N , Sultan Y . Clinical management of patients with VWD with a VHP VWF concentrate (The French experience) . Haemophilia . 1998;4:48–52
  47. Dobrkovska A , Krzensk U , Chediak JR . Pharmacokinetics, efficacy and safety of Humate-P in von Willebrand disease . Haemophilia . 1998;4(suppl 3):33–39
  48. Lillicrap D , Poon MC , Walker I , Xie F , Schwartz BA  Association of Hemophilia Clinic Directors of Canada . Efficacy and safety of the factor VIII/von Willebrand factor concentrate, Haemate-P/Humate P:ristocetin cofactor unit dosing in patients with von Willebrand disease . Thromb Haemost . 2002;87:224–230
  49. Gill CJ , Ewenstein BM , Thompson AR , Mueller-Velten G , Schwartz BA  Humate-P Study Group . Successful treatment of urgent bleeding in von Willebrand disease with factor VIII/VWF concentrate (Humate-P) (Use of ristocetin factor assay (VWF:RCo) to measure potency and to guide therapy) . Haemophilia . 2003;9:688–695
  50. Thompson AR , Gill CJ , Ewenstein BM , Mueller-Velten G , Schwartz BA  Humate-P Study Group . Successful treatment for patients with von Willebrand disease undergoing urgent surgery using factor VIII/VWF concentrate (Humate-P) . Haemophilia . 2004;10:42–51
  51. Mannucci PM , Chediak J , Hanna W , Byrnes J , Ledford M , Ewenstein BM , et al.   Treatment of von Willebrand disease with a high purity factor VIII/von Willebrand factor concentrate in the treatment of von Willebrand disease (A prospective multicenter study) . Blood . 2002;99:450–456
  52. 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;79:3130–3137
  53. Mannucci PM , Lattuada A , Ruggeri ZM . Proteolysis of von Willebrand factor in therapeutic plasma concentrates . Blood . 1994;83:3018–3027
  54. Cattaneo M , Moia M , Della Valle P , Castellana P , Mannucci PM . DDAVP shortens the prolonged bleeding time of patients with severe von Willebrand disease treated with cryoprecipitate. Evidence for a mechanism of action independent of released von Willebrand factor . Blood . 1989;74:1972–1975
  55. 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;77:1901–1905
  56. Mannucci PM . Hemostatic drugs . N Engl J Med . 1998;339:245–253
  57. Foster PA . The reproductive health of women with von Willebrand disease unresponsive to DDAVP (Results of an international survey) . Thromb Haemost . 1995;74:784–790
  58. Kouides PA . Females with von Willebrand disease (72 years as the silent majority) . Haemophilia . 1998;4:665–676
  59. Hallberg L , Hogdahl AM , Nilsson L, Rybo G . Menstrual blood loss—A population study . Acta Obstet Gynaecol Scand . 1966;45:320–351
  60. Hallberg L , Nilsson L . Determination of menstrual blood loss . Scand J Clin Lab Invest . 1964;16:244–248
  61. Higham JM , O’Brien PM , Shaw RW . Assessment of menstrual blood loss using a pictorial chart . Br J Obstet Gynaecol . 1990;97:734–739
  62. Claessens EA , Cowell CA . Dysfunctional uterine bleeding in the adolescent . Pediatr Clin North Am . 1981;28:369–378
  63. Edlund M , Blomback M , von Schoultz B, Andersson O . On the value of menorrhagia as a predictor for coagulation disorders . Am J Hematol . 1996;53:234–238
  64. Kadir RA , Economides DL , Sabin CA , Owens D, Lee CA . Frequency of inherited bleeding disorders in women with menorrhagia . Lancet . 1998;351:485–489
  65. Goodman-Gruen D , Hollenbach K . The prevalence of von Willebrand disease in women with abnormal uterine bleeding . J Womens Health Gend Based Med . 2001;10:677–680
  66. Conti M , Mari D , Conti E , Muggiasca ML , Mannucci PM . Pregnancy in women with different types of von Willebrand disease . Obstet Gynecol . 1986;68:282–285
  67. Kadir RA . Women and inherited bleeding disorders (Pregnancy and delivery) . Semin Hematol . 1999;3(suppl 4):28–35
  68. Castaman G , Eikenboom JCJ , Contri A , Rodeghiero F . Pregnancy in women with type 1 von Willebrand disease caused by heterozygosity for von Willebrand factor mutation C1130F . Thromb Haemost . 2000;84:351–352
  69. 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;111:1236–1239
  70. Mannucci PM , Cattaneo M . Alloantibodies in congenital von Willebrand’s disease . Res Clin Lab . 1991;21:119–125
  71. 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
  72. Mannucci PM , Federici AB . Antibodies to von Willebrand factor in von Willebrand disease . In:  Aledort LM ,  Hoyer LW ,  Lusher JM ,  Reisner HM ,  White GC editor. Inhibitors to Coagulation Factors . New York, NY: Plenum; 1995;p. 87–92
  73. Mannucci PM , Tamaro G , Narchi G , Candotti G , Federici A , Altieri D , et al.   Life-threatening reaction to FVIII concentrate in a patient with severe vWD and alloantibodies to von Willebrand Factor . Eur J Haematol . 1987;39:467–470
  74. Bergamaschini L , Mannucci PM , Federici AB , Coppola R , Guzzoni S , Agostoni A . Posttransfusion anaphylactic reaction in a patient with severe von Willebrand disease (Role of complement and alloantibodies to von Willebrand factor) . J Lab Clin Med . 1995;125:348–355
  75. Rodeghiero F , Castaman G , Dini E . Epidemiological investigation of the prevalence of von Willebrand’s disease . Blood . 1987;69:454–459
  76. Rodeghiero F, Castaman G: Epidemiology of von Willebrand disease, in Lee C, Kessler C (eds): Textbook of Hemophilia. (in press)
  77. Rodeghiero F , Castaman G . Congenital von Willebrand disease type 1 (Definition, phenotypes, clinical and laboratory assessment) . Best Pract Res Clin Haematol . 2001;14:321–335

PII: S0037-1963(04)00249-5

doi: 10.1053/j.seminhematol.2004.10.001

Seminars in Hematology
Volume 42, Issue 1 , Pages 29-35 , January 2005

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