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DOI: 10.1055/s-2006-949667
Prophylaxis and Treatment of Bleeding Complications in von Willebrand Disease Type 3
Publication History
Publication Date:
15 September 2006 (online)
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ABSTRACT
Treatment of type 3 von Willebrand disease (vWD) relies on infusion with plasma-derived factor concentrates containing von Willebrand factor (vWF). Patients with types 1 and 2 vWD who do not respond satisfactorily after receiving desmopressin need treatment with concentrates. The rationale for long-term prophylaxis in vWD is obvious: prophylaxis has been successfully used in hemophilia, and joint hemorrhages with development of hemophilic arthropathy can occur, especially in type 3 vWD. In Sweden, prophylaxis for vWD began during the 1960s, and we now have experience from a cohort of 37 patients treated for a median of 11 years (range, 2 to 45 years). The majority of subjects (n = 28) have type 3 vWD. The mean dose used for treatment is 24 units factor VIII/kg body weight given one to three times weekly. Indications for prophylaxis have included joint bleeds, bleeds from nose and mouth, menorrhagia and gastrointestinal bleeds. The annual number of bleeds has decreased dramatically following onset of prophylaxis. We conclude that long-term prophylactic treatment of vWD is warranted in the majority of cases with type 3 and in some cases, depending on the clinical phenotype, for patients with other subtypes. Additional studies are ongoing in an international effort, the von Willebrand Disease Prophylaxis Network.
KEYWORDS
von Willebrand disease - von Willebrand factor concentrates - factor VIII - bleeding - prophylaxis
REFERENCES
- 1 Von Willebrand E A. Hereditär pseudohemofili. Fin Lakaresallsk Handl. 1926; 68 87-112
- 2 Silwer J. von Willebrand's disease in Sweden. Acta Paediatr Scand. 1973; 238(suppl) 1-159
- 3 Lak M, Peyvandi F, Mannucci P. 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
- 4 Federici A B. Clinical diagnosis of von Willebrand disease. Haemophilia. 2004; 10(suppl 4) 169-176
- 5 Dobrkovska A, Krzensk U, Chediak J R. Pharmacokinetics, efficacy and safety of Humate-P® in von Willebrand disease. Haemophilia. 1998; 4 33-39
- 6 Lethagen S, Carlson M, Hillarp A. A comparative in vitro evaluation of six von Willebrand factor concentrates. Haemophilia. 2004; 10 243-249
- 7 Federici A B, Castaman G, Thompson A, Berntorp E. Von Willebrand's disease: clinical management. Haemophilia. 2006; 12(suppl 3) 152-158
- 8 Mannucci P M, Tenconi P M, 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
- 9 Berntorp E, Nilsson I M. Use of a high-purity factor VIII concentrate (Hemate P) in von Willebrand's disease. Vox Sang. 1989; 56 212-217
- 10 Mannucci P M, Chediak J, Hanna W et al.. Alphanate Study Group. Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate: a prospective, multicenter study. Blood. 2002; 99 450-456
- 11 Sadler J E, Mannucci P M, Berntorp E et al.. Impact, diagnosis and treatment of von Willebrand disease. Thromb Haemost. 2000; 84 160-174
- 12 Mannucci P M. Hemostatic drugs. N Engl J Med. 1998; 339 245-253
- 13 Castillo R, Escolar G, Monteagudo J, Aznar-Salatti J, Reverter J C, 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. 1997; 37 785-790
- 14 Nilsson I M, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992; 232 25-32
- 15 Van Creveld S. Prophylaxis of joint hemorrhages in hemophilia. Acta Haematol. 1971; 45 120-127
- 16 Berntorp E, Astermark J, Bookman S et al.. Consensus perspectives on prophylactic therapy for haemophilia: summary statement. Haemophilia. 2003; 9(suppl 1) 1-4
- 17 Berntorp E, Perini P. Long-term prophylaxis in von Willebrand disease. Blood Coagul Fibrinolysis. 2005; 16(suppl 1) S23-S26
- 18 Berntorp E, Baghaei F, Holmström M et al.. Long-term Prophylaxis in VWD. The Swedish Experience. The International Society on Thrombosis & Haemostasis XXth Congress (abst 01444) August 6-12, 2005 Sydney, Australia;
- 19 Ahnström J, Berntorp E, Lindvall K, Bjorkman S. A 6-year follow-up of dosing, coagulation factor levels and bleedings in relation to joint status in the prophylactic treatment of haemophilia. Haemophilia. 2004; 10 689-697
- 20 Nilsson I M, Bergentz S E, Larsson S A. Surgery in von Willebrand's disease. Ann Surg. 1979; 190 746-752
- 21 Berntorp E, Abshire T. The von Willebrand Disease Prophylaxis Network (vWD PN): Exploring a treatment concept. Thromb Res. 2006; 118(suppl 1) S19-S22
- 22 Feldman B M, Pai M, Rivard G E et al.. The Association of Hemophilia Clinic Directors of Canada Prophylaxis Study Group. Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost. 2006; 4 1228-1236
Erik BerntorpM.D. Ph.D.
Malmö Centre for Thrombosis and Haemostasis
Malmö University Hospital, SE-20502 Malmö, Sweden
Email: Erik.Berntorp@med.lu.se