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Pharmacotherapy of von Willebrand disease

, MD &
Pages 481-489 | Published online: 11 May 2013

Bibliography

  • Rodeghiero F, Castaman G, Dini E. Epidemiological investigation of the prevalence of von Willebrand's disease. Blood 1987;69:454-9
  • Franchini M, Lippi G. Recent acquisitions in acquired and congenital von Willebrand disorders. Clin Chim Acta 2007;377:62-9
  • Franchini M, Lippi G. The role of von Willebrand factor in hemorrhagic and thrombotic disorders. Crit Rev Clin Lab Sci 2007;44:115-49
  • Ruggeri ZM. Structure of von Willebrand factor and its function in platelet adhesion and thrombus formation. Best Pract Res Clin Haematol 2001;14:257-79
  • Furlan M. Von Willebrand factor: molecular size and functional activity. Ann Hematol 1996;72:341-8
  • Dent JA, Galbusera M, Ruggeri ZM. Heterogeneity of plasma von Willebrand factor multimers resulting from proteolysis of the constituent subunit. J Clin Invest 1991;88:774-82
  • Ruggeri ZM, Zimmerman TS. Variant von Willebrand's disease: characterization of two subtypes by analysis of multimeric composition of factor VIII/von Willebrand factor in plasma and platelets. J Clin Invest 1980;65:1318-25
  • Sadler JE, Budde U, Eikenboom JC, et al. Update on the pathophysiology and classification of von Willebrand disease: a report of the Subcommittee on von Willebrand Factor. J Thromb Haemost 2006;4:2103-14
  • Mannucci PM. How I treat patients with von Willebrand disease. Blood 2001;97:1915-19
  • Federici AB, Castaman G, Mannucci PM; Italian Association of Hemophilia Centers (AICE). Guidelines for the diagnosis and management of von Willebrand disease in Italy. Haemophilia 2002;8:607-21
  • Mannucci PM. Treatment of von Willebrand's disease. N Engl J Med 2004;351:683-94
  • Mannucci PM, Franchini M, Castaman G, Federici AB; on behalf of AICE. Evidence based recommendations on the treatment of von Willebrand disease in Italy. Blood Transfus 2009;7:117-26
  • Favaloro EJ, Franchini M, Lippi G. Biological therapies for von Willebrand disease. Expert Opin Biol Ther 2012;12:551-64
  • Franchini M, Lippi G. The management of patients with congenital von Wilebrand disease during surgery or other invasive procedures: focus on antihemophilic factor/von Willebrand factor complex. Biologics 2007;1:285-9
  • Mannucci PM. Hemostatic drugs. N Engl J Med 1998;339:245-53
  • Franchini M. The use of desmopressin as a hemostatic agent: a concise review. Am J Hematol 2007;82:731-5
  • Mannucci PM, Federici AB. Management of inherited von Willebrand disease. Best Pract Res Clin Haematol 2001;14:455-62
  • Rodeghiero F, Castaman G, Mannucci PM. Clinical indications for desmopressin (DDAVP) in congenital and acquired von Willebrand disease. Blood Rev 1991;5:155-61
  • Mannucci PM. Desmopressin (DDAVP) in the treatment of bleeding disorders: the first twenty years. Haemophilia 2000;6(Suppl 1):60-7
  • Kaufmann JE, Oksche A, Wollheim CB, et al. Vasopressin induced von Willebrand factor secretion from endothelial cells involves V2 receptors and cAMP. J Clin Invest 2000;106:107-16
  • 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
  • Mannucci PM, Bettega D, Cattaneo M. Patterns of development of tachyphylaxis in patients with haemophilia and von Willebrand disease after repeated doses of desmopressin (DDAVP). Br J Haematol 1992;82:87-93
  • Mannucci PM, Canciani MT, Rota L, Donovan BS. Response of factor VIII/von Willebrand factor to DDAVP in healthy subjects and patients with haemophilia A and von Willebrand disease. Br J Haematol 1981;47:283-93
  • Castaman G, Lethagen S, Federici AB, et al. Response to desmopressin is influenced by the genotype and phenotype in type 1 von Willebrand disease (VWD): results from the European Study MCMDM-1VWD. Blood 2008;111:3531-9
  • Federici AB, Mazurier C, Berntorp E, 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;103:2032-8
  • 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
  • Federici AB, James P. Current management of patients with severe von Wilebrand disease type 3: a 2012 update. Acta Haematol 2012;18:88-99
  • Smith TJ, Gill JC, Ambruso DR, Hathaway WE. Hyponatremia and seizures in young children given DDAVP. Am J Hematol 1989;31:199-202
  • Bond L, Bevan D. Myocardial infarction in a patient with hemophilia treated with DDAVP. N Engl J Med 1988;318:121
  • Byrnes JJ, Larcada A, Moake JL. Thrombosis following desmopressin for uremic bleeding. Am J Hematol 1988;28:63-5
  • Batlle J, López-Fernández MF, Fraga EL, et al. Von Willebrand factor/factor VIII concentrates in the treatment of von Willebrand disease. Blood Coagul Fibrinolysis 2009;20:89-100
  • Castaman G. Treatment of von Willebrand disease. Blood Transfus 2011;9:s9-s13
  • Goudemand J, Scharrer I, Berntorp E, et al. Pharmacokinetic studies on Wilfactin, a von Willebrand factor concentrate with a low factor VIII content treated with three virus-inactivation/removal methods. J Thromb Haemost 2005;3:2219-27
  • Mannucci PM, Chediak J, Hanna W, The Alphanate Study Group. Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate: a prospective, multicenter study. Blood 2002;99:450-6
  • Federici AB, Baudo F, Caracciolo C, et al. Clinical efficacy of highly purified, doubly virus-inactivated factor VIII/von Willebrand factor concentrate (Fanhdi) in the treatment of von Willebrand disease: a retrospective clinical study. Haemophilia 2002;8:761-7
  • Berntorp E, Wyndyga J; European Wilate Study Group. Treatment and prevention of acute bleedings in von Willebrand disease-efficacy and safety of Wilate, a new generation von Willebrand factor/factor VIII concentrate. Haemophilia 2009;15:122-30
  • Berntorp E, Archey W, Auerswald G, et al. A systematic overview of the first pasteurized VWF/FVIII medicinal product, Haemate P/Humate P: history and clinical performance. Eur J Haematol 2008;70(Suppl 1):3-35
  • Gill JC, Ewenstein BM, Thompson AR, Humate-P Study Group. Successful treatment of urgent bleeding in von Willebrand disease with factor VIII/von Willebrand factor concentrate (Humate-P): use of the ristocetin cofactor assay (VWF:RCo) to measure potency and to guide therapy. Haemophilia 2003;9:688-95
  • Thompson AR, Gill JC, Ewenstein BM, Humate-P Study Group. Successful treatment for patients with von Willebrand disease undergoing urgent surgery using factor VIII/von Willebrand factor concentrate (Humate-P). Haemophilia 2004;10:42-51
  • Lethagen S, Kyrle PA, Castaman G, the Haemate P Surgical Study Group. Von Willebrand factor/factor VIII concentrate (Haemate P) dosing based on pharmacokinetics: a prospective multicenter trial in elective surgery. J Thromb Haemost 2007;5:1420-30
  • Castaman G, Coppola A, Zanon E, et al. Efficacy and safety during formulation switch of pasteurized VWF/FVIII concentrate: results from an Italian prospective observational study in patients with von Willebrand disease. Haemophilia 2013;19:82-8
  • Rivard GE, Aledort L; Alphanate Surgical Investigators. Efficacy of factor VIII/von Willebrand factor concentrate Alphanate in preventing excessive bleeding during surgery in subjects with von Willebrand disease. Haemophilia 2008;14:271-5
  • Berntorp E. Prophylaxis in vone Willebrand disease. Haemophilia 2008;14(Suppl 5):47-53
  • Franchini M. Surgical prophylaxis in von Willebrand's disease: a difficult balance to manage. Blood Transfus 2008;6(Suppl 2):s33-8
  • Franchini M, Mannucci PM. Von Willebrand disease-associated angiodysplasia: a few answers, still many questions. Br J Haematol 2013;161:177-82
  • Franchini M, Targher G, Lippi G. Prophylaxis in von Willebrand disease. Ann Hematol 2007;86:699-704
  • Berntorp E, Petrini P. Long - term prophylaxis in von Willebrand disease. Blood Coagul Fibrinolysis 2005;16(Suppl 1):S23-6
  • Federici AB. Highly purified VWF/FVIII concentrates in the treatment and prophylaxis of von Willebrand disease. The PRO.WILL study. Haemophilia 2007;13:15-24
  • Borel-Derlon A, Federici A, Roussel-Robert V, et al. Treatment of severe von Willebrand disease with a high - purity von Willebrand factor concentrate (Wilfactin): a prospective study of 50 patients. J Thromb Haemost 2007;5:1115-24
  • Abshire T, Federici AB, Alvarez MT, et al., for the VWD PN. Prophylaxis in severe forms of von Willebrand's disease: results from the von Willebrand Disease Prophylaxis Network (VWD PN). Haemophilia 2013;19:76-81
  • Franchini M, Makris M, Santagostino E, et al. Non-thrombotic-, non-inhibitor-associated adverse reactions to coagulation factor concentrates for treatment of patients with hemophilia and von Willebrand's disease: a systematic review of prospective studies. Haemphilia 2012;18:e164-72
  • Koster T, Blann AD, Briet E, et al. Role of clotting factor VIII in effect of von Willebrand factor on occurrence of deep vein thrombosis. Lancet 1995;345:152-5
  • Kraaijenhagen RA, Anker PS, Koopman MM, et al. High plasma concentration of factor VIIIc is a major risk factor for venous thromboembolism. Thromb Haemost 2000;83:5-9
  • Kyrle PA, Minar E, Hirschl M, et al. High plasma levels of factor VIII and the risk of recurrent venous thromboembolism. N Engl J Med 2000;343:457-62
  • Makris M, Colvin B, Gupta V, et al. Venous thrombosis following the use of intermediate purity FVIII concentrate to treat patients with von Willebrand's disease. Thromb Haemost 2002;88:387-8
  • Mannucci PM. Venous thromboembolism in von Willebrand disease. Thromb Haemost 2002;88:378-9
  • Coppola A, Franchini M, Makris M, et al. Thrombotic adverse events to coagulation factor concentrates for treatment of patients with haemophilia and von Willebrand disease: a systematic review of prospective studies. Haemophilia 2012;18:e173-87
  • Franchini M, Gandini G, Giuffrida A, et al. Treatment for patients with type 3 von Willebrand disease and alloantibodies: a case report. Haemophilia 2008;14:645-6
  • James PD, Lillicrap D, Mannucci PM. Alloantibodies in von Willebrand disease. Blood 2013; Epub ahead of print
  • Mannucci PM, Kempton C, Laffan M, et al. Pharmacokinetics and safety of a novel recombinant human von Willebrand factor manufactured with a plasma-free method: a multicenter prospective clinical trial. Blood 2013; In press
  • Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor (rVWF) in the Treatment of Bleeding Episodes in Von Willebrand Disease (VWD). ClinicalTrials.gov NCT01410227. Available from: http://clinicaltrial.gov/ct2/show/NCT01410227?term=recombinant+von+willebrand+factor&rank=2 [Last access 10 April 2013]
  • Foster PA. The reproductive health of women with von Willebrand Disease unresponsive to DDAVP: results of an international survey. On behalf of the Subcommittee on von Willebrand Factor of the Scientific and Standardization Committee of the ISTH. Thromb Haemost 1995;74:784-90
  • Giles AR, Hoogendoorn H, Benford K. Type IIB von Willebrand's disease presenting as thrombocytopenia during pregnancy. Br J Haematol 1987;67:349-53

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