Medication Summary
The two principal drug categories used in the treatment of von Willebrand disease (vWD) are nontransfusional compounds (eg, desmopressin [DDAVP], antifibrinolytics) and transfusional compounds. Whenever possible, avoid transfusions.
DDAVP is the treatment of choice for individuals with vWD type 1. Responses to DDAVP are variable in patients with type 2 disease. Individuals with vWD type 3 have a virtually complete deficiency of vWF; therefore, because DDAVP acts by releasing stored vWF, the drug has no effect in type 3 disease.
Coagulation Factors
Class Summary
Replacing coagulation factors are essential to successfully managing bleeding episodes.
von Willebrand factor, recombinant (Vonvendi)
Recombinant von Willebrand factor that promotes platelet aggregation and platelet adhesion on damaged vascular endothelium. In adults aged 18 years or older with von Willebrand disease, it is indicated for on-demand treatment and control of bleeding episodes and for perioperative management of bleeding. In patients with severe type 3 von Willebrand disease receiving on-demand therapy, it is indicated for routine prophylaxis to reduce the frequency of bleeding episodes.
Antihemophilic factor/von Willebrand factor complex (Alphanate, Humate P, Wilate)
When DDAVP cannot raise the vWF level to hemostatically acceptable levels, a blood product containing vWF may be required. VWF, a protein found in normal plasma, is necessary for clot formation; when administered, it can temporarily correct coagulation defects of patients with classic hemophilia (hemophilia A), in whom a deficiency of FVIII exists. The specific activity of different brand products varies. Humate-P and Alphanate are products containing both FVIII and vWF. The dose depends on the patient's weight, the severity of hemorrhage, the severity of deficiency, the presence of inhibitors, and the desired FVIII level.
The clinical effect on the patient is the most important determinant of therapy. When inhibitors are present, dose requirements are extremely variable and are determined by clinical response. The length of treatment and the loading dose depend on the extent and location of the hemorrhage.
Alphanate is indicated for the prevention of excessive bleeding for surgical and/or invasive procedures in vWD when desmopressin is either ineffective or contraindicated. It is not indicated for patients with severe vWD (ie, type III) who are undergoing major surgery.
Humate-P is indicated for the treatment and prevention of spontaneous and trauma-induced bleeding episodes for patients with mild to moderate or severe vWD.
Antifibrinolytic Agents
Class Summary
Antifibrinolytics may be used to prevent the breakdown of formed blood clots in order to temper hemorrhage. These agents block the formation of plasmin. They may be used to manage mucosal bleeding, particularly in the nasopharynx and in the gastrointestinal and genitourinary tracts. Antifibrinolytics are most often used concomitantly with other medications for dental extractions and oral surgery.
Aminocaproic acid (Amicar)
Aminocaproic acid inhibits fibrinolysis via the inhibition of plasminogen activator substances and, to a lesser degree, through antiplasmin activity. Its main disadvantage is that thrombi that form during treatment are not lysed, and its effectiveness is uncertain. Aminocaproic acid has been used to prevent the recurrence of subarachnoid hemorrhage.
Tranexamic acid (Cyklokapron, Lysteda)
Tranexamic acid is an alternative to aminocaproic acid. It inhibits fibrinolysis by displacing plasminogen from fibrin.
Vasopressin-Related
Class Summary
These agents improve platelet function in qualitative disorders.
Desmopressin (DDAVP, Stimate)
Desmopressin is the treatment of choice for individuals with vWD type I. It causes a rapid (about 30 min; peaks in 90-120 min), 3- to 5-fold increase in the release of vWF and FVIII from endothelial cells.
Estrogens
Class Summary
Estrogen may be helpful in reducing menorrhagia. Even in type III disease, in which case vWF and FVIII levels are not necessarily increased, estrogen may mediate changes in the endometrium that lessen menstrual bleeding severity.
Ethinyl estradiol and levonorgestrel (Levora, Nordette, Lutera, Trivora)
Ethinyl estradiol reduces the secretion of luteinizing hormone and follicle-stimulating hormone from the pituitary by decreasing the amount of gonadotropin-releasing hormones.
