| Agent |
Mechanism of Action |
Dosage |
Benefits |
Side Effects |
Notes |
| Desmopressin |
Vasoconstrictor that causes release of VWF from endothelial
stores |
0.3 µg/kg iv desmopressin or 10 µg
dose of intranasal desmopressin |
No risk of transfusion-transmitted disease. Easily
self-administered (especially intranasal Stimate®). Useful for
mild to moderate bleeds, menorrhagia, prophylaxis for minor invasive procedures,
and dental work |
Flushing, headache, nausea, water intoxication, hyponatremia |
Can be used iv over 30 min, subcutaneously or intranasally.
Response to desmopressin should be determined at the time of diagnosis of
VWD, before its use. Tachyphylaxis occurs with desmopressin, and use is typically
limited to 1 or 2 days. Monitor sodium level and do not administer unnecessary
fluids |
| Antihemophilic
factor (human) (VWF-containing factor VIII concentrates [intermediate
purity factor VIII concentrates]) |
Increases blood levels of VWF and factor VIII |
Varies by treatment needed; 20-50 IU/kg every 12 to 24
hours to achieve and maintain hemostatic levels of VWF and factor VIII |
Low risk of transfusion-transmitted pathogens due to
purification and specific viral inactivation steps. Rich in VWF. Contents
listed by VWF:RCo |
Rare development of alloantibodies in type 3 disease
(43).
High cost |
Intermediate-purity factor VIII concentrates rich in VWF,
with ratio of VWF:RCo:factor VIII includes Humate-P®, 2.5:1;
Koate-DVI®, 1.2:1; and Alphanate®, 0.5:1
(39) |
| Cryoprecipitate |
Increases blood levels of VWF and factor VIII |
Varies by treatment needed. Give q 12-24 h |
Available in most settings if concentrates are not available
for emergency treatment |
Risk of blood-borne transmissions
(48) |
Rarely used secondary to other choices. Each bag contains
80-100 units of VWF |
| Aminocaproic
acid (Amicar®) |
Inhibits fibrinolysis |
Initial loading dose of 5000 mg given before a procedure;
then 50 mg/kg qid either po or iv for bleeding or surgical prophylaxis |
No transfusion-transmission risk. Particularly useful
in controlling mucocutaneous or oral bleeding |
Contraindicated in bleeding of upper urinary tract because
of risk of obstructive uropathy from clotting
(49) |
Add with dental extractions, tonsillectomy, menorrhagia,
mucosal hemorrhage (Amicar®, tranexamic acid)
(50)
Adjunct to agents that increase VWF |
| Tranexamic
acid (Cyklokapron®) |
Inhibits fibrinolysis |
20-25 mg/kg q 8-12 h po, iv, or topical |
More potent than aminocaproic acid |
Contraindicated in bleeding of upper urinary tract because
of secondary risk of obstructive uropathy from clotting
(49) |
Add with dental extractions, tonsillectomy, menorrhagia,
mucosal hemorrhage (Amicar®, tranexamic acid)
(50)
Adjunct to agents that increase VWF |
| Estrogenic compounds |
Mechanism unknown, but increases serum level of VWF and
factor VIII |
Variable, depending on type of preparation |
May reduce menorrhagia |
Headache, hypertension |
Not able to predict response. May also have favorable
effect on endometrium
(49) |
| Fibrin
sealant (Tisseel®) |
A concentrate of fibrinogen combined with thrombin and
calcium to form gelatinous fibrin-rich glue |
Forms a gel for topical use |
May control local bleeding. Useful in some surgeries |
Risk of blood-borne disease when cryoprecipitates are
used as a source of fibrinogen |
May be an adjunct to other therapies in controlling bleeding |
| VWF concentrate |
Raises VWF levels |
Dose by VWF:RCo units |
Highly purified plasma-derived VWF concentrate. Viral
inactivation |
Rare instance of development of VWF antibodies in type
3 VWD
(43) |
Start 12-24 hours before needed, level secondary to de
novo synthesis of factor VIII. Must use in conjunction with factor VIII
concentrate in an acute bleed. Not available in U.S. |
iv = intravenous; IU = international units; po = orally; qid = four times
daily; VWD = von Willebrand's disease; VWF = von Willebrand's factor; VWF:RCo
= von Willebrand's factor:ristocetin cofactor.
