In most patients, symptoms are moderate and treatment is required only in cases of marked traumatic injury or for surgery.
In some cases, simple measures such as local compression, gauze plugs, biological glue or hormone therapy may be sufficient.
Two forms of replacement therapy are available:
Desmopressin (Minirin®) induces release of intracellular von Willebrand factor thus increasing levels of circulating factor VIII. It can be given intravenously or intranasally, with the latter route being preferred for minor bleeding episodes.
The efficacy of Desmopressin depends on the type of von Willebrand disease: it is normally effective in type 1 von Willebrand disease, of varying efficacy in types 2A and 2M, and only briefly effective in type 2N.
It is ineffective in type 3 and is contraindicated in type 2B, where it can cause thrombocytopenia.
With repeated administration every 12 to 24 hours, response becomes less effective due to tachyphylaxis. The initial response may be reproduced if an interval of 2 to 3 days is left between successive administrations.
Some patients respond poorly to Desmopressin, in which case replacement therapy may be used.
This approach involves administration of von Willebrand factor either alone or in combination with factor VIII.
Administration of von Willebrand factor alone is associated with increased levels of factor VIII. However, this increase in factor VIII occurs only 12 to 24 hours after administration of von Willebrand factor.
Simultaneous administration of factor VIII and von Willebrand factor produces a concomitant increase in factor VIII and von Willebrand factor.
It should be noted that in acquired von Willebrand disease, these two therapeutic options may be ineffective, with intravenous administration of immunoglobulins being necessary in certain cases.
Further, special care may be required for treatment of von Willebrand disease during pregnancy and throughout the post-partum period.
Follow-up treatment is based on the results of clotting tests, in particular assay of clotting activity of factor VIII and of VWF ristocetin cofactor activity.