von Willebrand disease (vWD), the most common of the hereditary
bleeding disorders, arises from quantitative or qualitative defects in
von Willebrand factor (vWF). vWF is a multimeric
plasma protein that plays a key role in primary and secondary haemostasis. In the current classification scheme, vWD is divided into six subtypes that are based on the nature of the vWF defect. Therapeutic strategies depend on the accurate identification of these subtypes. In most clinical situations,
desmopressin is effective treatment for the great majority of patients with mild (type 1) disease, while replacement
therapy with
factor VIII/vWF concentrates that contain high levels of vWF activity is required for most type 2 and nearly all
type 3 vWD patients. Several
factor VIII/vWF replacement products are available, one of which (
Humate P) has been approved for the treatment of vWD by the US Food and Drug Administration. Preliminary results of recent studies support the hypothesis that treatment with
factor VIII/vWF concentrates based upon the content of vWF activity as reflected in the
ristocetin cofactor assay is practicable, safe and efficacious. The establishment of optimal treatment regimens with respect to dose intensity and duration will require further study.