Use of ristocetin cofactor activity in the management of von Willebrand disease.
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.
Boston Hemophilia Center, Brigham & Women's Hospital and Children's Hospital, and Harvard Medical School, Boston, MA 02115, USA. bewenstein@partners.org
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D001681:Biological Assay; D016903:Drug Monitoring / Q000379:methods; D005169:Factor VIII / Q000627:therapeutic use; D006801:Humans; D012310:Ristocetin; D014842:von Willebrand Diseases / Q000097:blood* / Q000188:drug therapy; D014841:von Willebrand Factor / Q000627:therapeutic use
D014841:von Willebrand Factor; D012310:Ristocetin; D005169:Factor VIII