title: Use of recombinant factor VIIa in hereditary bleeding disorders.
abstract: Recombinant factor VIIa is effective as a factor VIII or IX bypassing agent and is relatively safe for the management of bleeding and surgical procedures in patients with factor VIII or IX inhibitors (congenital or acquired hemophilia). It is one of several options in the overall treatment strategy for patients with these difficult conditions. This drug has also been used off label in a limited number of patients with other hereditary bleeding disorders (e.g., factor VII deficiency or antibodies to other clotting factors, and platelet disorders such as Glanzmann thrombasthenia). This paper reviews the proposed mechanisms of action, clinical effectiveness, and safety of rFVIIa for treatment of these disorders and points out areas that require further studies. With expanding indications and more widespread use, thromboembolic complications must be carefully monitored, especially in patients at risk for thromboembolism. The high cost of this drug may be a limiting factor.
authors.affiliation: Division of Hematology and Hematologic Malignancies, Department of Medicine, Southern Alberta Hemophilia Clinic, University of Calgary, Alberta, Canada. mcpoon@ucalgary.ca
authors.forename: M C
authors.initials: MC
authors.lastname: Poon
mesh_terms: D025861:Blood Coagulation Disorders, Inherited / Q000188:drug therapy; D001791:Blood Platelet Disorders / Q000188:drug therapy; D015942:Factor VIIa / Q000009:adverse effects / Q000235:genetics / Q000494:pharmacology / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy; D002836:Hemophilia B / Q000188:drug therapy; D006474:Hemorrhagic Disorders / Q000188:drug therapy*; D006801:Humans; D008954:Models, Biological; D011994:Recombinant Proteins / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use
chemical_list: D011994:Recombinant Proteins; D015942:Factor VIIa