Glossary of Terms

Antihemophilic Factor/von Willebrand Factor Complex (Human) Dried, Pasteurized The generic name for Humate-P^®, a product made from human plasma, used in the treatment of von Willebrand disease (VWD). Humate-P contains concentrated von Willebrand factor and factor VIII.

Bleeding disorder A disease in which the body is unable to form blood clots as quickly or as effectively as normal. The family of bleeding disorders includes von Willebrand disease, hemophilia A, hemophilia B, platelet function disorders, and a variety of rare factor deficiencies. Depending on the type of disorder, it may be inherited or acquired.

Blood clotting The process by which a clump forms to repair a blood vessel that has been damaged.

Desmopressin acetate A synthetic drug, which is a copy of a natural hormone. It acts by releasing von Willebrand factor stored in the lining of the blood vessels. Two brands of desmopressin acetate are Stimate^®, which is a high-concentration nasal spray, and DDAVP^®, which comes in an injectable form.

Factor VIII A protein in the blood that is essential for clotting. Factor VIII levels are low in people with von Willebrand disease and hemophilia A.

Hematologist A physician specializing in diseases of the blood.

Hemoglobin A substance in the red cells of blood responsible for carrying oxygen. When a person has iron deficiency anemia caused by loss of blood, hemoglobin levels are lower than normal.

Hemophilia A term used to describe bleeding disorders caused by low levels of factor VIII (hemophilia A) or factor IX (hemophilia B).

Hemostasis The process by which the flow of blood is stopped when a blood vessel is injured.

HTC (Hemophilia Treatment Center) About 140 federally funded treatment centers that offer comprehensive health management and prevention services to people with bleeding disorders. (For more information about HTCs, see the Centers for Disease Control Web site at http://www.cdc.gov/ncbddd/hbd/care_model.htm).

Infusion Injection of a solution into a vein in the body for medicinal or therapeutic purposes.

Menorrhagia Bleeding during the menstrual cycle that is heavier than normal or lasts longer than normal.

Multimer A large von Willebrand factor protein, formed when single VWF protein molecules attach to one another.

Plasma The portion of blood that remains after red cells, leukocytes, and platelets are removed. Plasma contains clotting factor proteins, including von Willebrand factor and factor VIII, as well as immunoglobulins and albumin.

Platelets Small cells (less than 1/10,000 of a centimeter in diameter) circulating in the blood that stick to and spread on the walls of a damaged blood vessel to promote clotting.

Proteins (for blood clotting) Substances (including von Willebrand factor and factor VIII) that circulate in the bloodstream and perform various roles, including blood clotting.

Stimate^® (desmopressin acetate) Nasal Spray, 1.5 mg/mL A brand of high-concentration desmopressin acetate (intranasal) manufactured by Ferring AB and distributed by CSL Behring.

Thromboembolism The blocking of a blood vessel by a blood clot dislodged from its site of origin.

Vasoconstriction The first stage in blood clotting in which the blood vessel constricts to reduce the flow of blood to the damaged area.

von Willebrand disease (VWD) A disease in which the von Willebrand factor in a patient’s blood is deficient or defective. There are three main types of VWD:

Type 1 VWD A form of VWD in which the von Willebrand factor is present at lower than normal levels, affecting blood clotting.

Type 2 VWD A form of VWD disease in which the von Willebrand factor does not work properly, affecting blood clotting. There are 4 subtypes of Type 2 VWD.

Type 3 VWD The most severe form of von Willebrand disease, in which the von Willebrand factor is almost totally missing.

von Willebrand factor (VWF) The clotting protein that is deficient in VWD. VWF is either present at lower than normal levels or does not work properly.

von Willebrand factor (VWF) concentrate A product made from human plasma for use in the treatment of VWD; also contains factor VIII.

Important Safety Information for Stimate^® (desmopressin acetate) Nasal Spray
STIMATE^® Nasal Spray (desmopressin acetate) is contraindicated in individuals with a known hypersensitivity to desmopressin acetate or to any of the components of STIMATE^®. STIMATE^® should not be used in patients with type 2B von Willebrand disease since platelet aggregation may be induced.

Adverse reactions reported with use of injectable and/or intranasal desmopressin acetate include headache, nausea, somnolence, dizziness, chest pain, palpitations and tachycardia, water intoxication, hyponatremia, seizures possibly leading to coma, and severe allergic reactions, including anaphylaxis. See full prescribing information for STIMATE^® Nasal Spray for a complete list.

Important Safety Information

Antihemophilic Factor/von Willebrand Factor Complex (Human), Dried, Pasteurized, Humate-P^® is indicated in adult patients for treatment and prevention of bleeding in hemophilia A (classical hemophilia). Humate-P^® is also indicated in adult and pediatric patients with von Willebrand disease (VWD) for (1) treatment of spontaneous and trauma-induced bleeding episodes, and (2) prevention of excessive bleeding during and after surgery. This applies to patients with severe VWD, and patients with mild and moderate VWD for whom use of desmopressin is known or suspected to be inadequate.

Humate-P^® is contraindicated in individuals with a history of anaphylactic or severe systemic response to antihemophilic factor or von Willebrand factor preparations or to any of its components.

Thromboembolic events have been reported in VWD patients receiving coagulation factor replacement, especially in the setting of known risk factors for thrombosis. Caution should be exercised and antithrombotic measures considered.

Humate-P^® is derived from human plasma. As with all plasma-derived products, the risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.

Although few adverse reactions have been reported the most common are allergic-anaphylactic reactions, including urticaria, chest tightness, rash, pruritus, edema, shock, chills and fever, and hypervolemia. For patients undergoing surgery, the most common adverse reactions are postoperative wound or injection-site bleeding.

Please see accompanying full prescribing information for Humate-P^®.