Coagulation

The three major coagulation or bleeding disorders are hemophilia A, hemophilia B and VWD. Hemophilia A is a blood clotting disorder caused by a deficiency in factor VIII due to a mutation in the factor VIII gene. Inheritance is X-linked recessive; therefore, males are primarily affected. The incidence of hemophilia A is 1 in 10,000. It accounts for about 80 percent of all hemophilia cases. Hemophilia B, like hemophilia A, primarily affects males, but is caused by a deficiency in factor IX due to a mutation in the factor IX gene. About 1 in 50,000 people are born with hemophilia B.

Both types of hemophilia can lead to spontaneous internal bleeding as well as bleeding following injuries or surgery. These bleeding episodes can cause severe joint damage, neurological damage, damage to other organ systems involved in the hemorrhage, and, in rare cases, death.

VWD, which is more common than hemophilia, results from a deficiency of von Willebrand factor (VWF), a protein needed for normal clotting. VWF serves two roles in stemming bleeding at the site of an injury. First, VWF ensures normal platelet function. Second, VWF keeps FVIII in circulation longer. VWD affects both males and females equally. Worldwide, the incidence of VWD is approximately 1 in 100 people.

CSL Behring offers a broad range of therapies for bleeding disorders, including recombinant factor VIII, plasma-derived factor VIII and a plasma-derived factor VIII/VWF concentrate that is indicated for prophylaxis and treatment of hemophilia A and for prophylaxis and treatment of spontaneous and trauma-induced bleeding episodes in VWD. Our monoclonal antibody purified factor IX product is indicated for the prevention and control of bleeding in patients with hemophilia B. Factor X is provided only by CSL Behring and is used for treating patients with factor X deficiency. With an incidence of 1 in 1-1.5 million people, Factor X deficiency is an especially rare disease.

Treatment

Treating the bleeding episodes involves the prompt and proper use of clotting factor concentrates. CSL Behring provides several therapies for bleeding disorders. These include recombinant factor VIII, plasma-derived factor VIII, and pd factor VIII containing VWF, which are indicated for hemophilia A and to treat spontaneous and trauma-induced bleeding episodes in severe VWD and in mild and moderate VWD where use of desmopressin is known or suspected to be inadequate.

Factor IX is indicated for the prevention and control of bleeding in patients with hemophilia B.