Following is information about some of the diseases and conditions and the plasma protein therapies that are used to treat them.
Blood Clotting Factors
A person with hemophilia, males, has inherited a missing or low supply of one of the factors needed for normal blood clotting. Depending on the levels of these factors in the blood, persons with hemophilia may experience bleeding after dental work, surgery, or trauma. They also may experience joint bleeds and suffer internal bleeding with no trauma or injury and without apparent cause. Treatment to prevent this spontaneous bleeding typically requires the infusion of blood clotting factor (Factor VIII for hemophilia A or Factor IX for hemophilia B) one-to-three times a week.
Patients with von Willebrand disease, the most common inherited bleeding disorder, may need to receive von Willebrand Factor to treat bleeding episodes. von Willebrand Disease can affect women as well as men, who historically have suffered more from hereditary bleeding disorders. Recombinant analog therapies, those which are not plasma-derived but are made from recombinant DNA technology, for bleeding disorders (antihemophilic factors) are specialized therapies for coagulation deficiencies, including Factor VII, Factor VIII and Factor IX. Recombinant analog therapies are genetically adapted cell cultures, where an original cell is scientifically modified (reprogrammed) to produce specific proteins. These proteins then are harvested for use in therapies.
Intravenous Immune Globulin (IVIG)
Immune deficiency diseases are disorders in which the immune system fails to properly recognize and react to invading microorganisms, such as bacteria, viruses, and parasites, allowing affected individuals to succumb to abnormal and often life-threatening or fatal infections. Primary immunodeficiency diseases are a group of 100 diseases characterized by functional defects that are intrinsic to the cells and tissues of the immune system. They affect both children and adults and are usually but not always genetically determined. Distinct from primary immunodeficiencies are secondary or acquired defects of the immune system caused by outside agents, such as chemotherapy, radiation, or HIV infection, which leave the host immunosuppressed or compromised.
IVIG is the only effective treatment for primary immunodeficiency disease and also has been proven clinically beneficial in the treatment of secondary immune deficiency diseases and other neurological disorders. In addition to primary immunodeficiency disease, individual United States licensed IVIG products are labeled for the treatment of:
- a) Kawasaki's disease.
- b) chronic lymphocytic leukemia or HIV infection during childhood to prevent bacterial infections.
- c) bone marrow transplantation to prevent graft versus host disease and bacterial infections in adults.
- d) idiopathic thrombocytopenic purpura (ITP).
- e) chronic inflammatory demyelinating polyneuropathy (CIDP)
Some of these U.S. Food and Drug Administration (FDA) licensed indications require periodic IVIG infusions over a limited time. Other indications are chronic and require frequent (i.e., monthly) and lifelong infusions, as is the case with primary immunodeficiency disease.
Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin (AAT) deficiency (A1AD) is one of the most prevalent, potentially lethal hereditary disorders. The function of AAT is to inhibit destructive enzymes, or protease, that cause damage to the liver (e.g., fibrosis and cirrhosis) and to the lungs (e.g., emphysema). A1AD, also known as genetic COPD, is the leading cause of pediatric liver transplants and causes chronic obstructive pulmonary disease with a high frequency of panacinar emphysema in adults. Although an estimated 100,000 people are stricken with A1AD, only 5 percent know the cause of their illness. The only known treatment for A1AD is the weekly infusion of AAT, the protease inhibitor present in high concentration in human plasma.
Human serum albumin is the most abundant protein in human blood plasma. Albumin is widely used in the treatment of severe conditions such as burns, trauma and shock. It has a proven safety track record, having been used for more than 50 years by tens of millions of patients worldwide. For more information about albumin, click here.
Fibrin Sealant is a plasma protein therapy used as surgical glue to control bleeding. The main active ingredient of fibrin sealant is fibrinogen, a protein that forms a clot when combined with thrombin - another protein that facilitates blood clotting. Among other uses, fibrin sealant is used during surgery to control bleeding where a surgeon is operating; to speed wound healing; and to cover holes made by standard sutures. Fibrin sealants speed up the formation of a stable clot; can be applied to very small blood vessels and to areas that are difficult to reach with conventional sutures; reduce the amount of blood lost during surgery; lower the risk of postoperative inflammation or infection; and are conveniently absorbed by the body during the healing process. They are particularly useful for minimally invasive procedures and for treating patients with blood clotting disorders.