Treatments
Plasma protein therapies either are manufactured using human plasma or recombinant as the starting material to produce life-saving therapies. Following is information about some of the medical diseases and conditions that plasma protein therapies are used to treat.
Blood Clotting Factors
A person with hemophilia, which primarily affects 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. Factor VIIa and anti-inhibitor are used for hemophilia patients that have developed inhibitors against Factor VIII or Factor IX.
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; and d) idiopathic thrombocytopenic purpura. 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.
IVIG is a solution of immunoglobulins containing antibodies that are normally present in human adult blood. The solution is prepared from pools of serum collected from large numbers of donors, extensively processed to help ensure safety, including viral inactivation. IVIG has become the mainstay of therapy for primary immunodeficiency disease because many of these disorders affect B-lymphocyte function thereby affecting those patients' abilities to produce immunoglobulins and functional antibodies.
Immunoglobulin replacement therapy is critical to prevention of such infections as sepsis and meningitis and also prevents ongoing chronic lung disease such as bronchitis and recurrent pneumonia. Because IVIG involves the infusion of a large protein molecule, patients must be monitored for adverse events such as headaches, fever, chills, and, in extreme cases, anaphylactic shock. Adverse events are rare, however, if the rate and volume of the infusion are carefully controlled. Although some physician offices offer infusion services, only the most sophisticated practices infuse IVIG. Certain patients, such as those who are immunoglobulin 'A' deficient, patients with a history of transfusion reactions, and patients with complications and comorbidities should receive their first two or three infusions -- or in some circumstances all their infusions -- in the hospital outpatient department. Furnishing treatments in other settings for such patients may compromise patient safety and access to quality care. Because of the life-threatening nature of immune deficiency diseases and the benefits of IVIG therapy, patients without access to IVIG therapy quite simply could die.
Alpha-1 Antitrypsin
Alpha-1Antitrypsin (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 causes 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. AAT is purified from pooled human plasma, and then processed to reduce the potential for transmission of infectious agents. A 70 kg person with emphysema caused by A1AD should be infused with 4,200 mg of AAT each week. Like IVIG, AAT typically is not self-administered. Because A1AD is such a rare and life-threatening disorder and AAT is the only known treatment, ensuring patients access to this therapy is critical.