Glossary

Activated Prothrombin Complex Concentrates: Two prothrombin complex concentrates are purposely "activated" so that they contain some FIX, FX, etc. in active form (FIXa, FXa, etc.). Autoplex T and FEIBA are to be used in inhibitor patients only. Back to Glossary index.

Afibrinogenemia: The absence of fibrinogen from the blood. Base pairs—The smallest piece of information in a gene. Back to Glossary index.

AIDS (Acquired Immune Deficiency Syndrome): a disease that attacks and destroys the body's immune system, leaving the patient open to infections and other diseases and causing weight loss, central nervous system problems, and death. It is caused by the human immunodeficiency virus. Back to Glossary index.

Anemia: abnormally low total volume of blood or low levels of hemoglobin, or red blood cells in the blood. Back to Glossary index.

Antifibrinolytic: Inhibiting the breakdown of fibrin, the blood component that forms the essential portion of a blood clot. Back to Glossary index.

Arthropathy: joint disease. Back to Glossary index.

Arthroscopy: a surgical procedure to examine the inside of a joint. Back to Glossary index.

Asymptomatic: showing no outward signs of a condition. Back to Glossary index.

Autosomal: Relating to any chromosome that is not a sex chromosome. Back to Glossary index.

Autosome: any of the non-sex chromosomes. Back to Glossary index.

Bethesda Unit (BU): is a term that references the inhibitor activity. A person whose blood test shows one Bethesda unit has enough antibodies to neutralize 50% of the clotting factor administered, within a two-hour period. Back to Glossary index.

Bleeding Disorders: diseases in which the blood does not clot normally. Back to Glossary index.

Break Through Bleed: a phrase used to describe a bleed that occurs despite the fact that a patient is on prophylaxis treatment. This is in reference to the bleeding episode breaking through the treatment regimen. Back to Glossary index.

Carriers: individuals who have a disease-causing gene but do not have the condition themselves, because they have one normal gene. Back to Glossary index.

Christmas Disease: is simply another name for Hemophilia B, where the person is deficient in factor IX. Back to Glossary index.

Chromosomes: threadlike structures inside human cells that contain thousands of genes and that are passed down through families. Back to Glossary index.

Clotting Factors: blood proteins required for blood to clot normally, often called "factors." Back to Glossary index.

Coagulation: blood clotting. Back to Glossary index.

Coagulation Cascade: is a series of reactions that results in the formation of a fibrin net that holds the platelets together and forms a clot. Back to Glossary index.

Coagulation Pathways: is the name for the different segments of the coagulation cascade. The intrinsic and extrinsic pathways are the two branches of the coagulation cascade that function independently and can independently trigger the common pathway. The completion of the common pathway results in clot formation. Back to Glossary index.

Coagulopathy: A disorder that prevents normal clotting of the blood. Back to Glossary index.

Creutzfeldt-Jakob Disease (CJD): an extremely rare disease causing brain damage and death. It is not thought to be transmitted by blood or blood-derived products but instead a spontaneous transformation of normal prions found in the human body. CJD should not be confused with vCJD as the origins and rapidness of onset differ greatly even though the symptoms are generally the same. Back to Glossary index.

Cryoprecipitate: a concentrated form of plasma that contains factor VIII. Back to Glossary index.

CVAD / Port: Central Venous Access Devices are used for people who need frequent IV infusions. By leaving a permanent or long term catheter in the person, accessing a vein can be made easier and lead to less damage to the blood vessels. Back to Glossary index.

Deoxyribonucleic acid (DNA): The chemical substance that makes up genes. Back to Glossary index.

Desmopressin Acetate: (DDAVP, Stimate) a drug used to treat mild hemophilia A and von Willebrand disease that doubles or triples factor VIII levels in the blood. When given to persons who have the capability of producing some FVIII or vWF, the drug effects a rapid, transient increase in FVIII and vWF. It can be given intravenously, subcutaneously, or by intranasal spray. The intranasal spray form is called Stimate. Back to Glossary index.

Dysfibrinogenemia: Malfunction of fibrinogen in the blood. Back to Glossary index.

Factor IX Concentrate: Factor IX products which contain very little or no coagulation factors other than FIX, which include AlphaNine SD, Benefix, and Mononine. Back to Glossary index.

Factor VIII Products Rich in von Willebrand Factor: In certain plasma-derived FVIII concentrates, the high molecular weight multimers of von Willebrand factor are preserved. One product, Humate-P, has been approved by the FDA for use in patients with von Willebrand disease. Two other products, Alphanate and Koate DVI, may also be effective in preventing or controlling bleeding in persons with vWD. There is also a vWF product manufactured in France that is currently in U.S. clinical trials sponsored by the American Red Cross. Back to Glossary index.

Fibrinogen: Factor I, a protein in the blood that is converted to fibrin by the action of thrombin. Back to Glossary index.

Fusion: a surgical procedure in which a joint is removed and the cut ends of the bones are held together with screws or clamps. Fusion relieves the pain of advanced joint disease and strengthens weak joints. Back to Glossary index.

Gene: a section of DNA, the chemical code of the body that controls production of a body protein. Back to Glossary index.

Gene Therapy: a scientific method to replace disease-causing genes with normal ones. Back to Glossary index.

Generation: is a descriptor used to describe recombinant clotting factor products and the technology used to create them. Please check out our Generations Page for more information. Back to Glossary index.

Heated in Aqueous Solution (Pasteurized): Factor VIII concentrates that are heated for 10 hours at 60oC in aqueous solution in the presence of stabilizers such as sucrose or neutral amino acids. Products include Humate-P and Monoclate P. Back to Glossary index.

Hemophilia: a bleeding disorder in which a clotting factor (usually factor VIII or IX) is missing or does not function normally, afflicting primarily males.For more detailed information on hemophilia please visit our Hemophilia Information Page. Back to Glossary index.

Hemophilia Treatment Center (HTC): a federally funded clinic where a team of doctors, nurses, social workers, and physical therapists work together to deliver comprehensive care to people with bleeding disorders. Back to Glossary index.

Hepatitis: Inflammation of the liver. Back to Glossary index.

Hepatitis A: One of the common viral causes of hepatitis. Hepatitis A may be contracted through close personal contact or ingesting contaminated food or substances. This form of hepatitis is not chronic and may last anywhere from a couple weeks to a few months. Vaccinations are available for people over 1 year of age.

Hepatitis B: One of the common viral causes of hepatitis. The Hepatitis B virus may be contracted through the exchange of certain body fluids such as seamen and blood. Commonly infection is transmitted through sex and/or sharing needles. This form of Hepatitis can be either acute (short term) or chronic (ongoing and continuous). Hepatitis B Vaccines are available and the CDC recommends vaccination for everyone, including infants.

Hepatitis C: One of the common viral causes of hepatitis. This form of hepatitis is passed through blood to blood transmission, such as contaminated blood products (prior to the blood safety screening that exists today), sharing needles and unsafe medical practices. Hepatitis C is normally a chronic condition that over extended periods of time can result in cirrhosis and/or liver cancer. There is no vaccine for the Hepatitis C Virus.

Hereditary: that which can be passed down through families. Back to Glossary index.

HIV (Human Immunodeficiency Virus): the virus causing AIDS. Back to Glossary index.

Hypofibrinogenemia: A low or deficient level of fibrinogen in the blood. Back to Glossary index.

ITT/ITE Therapy: Immune Tolerance Therapy is a method people with inhibitors my try to teach their immune system to tolerate the clotting factor. Back to Glossary index.

Immunoaffinity Purified: Factor VIII or FIX concentrates that are purified using murine monoclonal antibodies attached to an affinity matrix. Viral attenuation is augmented before immunoaffinity purification by pasteurization (Monoclate P) or by detergent-solvent treatment (Hemofil M and Monarc-M). In the case of Mononine (a coagulation FIX product), viral attenuation is augmented by sodium thiocyanate and ultrafiltration. Back to Glossary index.

Infiltration: frequently used to describe the event that occurs when IV medication enters the surrounding tissue by escaping the vein. Back to Glossary index.

Inhibitor: inhibitors are antibodies to a clotting factor protein. For more information please go to our Inhibitor Page. Back to Glossary index.

Infusion: a bleeding disorders treatment in which clotting factor is put directly into a vein. Back to Glossary index.

IV / Intravenous: refers to the act of accessing a vein. With Hemophilia and many other bleeding disorders the treatment is often called an intravenous therapy because the medication is delivered directly into the vein. Back to Glossary index.

Joint Bleeds: bleeding into joints that can cause joint disease and is treated by infusion of clotting factor. Bleeds can occur spontaneously, meaning without injury. Back to Glossary index.

Mutation: A defect or change in a gene. Back to Glossary index.

Obligate Carrier: A woman who, on the basis of family history, definitely carries the gene for hemophilia. Obligate carriers can be (1) the daughter of a biological father with hemophilia; (2) the mother of more than one son with hemophilia; or (3) the mother of a son with hemophilia who has one other blood relative with hemophilia. Back to Glossary index.

Orthopedic: having to do with the bones. Back to Glossary index.

Plasma: the liquid part of blood. Back to Glossary index.

Platelet: a component of the blood that is needed for clotting. Back to Glossary index.

Port / CVAD: Central Venous Access Devices are used for people who need frequent IV infusions. By leaving a permanent or long term catheter in the person, accessing a vein can be made easier and lead to less damage to the blood vessels. Back to Glossary index.

Prenatal Diagnosis: Determining the medical condition of a child before it is born. Back to Glossary index.

Prion: a tiny protein that is normally found in the human body and used for various purposes. While normally prions are a harmless necessity, an infectious prion can lead to some very serious diseases. Back to Glossary index.

Prophylaxis: treatment to prevent bleeding episodes. For more information on prophylaxis you may want to visit our Prophylaxis Page. Back to Glossary index.

Protein: Any of a large class of substances consisting of amino acids. Proteins occur in all animal and vegetable matter and are necessary for growth and repair. Back to Glossary index.

Prothrombin: Factor II, a protein in the blood that is converted to thrombin in the coagulation process. Back to Glossary index.

Prothrombin Complex Concentrate (PCCs): These are plasma-derived and can be used to treat patients with deficiencies of factors II, VII and X as well as certain patients with inhibitors to factors VIII and IX. . PCCs contain factors II, VII, IX, and X and proteins C and S (plus small amounts of activated coagulation factors), however these products vary in the amount of factor they contain. Examples of these products include Bebulin VH, Konyne 80, Profilnine SD, and Proplex T. Back to Glossary index.

PUP/PUPs: Previously untreated patient(s). This term is frequently used in studies and research on bleeding disorders. Just as it sounds, previously untreated patients refers to people with bleeding disorders that have never treated with factor products. Back to Glossary index.

Recombinant Factor: Recombinant (r) FVIII refers to genetically engineered or cloned FVIII that is not derived from human or animal plasma.

• Recombinant FVIII (rFVIII) products are Recombinate, Kogenate FS, Helixate FS, Xntha and Advate.
• A recombinant FIX (rFIX) product, BeneFix.
• A recombinant FVII (rFVII) a product, NovoSeven RT. No human serum proteins are used in its production or formulation.

Back to Glossary index.

Symptomatic: showing outward signs of a condition. Thrombin: An enzyme derived from prothrombin that converts fibrinogen to fibrin. Back to Glossary index.

Target Joint: a joint where bleeds tend to occur more often than other joints. Back to Glossary index.

Titer: is a reference to the concentration of antibodies in the blood. In the world of bleeding disorders this term is usually used when talking about the concentration of antibodies to clotting factor a person with inhibitors has in their blood. Back to Glossary index.

Trauma: injury. Back to Glossary index.

vCJD (variant Creutzfeldt-Jakob Disease): an extremely rare prion disease causing brain damage and death. It is unsure if vCJD is the same as BSE (bovine spongiform encephalopathy, or mad cow disease) or just very similar. By all accounts, vCJD is thought to be transmissible through blood transfusions. Back to Glossary index.

Viral Inactivation: methods that kill viruses in clotting factors. Back to Glossary index.

von Willebrand Disease: a bleeding disorder in which von Willebrand factor, a protein that works with factor VIII, is missing or does not function normally; affects females and males. For more specific information, please check out our von Willebrand page. Back to Glossary index.