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.
Afibrinogenemia: The
absence of fibrinogen from the blood. Base pairs—The smallest piece
of information in a gene.
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.
Anemia: abnormally low
total volume of blood or low levels of hemoglobin, or red blood cells
in the blood.
Antifibrinolytic: Inhibiting
the breakdown of fibrin, the blood component that forms the essential
portion of a blood clot.
Arthropathy: joint disease.
Arthroscopy: a surgical
procedure to examine the inside of a joint.
Asymptomatic: showing
no outward signs of a condition.
Autosomal: Relating to
any chromosome that is not a sex chromosome.
Autosome: any of the
non-sex chromosomes.
Bleeding Disorders: diseases
in which the blood does not clot normally.
Carriers: individuals
who have a disease-causing gene but do not have the condition themselves,
because they have one normal gene.
Chromosomes: threadlike
structures inside human cells that contain thousands of genes and that
are passed down through families.
Clotting Factors: blood
proteins required for blood to clot normally, often called "factors."
Coagulation: blood clotting.
Coagulation Factor IX Concentrate:
Factor IX products which contain very little or no coagulation factors
other than FIX, which include AlphaNine SD, Benefix, and Mononine.
Coagulopathy: A disorder
that prevents normal clotting of the blood.
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; however, this is
an area of active research.
Cryopreciptate: a concentrated
form of plasma that contains factor VIII.
Deoxyribonucleic acid (DNA):
The chemical substance that makes up genes.
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.
Dysfibrinogenemia: Malfunction
of fibrinogen in the blood.
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.Fibrinogen:
Factor I, a protein in the blood that is converted to fibrin by the action
of thrombin.
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.
Gene: a section of DNA,
the chemical code of the body that controls production of a body protein.
Gene Therapy: a scientific
method to replace disease-causing genes with normal ones.
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.
Hemophilia: a bleeding
disorder in which a clotting factor (usually factor VIII or IX) is missing
or does not function normally, afflicting primarily males.
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.
Hepatitis: a disease
caused by injury or infection of the liver.
Hereditary: that which
can be passed down through families.
HIV (Human Immunodeficiency Virus):
the virus causing AIDS.
Hypofibrinogenemia: A
low or deficient level of fibrinogen in the blood.
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.
Infusion: a bleeding
disorders treatment in which clotting factor is put directly into a vein.
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.
Mutation: A defect or
change in a gene.
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.
Orthopedic: having to
do with the bones.
PCCs: Prothrombin complex
concentrates. 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. These products vary in the amount
of factor they contain.
Plasma: the liquid part
of blood.
Platelet: a component
of the blood that is needed for clotting.
Prenatal diagnosis: Determining
the medical condition of a child before it is born.
Prophylaxis: treatment
to prevent bleeding episodes.
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.
Prothrombin: Factor II,
a protein in the blood that is converted to thrombin in the coagulation
process.
Prothrombin Complex Concentrate:
PCC contains factors II, VII, IX, and X and proteins C and S (plus small
amounts of activated coagulation factors). Examples of these products
include Bebulin VH, Konyne 80, Profilnine SD, and Proplex T.
Recombinant Factor: Recombinant (r) FVIII refers to genetically engineered
or cloned FVIII that is not derived from human or animal plasma. Currently
licensed rFVIII products are Recombinate, Kogenate, Helixate, ReFacto
and Advate.
- A recombinant FIX (rFIX) product, BeneFix.
- A recombinant factor VIIa product, NovoSeven. No human serum proteins
are used in its production or formulation.
Symptomatic: showing
outward signs of a condition.Thrombin: An enzyme derived from prothrombin
that converts fibrinogen to fibrin.
Target Joint: a joint
where bleeds tend to occur more often than other joints.
Trauma: injury.
Viral Inactivation: methods
that kill viruses in clotting factors.
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.
|
