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Activated Prothrombin Complex ConcentratesTwo prothrombin
complex concentrates are purposely "activated" so that they contain some
FIX, FX, etc. in active form (FIXa, FXa, etc.). Autoplex T and FEIBA
to be used in inhibitor patients only.
AntifibrinolyticInhibiting the breakdown
of fibrin, the blood component that forms the essential portion of a
Artificial inseminationA method in
which donor sperm are injected into the woman's uterus in order to fertilize
AutosomalRelating to any chromosome
that is not a sex chromosome.
AfibrinogenemiaThe absence of fibrinogen
from the blood. Base pairsThe smallest piece of information in
CarriersIndividuals who carry the
gene for a condition but do not have the condition themselves.
Chromosomesthreadlike structures inside
human cells that contain thousands of genes and that are passed down
ClonedGenes that have been copied
by chemical methods in a laboratory.
Clotting factorsProteins needed to
make blood clot.
Coagulation Factor IX ConcentrateFactor
IX products which contain very little or no coagulation factors other
than FIX, which include AlphaNine SD and Mononine.
CoagulopathyA disorder that prevents
normal clotting of the blood.
Deoxyribonucleic acid (DNA)The chemical
substance that makes up genes.
Desmopressin (DDAVP, Stimate)Desmopressin
acetate is a synthetic analogue of the natural pituitary antidiuretic
hormone, 8-arginine vasopressin. 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.
Dry Heat TreatedNo currently available
FVIII products are exclusively dry heat-treated. Currently available
products that are dry heat-treated include Proplex T and Konyne 80. Proplex
T is dry-heated at 60oC for 144 hours. Konyne 80 is heated at 80oC for
DysfibrinogenemiaMalfunction of fibrinogen
in the blood.
Factor VIII Products Rich in von Willebrand FactorIn
certain plasma-derived FVIII concentrates, the high molecular weight
of von Willebrand factor are preserved. One product, Humate-P, has been
approved by the FDA for use in patients with von Willebrand disease.
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.
FibrinogenFactor I, a protein in the
blood that is converted to fibrin by the action of thrombin.
Gene sequenceThe order of base pairs
in a gene.
Gene therapyMethods to correct a gene
mutation by adding an intact (normal) one or changing one that is already
present. GenesUnits of DNA that contain all the hereditary information
needed to make a product, such as a protein.
Genetic markersDNA pieces that are
easy to identify but that themselves have nothing to do with the gene
being examined, such as the gene for hemophilia.
Germline mosaicismA situation that
occurs when mutations are found in some, but not all, of a woman's eggs.
These mutations occur during egg cell development and are not found in
the woman's other cells, such as blood cells.
Germline therapyGene therapy directed
at embryos; this method replaces all of the altered genes that cause
so that the gene is not passed on to future generations.
GlycoproteinA protein compound that
also contains carbohydrate.
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.
Hereditary diseaseA disease that can
be passed down through families.
HypofibrinogenemiaA low or deficient
level of fibrinogen in the blood.
Immunoaffinity PurifiedFactor 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
InheritedPassed down through families.
Invasive procedureA procedure that
requires cutting into the body.
InversionA part of a gene that is flipped
around so that it can no longer be "read" in the correct direction.
In vitro fertilizationa technique
in which sperm and eggs are mixed together outside the body (usually
glass laboratory plate) with the goal of creating a fertilized egg.
LyonizationThe process of shutting
down one of the X chromosomes in each female cell. Usually, half of the
cells shut down one of the X chromosomes and the other half shut down
the other one.
Maternal transmissionWhen a gene is
passed from the mother.
MutationA defect or change in a gene.
Noninvasive procedureA procedure that
does not require cutting into the body.
Nonrandom X inactivationA phenomenon
in which all the cells in a woman's body shut down the same X chromosome.
Obligate carrierA woman who, on the
basis of family history, definitely carries the gene for hemophilia.
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.
Paternal transmissionwhen a gene is
passed from the father.
PCCsProthrombin 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
factors VIII and IX. These products vary in the amount of factor they
PlateletA component of blood that
contributes to coagulation.
Prenatal diagnosisDetermining the
medical condition of a child before it is born.
ProteinAny 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.
ProthrombinFactor II, a protein in
the blood that is converted to thrombin in the coagulation process.
Prothrombin Complex ConcentratePCC
contains factors II, VII, IX, and X and proteins C and S (plus small
of activated coagulation factors). Examples of these products include
Bebulin VH, Konyne 80, Profilnine SD, and Proplex T.
Recombinant FactorRecombinant (r)
FVIII refers to genetically engineered or cloned FVIII that is not derived
human or animal plasma. Currently licensed rFVIII products are Recombinate
and Kogenate. Recombinate is also distributed under the trade name Bioclate,
and Kogenate is distributed as well under the name Helixate.
A recombinant FIX
(rFIX) product, BeneFix, has also been licensed.
A recombinant factor VIIa product (NovoSeven) has recently been licensed
in the United States. No human serum proteins are used in its production
Another rFVIII product, ReFacto, a deletion rFVIII product that lacks
the B domain and contains no albumin stabilizer, has just been licensed
in the U.S.
Restriction enzymes Proteins that
cut DNA at specific sites.
Solvent Detergent Treated Factor VIII
concentrates that are manufactured using combinations of the solvent,
Tri(n-Butyl) Phosphate (TNBP), with a detergent, such as cholate, Tween
80 (polysorbate), or Triton-X-100, to inactivate lipid-enveloped potential
viral contaminants (lipid-enveloped viruses include HIV, HBV, HCV). Alphanate
and Koate DVI are solvent-detergent treated using TNBP and Polysorbate
80. Hemofil M and Monarc-M are solvent-detergent treated with TNBP and
Triton X-100. A coagulation FIX product (AlphaNine SD) is solvent-detergent
treated using TNBP and Polysorbate 80, as is the prothrombin complex
ThrombinAn enzyme derived from prothrombin
that converts fibrinogen to fibrin.
Vapor TreatedTwo coagulation products
currently licensed in the U.S. use vapor (steam) treatment for viral
Bebulin, a FIX complex concentrate, and FEIBA VH, an activated prothrombin
complex concentrate, are both vapor treated for 10 hours at 60oC and
mbar pressure, followed by 1 hour at 80oC under 1375 mbar pressure.
von Willebrand factorA protein that
carries factor VIII in the blood and helps factor VIII work properly.
X-linkedGenes that are present on
the X chromosome.
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