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Glossary
To help you better understand the content provided on this Web site, we have provided
below a list of frequently used words and their meanings. If you do not see a word
that you may have a question about, please
contact us directly and we will try to answer any questions you may have.
Albumin - Human albumin is a protein manufactured by
the liver and found primarily in human plasma. Normally it constitutes about 55%
of all plasma proteins. Albumin performs multiple functions, including the transport
of many small molecules in the blood, such as bilirubin, calcium, and magnesium.
Albumin also binds to toxins and heavy metals, thereby preventing damage they might
otherwise cause to your body.
Alpha1-antitrypsin deficiency
- An inherited genetic condition where the body is unable to produce adequate amounts
of an enzyme called alpha1-antitrypsin (AAT).
Inadequate amounts of AAT can lead to chronic conditions affecting primarily the
lungs in adults and, less frequently, the liver in children and adults.
Ambulatory infusion - Infusion of medication on an
outpatient basis. A patient visits a clinic or a doctor's office to receive treatment
and goes home the same day.
Anaphylactic reaction - A serious, often life-threatening
allergic reaction that is characterized by low blood pressure, rash, difficulty
breathing (shortness of breath and wheezing), and shock (poor tissue perfusion,
which means that there is not enough blood flow into the body tissues).
Antibody - Any of a large number of proteins produced
by specialized B-cells (plasma cells) after exposure to an antigen, which act exclusively
against that antigen in an immune response. (Also called immunoglobulin.) Examples
include immunoglobulin G (IgG), IgM, IgA, IgD, and IgE.
Antigen - Foreign substance that enters the body and
induces the formation of antibodies because it is recognized by the immune system
as a threat. It may be a foreign substance from the environment (such as chemicals,
bacteria or viruses) or formed within the body (such as toxins produced by bacteria
or viruses).
Antithrombin III deficiency - Antithrombin III (AT
III) is a blood protein that inhibits or stops the clotting factors in blood. AT
III deficiency is a rare, inherited, or spontaneously occurring condition that affects
1 in every 2000 to 5000 males and females worldwide. Individuals with this condition
lack the AT III protein and spontaneous clotting can occur as a result of normal,
daily activity.
Autoimmune disorders - Any of a large group of diseases
(for example idiopathic thrombocytopenic purpura (ITP), lupus, or rheumatoid arthritis)
that occur when the body tissues are attacked by its own immune system. The immune
system is a complex organization within the body that, when functioning normally,
seeks out and destroys invaders of the body, particularly infections. A healthy
immune system distinguishes things within the body as "self" or "nonself" and gets
rid of those that are "nonself" (for example, bacteria or viruses). In patients
with these diseases, the immune system does not work properly, and treats its own
body tissues as "nonself." This results in production of antibodies against the
body's own tissues, causing inflammation and tissue destruction.
Bone marrow - The soft, spongy tissue, which is found
in the center of most large bones. It produces white blood cells, red blood cells,
and platelets.
Caprylate - A naturally occurring fatty acid (that
is, it results from breakdown of fats) used by Talecris to rapidly inactivate viruses
and gently purify IGIV. Caprylate is a proven, safe substance that is present in
high amounts in intralipids for total parenteral nutrition (TPN) and has been used
for years as a stabilizer for albumin, another plasma-derived product.
Chromatography - A purification method in which a liquid
chemical mixture is separated into its components by passing (or filtering) it through
a stationary material (liquid or solid). This process removes pathogens and other
contaminants (impurities, infectious agents) to help purify the IGIV.
Coagulation – A series of complex reactions in the
human body that eventually cause a clot to form, which stops bleeding. There are
more than a dozen blood coagulation factors and other proteins found in human blood.
In most individuals, these factors interact with one another, but an individual
with a coagulation disorder is missing or deficient in one of these factors.
COPD (chronic obstructive pulmonary disease) - A term
used for a group of conditions that affect the lungs' ability to take in and expel
air. Emphysema is an example of COPD.
Efficacy - The ability of a drug to produce an effect.
Endotoxin testing - Endotoxins are part of the membrane
of gram-negative bacteria, and testing for the presence of endotoxins is often performed
during many stages of the manufacturing of prescription drugs.
Fibrin - A protein that makes up a blood clot.
Fractionation - A process for separating a mixture
(eg, plasma) into its components.
Gene therapy - An experimental process whereby the
proteins that come from missing or deficient genes could be produced within the
body following administration of genes into the body's muscle or liver. It's believed
that if the missing or deficient genes can be replaced and caused to function normally,
then the missing protein could be produced in a more natural way, possibly leading
to the need of fewer or no administrations of the missed protein.
Genes - The functional and physical unit of heredity
passed from parent to child. Genes are sequences of DNA. Many genes contain the
information for making a specific protein; others are regulatory in function and
determine when and where in the body proteins are made.
Genome - All the genetic material (DNA) in all chromosomes
of a cell of a particular organism.
Genotype - The total hereditary information present
in an individual. Also, genotype can refer to the specific genes that produce a
certain characteristic, for example, eye color, or protein, such as Alpha-1.
Hemophilia - Hemophilia is a genetic disorder characterized
by the deficiency or absence of one of the proteins (or factors) in blood plasma
that is responsible for clotting. Low levels of one of these plasma proteins result
in bleeding and difficulty clotting in the affected individual.
Hemostasis - Hemostasis is the state of balance in
the blood's coagulation system. There are more than a dozen blood coagulation factors
and other proteins found in human blood. These factors interact with one another,
performing a series of complex reactions.
Humoral immunity - Type of immunity in which B-cells
transform to plasma cells, which then produce antibodies against foreign agents
(antigens) after being stimulated by T-cells.
Hyperimmune globulin therapy – These are concentrated
immunoglobulins used to treat patients whose immune systems have been compromised.
Idiopathic thrombocytopenic purpura (ITP) - A rare
autoimmune disorder characterized by an acute shortage of platelets (cell fragments
responsible for blood clotting), which results in bruising and spontaneous bleeding.
IGIV - See Immune Globulin Intravenous.
Immune globulin intravenous - Also known as IGIV or
IVIG, belongs to a group of medicines that work by affecting a patient's immune
system. For decades IGIV has been used to treat a variety of infectious or inflammatory
diseases, as well as a variety of autoimmune disorders.
Immune system - The body system, made up of many organs
and cells, which protects and defends the body against infection, disease-causing
organisms and foreign substances by producing the immune response. This system includes
the thymus, bone marrow, and lymphoid tissues. The main function of the immune system
is to distinguish things within the body as "self" or "nonself" and eliminate those
that are "nonself" (for example, bacteria and viruses).
Immunoglobulin - A specific protein that is produced
by specialized B-cells (plasma cells) against an antigen to aid in fighting infection.
Some immunoglobulins (gamma globulins) take part in various immune responses of
the body to bacteria or foreign substances (allergens, tumor, or transplanted tissue).
(Also called antibodies.) Examples include IgG, IgM, IgA, IgD and IgE.
Immunomodulatory therapy - Therapy capable of changing
the way the body's immune system works either by suppressing or strengthening its
function.
Inflammation – The body's reaction to tissue injury
or infection, which serves to destroy both the injurious agent and the injured tissue.
It is usually characterized in the acute form by pain, heat, redness, swelling,
and loss of function.
Infusion - Delivery of a liquid medication into the
body.
IVIG - see Immune Globulin Intravenous.
Licensure-relevant trial program - Generally drugs
and biologics are licensed for specific indications after meeting a trial's endpoints.
Low pH - A solution of pH less than 7, optimal for
stabilizing IGIV. It acts as an extra step in virus destruction for increased safety
against pathogens, and stabilizes the IGIV, thus eliminating the need to use sugar
stabilizers.
Lymphoid tissue - Tissue that is particularly rich
in T-cells, B-cells, and plasma cells, as well as assistant cells, such as macrophages
and reticular cells. Lymphoid tissue includes the lymph nodes, spleen, thymus, Peyer's
patches, pharyngeal tonsils, and adenoids.
Neutrophil Elastase - An enzyme that functions as a
proteinase. It is made by white blood cells that move through tissues and guard
the body against invaders.
Osmolality - The concentration of osmotically active
particles (eg, salts, sugars, ions) in a solution. The solution can be a body fluid,
such as plasma or urine. The higher the osmolality in body fluids, the greater the
force exerted to draw water out of adjacent tissues or cells (eg, shrinking of red
blood cells which may subsequently lead to aggregation and clotting problems). The
lower the osmolality, the greater the force exerted to push water into adjacent
tissues or cells (eg, swelling of red blood cells which may lead to intravascular
hemolysis and subsequently to renal failure).
Panniculitis - A swelling or inflammation of tissue
underneath the skin. Severe Alpha-1 patients can sometimes experience multiple painful
nodules underneath the skin.
Paranasal sinuses - Air-filled areas of the respiratory
part of the nasal cavity that extend into the bones of the head (frontal, ethmoid,
sphenoid, and maxillary), and vary in size and form in different individuals.
Parenteral - Put into the body by means other than
the digestive system, eg, into a vein via transfusion (intravenous), injected through
the skin (subcutaneous), or into the muscle (intramuscular).
Parvovirus B19 - The B19 virus belongs to the Parvovirdae
family of small DNA viruses and is best known for causing a common childhood skin
condition called "fifth" disease, more generally known as "slapped cheek syndrome."
Pathogenic prion - Results when a normal prion protein
is abnormally converted into an infectious protein particle.
Pathogens - Any disease-producing agent (such as virus,
bacterium, fungus or other microorganism).
pH - Provides a measure on a scale from 0 to 14 of
how acidic or alkaline [basic] a solution is (where 7 is neutral, greater than 7
is alkaline [basic] and less than 7 is acidic).
Phenotype - The physical expression of the characteristics
produced by genes present in an individual. Eye color, blood type, and hair color
are examples of phenotypes produced by genes. Phenotypes can also refer to the actual
protein produced by a certain gene.
Plasma - Colorless watery fluid of blood and lymph,
which contain no cells, and in which red blood cells (erythrocytes), white blood
cells (leukocytes), and platelets are suspended. Plasma is the substance that remains
after all blood cells are removed from blood.
Plasma cell - A type of white blood cell. Many B-cells
mature into plasma cells, which can produce antibodies.
Plasmin - An enzyme that dissolves the fibrin of blood
clots.
Primary humoral immunodeficiency disease (PI) - An
immunodeficiency one is born with that usually results from some genetic defect
(changes to the genetic material, DNA), in which the body is unable to produce antibodies
in response to an antigen (see humoral immunity).
Primary immune response - The way a body reacts the
first time it is exposed to a particular antigen. Usually it is less extensive than
the secondary immune response. It is slower, takes longer for the body to react,
doesn't last long, and produces fewer antibodies.
Primary immunodeficiency - Inability of a body to produce
primary immune response. It is classified into four main groups depending on which
component of the immune system is deficient: B-cells (primary humoral immunodeficiency
disease), T-cells, phagocytic cells, or complement.
Prion - A microscopic protein particle similar to a
virus but lacking nucleic acid. A prion is likely the infectious agent responsible
for scrapie and other degenerative diseases of the nervous system.
Protein - Plasma protein contains many different therapeutic
proteins, including protective antibodies, albumin, and coagulation factors. These
proteins are extracted from the plasma, purified, and used to manage serious and
often life-threatening conditions.
Proteinase inhibitor - An enzyme that stops or slows
the activity of proteinases. Proteinases are enzymes that break down proteins. AAT
is a proteinase inhibitor that stops a normally present proteinase in the lungs
from attacking normal lung tissue. Without sufficient levels of AAT, lung tissue
can be destroyed over time, leading to decreased lung function.
Recombinant - Of, relating to, containing, or produced
by genetic engineering.
Replacement therapy - Therapy designed to replace (if
it's missing) or supplement (if the body does not make enough) a substance needed
for a body to work properly. For example, a vitamin replacement therapy is used
for a deficiency that may result from poor nutrition; hormone replacement therapy
is used in the case of certain abnormalities in which the body does not make enough
of an important hormone. Replacement may be physiological (by inducing the body
to produce the missing substance) or it may involve use of a substitute for the
missing substance (for example, a synthetic hormone or a blood transfusion). (Also
see "Augmentation therapy.")
Risk factors - Most products prescribed for medical
treatment contain features that may increase the risk of adverse events (side effects
experienced due to a specific therapy). The individual product features mentioned
on this Web site, as well as a combination of those product features, can pose a
substantial risk factor for specific patient groups. Remember, your doctor or healthcare
provider is the single best source of information regarding you and your health.
Please consult your doctor if you have any questions about your health, your symptoms,
or your therapy.
rPlasmin - A recombinant version of plasmin.
Subcutaneous - Also referred to as "subQ," meaning
"under the skin" and often refers to the method of delivering a prescription drug
therapy.
T-cells (also called T lymphocytes) - White blood cells
that are primarily involved in cell-mediated immunity, as well as in the control
of B-cell function.
Therapeutic equivalence - A situation in which one
product is considered to be essentially the same as another in terms of efficacy
and tolerability.
Thrombolytic - Thrombolytic therapy involves the use
of a drug or procedure to lyse, break, or dissolve blood clots.
Tissue - A group of similar cells united to perform
a specific function.
Tolerability - The ability of a patient to tolerate
or endure a specific treatment. This is usually assessed in relation to the severity
(mild, moderate, or severe), frequency and duration of adverse events (side effects
experienced due to a specific therapy).
Transgenic - Refers to the alteration of DNA by incorporating
genetic material from another organism.
Vasculitis - Swelling or inflammation of blood vessels,
sometimes occurring in severe Alpha-1 patients.
White blood cells - Cells of the immune system responsible
for both directly (T-cells and macrophages) and indirectly (antibody-producing B-cells)
attacking foreign invaders of the body.
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