Hemophilia Definitions

There is an entire vocabulary of new words to digest and look up if you are new to factor VIII treatment. Here is a quick reference glossary to help guide your way.

Adverse Event

An untoward response that occurs to a patient that may or may not be related to the prescribed use of a drug, medical device, or other therapy. This may occur in the context of a clinical trial or in the use of a licensed product.

Aseptic

Describes the absence of bacteria or other pathological microorganisms; sterile.

Bethesda Assay

This is the most widely used test for measuring antibodies that inhibit the activity of FVIII. A Bethesda unit is the amount of antibody that will decrease FVIII activity in a laboratory test by 50% in a 2-hour period.

Chromatography

A technique for purifying material by running a solution through a bed of material that has more affinity for some components of the mixture than others. As the mixture flows through, it separates into zones rich in one component and diminished in others. With enough time and space, and employing different chemistries for separation, a single component may be purified from a complex starting material.

Diluent

A liquid used to dilute or reconstitute a concentrated substance prior to application. Sterile water is the diluent used to reconstitute Kogenate^® FS, Antihemophilic Factor (Recombinant).

Efficacy

A substance’s capacity or power to produce an intended effect. A drug is said to have efficacy when it produces the desired measurable, positive, clinical effect.

Factor VIII

A blood protein that is important in blood coagulation. The coagulation system forms a clot that seals a wound and stops the bleeding.

Fermentation

A process of growing and multiplying cells in an environment that contains an appropriate nutrient-rich substance developed to optimize cell growth.

Formulation

The materials that provide stability and ease of handling and administration of the active ingredient in a drug.

Hemostatic Agent

Any drug, medicine, or blood component that promotes the clotting of blood to stop bleeding.

Human albumin/animal albumin

Albumin is a protein that circulates in the blood in high concentration. Human albumin is extracted from human blood or plasma. Human or animal albumin may be used in recombinant FVIII as a nutrient in cell culture and/or as a stabilizer in the final bottle, depending upon the product.

Human Plasma Protein Solution (HPPS)

Human Plasma Protein Solution consists of several human plasma proteins produced in a manner similar to human albumin. HPPS is pasteurized to inactivate many types of viruses.

Immunoaffinity Chromatography

Antibodies bind tightly to a specific site on a protein molecule. Antibodies bound to a chromatography column can selectively bind the specific protein from a complex mixture. This is the unique chemistry used in immunoaffinity chromatography.

Incidence

The number of new cases of a specific disease/adverse or other event during a specified period of time, in a specific population.

Inhibitor

Sometimes when human FVIII is used, the immune system will react to it as aforeign substance and produce antibodies that will bind to the factor VIII protein and reduce its activity.

Pathogen

A general term for an organism that can cause disease. Common human pathogens include viruses, bacteria, and parasites.

PTPs

Previously treated patients: for example, patients who have received any antihemophilic factor treatment.

PUPs

Previously untreated patients: that is, patients who have never received antihemophilic factor treatment.

Purification

A process to remove the unwanted materials from a product.

Recombinant

Genetic material from two or more sources is incorporated into a single combined molecule such as DNA. A gene produced in a recombinant fashion can be replicated many times. Recombinant proteins are proteins that are produced using recombinant genetic material. In manufacturing, recombinant human FVIII is made by non-human cells (such as hamster cells) in culture by inserting the human factor VIII gene into the genetic material of the hamster cell.

Side Effect

An undesired effect of a drug product. Typically, side effects are considered adverse events.

Sucrose

Sucrose is composed of one glucose unit and one fructose sugar unit and is commonly referred to as "table sugar".

Venipuncture

The puncture of a vein to obtain access to the vascular system for any therapeutic purpose, such as obtaining a blood sample or infusion of a diagnostic or therapeutic agent.

Volume

For infused treatments, volume describes the quantity of liquid drug.