Glossary

Acetylsalicyclic Acid — A pain reliever medicine used to treat mild pain and fever. In hemophilia with inhibitors, it is extremely important to avoid taking any aspirin, non-steroidal anti-inflammatory drugs (NSAIDs) — like ibuprofen — or any medications containing ASA (acetylsalicylic acid) without talking to your doctor. Such pain medications might cause bleeding to last longer, so they are best avoided.

Acquired Hemophilia — A rare bleeding disorder. A person is not born with it, but develops it over the course of their lifetime. It develops when antibodies stop factor VIII from working in the blood. Should not be confused with hemophilia with inhibitors, because in the case of hemophilia with inhibitors, a person is born with hemophilia (A or B).

APCCs (Activated Prothrombin Complex Concentrates) — A type of bypassing agent made from human plasma. It helps the body to form a clot and stop bleeding.

Antibody — A type of protein produced by the body's immune system. The immune system makes them when the body believes a harmful substance (like a bacteria or virus) is present.

Arthropathy — Joint disease. In hemophilia with inhibitors, bleeds that aren't treated quickly can lead to joint damage, if they keep happening.

Aura — A sensation that some people with hemophilia get when they start to have a bleed. It can be a strange, hard-to-explain feeling. Since it can happen even before physical signs of a bleed are seen, it's important to trust this sense. Treatment at this stage will help stop bleeding early — and result in less tissue and joint damage.

Bethesda Units (BU) — A unit of measurement. Used to measure the amount (or titer) of inhibitors in the blood.

Blood Clot — A scab that forms over a cut or injury in order to stop bleeding.

Bypassing Agents — Medications that go around (bypass) the clotting factors being blocked by an inhibitor. They help the body to form a clot and stop bleeding.

Central Venous Access — A tube placed in the chest so that it is faster and easier to infuse medicine into the veins located near the heart.

Clotting Factor — Also called coagulation factors. Substances in the blood that help the body to form a clot and stop bleeding.

Clotting Factor Concentrates — Also called high-dose clotting factor concentrates. A medicine used by people with hemophilia to replace the clotting factor missing in their bloodstream and stop bleeding. Also higher-dose concentrates are used by people with hemophilia and low titer levels of inhibitors, in order to overcome the inhibitors and stop a bleed.

Coagulation — The process that causes blood to thicken, form a clot, and stop bleeding.

Complication — A secondary disease or condition that develops in the course of a primary condition, arising as a result of it or from other causes.

Dose — The quantity of medicine that is prescribed and taken at a specific time.

Factor IX (Clotting Factor IX) — A protein in the blood, it is needed for clotting and stopping a bleed. Low levels of Factor IX lead to Hemophilia B.

Factor Replacement Treatment — Treatment that injects clotting factors into the bloodstream of a person with hemophilia. The purpose is to replace missing clotting factors, help form blood clots, and stop bleeds.

(Recombinant) Factor VIIa (rFVIIa) — A synthetic (human-made) bypassing agent.

Factor VIII (Clotting Factor VIII) — A protein in the blood, it is needed for clotting and stopping a bleed. Low levels of Factor VIII lead to Hemophilia A.

Joint Fusion — Surgery for treating arthritis and joint problems. In it, two bones (found on both sides of a joint) are connected directly to each other. This gets rid of the joint pain, by getting rid of the joint itself.

Gene Mutation — A permanent change in the DNA inherited from someone's parents. It can be harmless or lead to the development of disease.

Genetics — The study of heredity. The process that passes a parent's genes on to their children.

Hematologist — A doctor who specializes in the diagnosis, treatment, prevention, and/or investigation of bleeding disorders and other blood disorders and malignancies.

Hemophilia — A medical condition in which the blood does not clot normally, making it harder to stop bleeds when they occur.

Hemophilia Treatment Center — A medical clinic that provides complete care for people with hemophilia.

High Responder — A person whose immune system produces inhibitors of more than 5 Bethesda Units (BU) after receiving infusions of clotting factor concentrates.

High Titer Inhibitor — An inhibitor level of more than 5 Bethesda Units (BU). High titers of inhibitors are faster to destroy infused factor concentrates.

Ibuprofen — A non-steroidal anti-inflammatory drug (NSAID) used to treat minor aches and pains. In hemophilia with inhibitors, it is extremely important to avoid taking any NSAIDs — like ibuprofen — without talking to your doctor. Such pain medications might cause bleeding to last longer, so they are best avoided.

Immune System — The body's defense system that protects a person against bacteria, viruses, and other microorganisms that can cause disease.

Immunologist — A doctor who specializes in the diagnosis, treatment, and management of allergies, asthma, and other disorders related to the immune system.

Infection — A disease that damages body tissue. Caused by the attack of viruses, bacteria, other microorganisms, and the toxins they produce.

Infectious Disease Specialist — A doctor who specializes in the diagnosis and treatment of infectious diseases.

Inflammation — The body's normal protective response to an injury, irritation, or surgery. This natural defense of the body brings increased blood flow to an area. Symptoms of inflammation may include swelling, pain, increased warmth to the touch, and redness of the skin.

Infusion — The administration of clotting factor concentrates.

Inhibitors — A type of protein molecule produced by the immune system, called an antibody. Sometimes antibodies attack clotting factor by mistake. When this happens, it makes hemophilia more difficult to treat.

Joint — A point where two or more bones come together, such as the knee, hip, elbow, or shoulder.

Low Responder — A person whose immune system produces inhibitors of less than 5 Bethesda Units (BU) after receiving an infusion of clotting factor concentrates.

Low Titer Inhibitor — An inhibitor level of less than 5 Bethesda Units (BU). A person with a low inhibitor titer may be able to control bleeding with normal amounts of clotting factor concentrates.

Mild Hemophilia — Usually means Factor VIII or IX levels of 5%–30% of normal, with severe bleeding due to major trauma or surgery.

Moderate Hemophilia — Usually means Factor VIII or IX levels of 1%–5%, with occasional bleeding that is not caused by an injury (also called spontaneous bleeding), or severe bleeding due to trauma or surgery.

Neutralize — To stop something from working.

Non-Factor Therapy — A treatment for people with hemophilia A with factor VIII inhibitors that activates a different part of the coagulation cascade to mimic the action of the missing factor VIII.

Non-steroidal Anti-inflammatory Drugs (NSAIDs) — A drug used to treat minor aches and pains. In hemophilia with inhibitors, it is extremely important to avoid taking any NSAIDs — like ibuprofen — or any medications containing ASA (acetylsalicylic acid) without talking to your doctor. Such pain medications might cause bleeding to last longer, so they are best avoided.

Occupational Therapist — A specialist who treats individuals with injuries, illnesses, or disabilities by training, or retraining them, on everyday activities. They help such individuals develop, recover, and improve the skills needed for daily living and working.

On-demand Therapy — Treatment that is given immediately as soon as it is needed, such as the infusion of factor concentrates to stop a bleeding episode.

Orthopedics — A medical specialty that deals with the diagnosis, treatment, and care of the musculoskeletal system — bones, joints, muscles, ligaments, tendons, nerves, and skin.

Peripheral Venous Access — A way to infuse medication by using veins found in the arms, legs, hands, or feet.

Persistent Inhibitor — Inhibitors that remain over a person's lifetime and do not go away.

Physical Therapist — A health care practitioner who helps people to move properly and increase their strength and flexibility, and treats injuries, balance problems, and movement issues using special exercises.

Rheumatologist — A doctor who specializes in the diagnosis and treatment of arthritis and other diseases of the joints, muscles, and bones.

Risk Factor — Something that increases a person's chances of developing a disease.

Saturated Fats — These fats have a chemical makeup where carbon atoms are saturated with hydrogen atoms. These fats are usually solid at room temperature. Foods high in these fats raise the level of cholesterol in the blood, which can increase the risk of heart disease and stroke. These fats can be found in many foods. Fatty red meat, milk, butter, cheese, baked goods, fried foods, and many oils can contain high levels of this type of fat.

Severe Hemophilia — Usually means Factor VIII or IX levels of less than 1%, with spontaneous bleeds (mostly in joints and muscles).

Spasm (Muscle spasm) — A sudden, painful tightening of a muscle or group of muscles. They usually do not last for more than a few minutes, but the cramping can be severe and painful.

Synovectomy — A surgical procedure that removes inflamed joint tissue in order to reduce pain.

Synovial Capsule — A thin, flexible tissue that surrounds a joint to protect it and helps it get nutrients.

Synovitis — Inflammation of the joint lining (synovium) caused by bleeding into the joint.

Testosterone — A hormone that plays a key role in the development of male characteristics. It promotes and maintains bone density, muscle mass, sperm production, and sex drive.

Titer — The level or concentration amount of a substance in a solution.

Transient Inhibitor — Temporary or short-lived inhibitors that usually disappear.

Urinary Tract — The body's drainage system for producing, storing, and expelling urine. The urinary tract includes two kidneys, two ureters, a bladder, and a urethra.

Vein — Blood vessels that carry blood from tissues back toward the heart.

Venous Access — A pathway to give treatment directly into the bloodstream.

Virus — A microorganism that can cause disease. They are much smaller than bacteria and can make people sick by infecting cells, changing the normal functioning of cells, multiplying quickly, and causing damage.

X Chromosome — One of the two sex chromosomes. A human female normally has a pair of X chromosomes (XX), while a human male normally has one X chromosome and one Y chromosome (XY). In males, one abnormal X chromosome may lead to the development of an abnormal factor VIII or FIX protein, or reduced amounts of those proteins.