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Patient Brochure


Websites to explore for more information about BT

The following organizations provide disease education, additional support, and expert opinions. This list of independent organizations is provided as an additional resource for obtaining information related to BT. This list does not indicate endorsement by Bristol Myers Squibb of an organization or its communications.


Alpha globin: One of two protein building blocks of hemoglobin

Anemia: Low red blood cell (RBC) count

Beta (β) globin: One of two protein building blocks of hemoglobin

Beta (β) thalassemia (BT): A blood disorder where your body makes less hemoglobin than normal

Blood pressure: The force of circulating blood on the walls of blood vessels

Bone marrow: The soft interior of the bones where new blood cells are created

Chelation therapy: A chemical compound used to remove toxic metals from the body

Erythroid cells: An immature red blood cell

Erythroid maturation agent (EMA): Treatment that helps red blood cells to mature

Erythropoiesis: The formation of red blood cells in blood-forming tissue within the bone marrow

Extramedullary Hematopoietic (EMH) Masses: When blood cells form outside of the bone marrow

Gene: The physical or mental traits that are passed down from parent to child

Hemoglobin (Hgb): Oxygen-carrying protein found in red blood cells

Hormone: A chemical made by your body that travels in the bloodstream to send messages throughout your body

Hydroxyurea: A type of medicine used to treat certain cancers

Immunosuppressant: An agent that decreases the body’s immune response

Ineffective erythropoiesis (IE): The inability of immature red blood cells to become mature

Kilogram (kg): A unit for measuring your weight. One kg is 2.2 pounds

Milligram (mg): A unit for measuring weight

mg/kg: The dose of your medication based on your body weight

Placebo: An inactive substance that looks the same as, and is given the same way as, an active drug or treatment being tested

Protein: An important part of cells that is responsible for the structure, function, and maintenance of your body and organs

Red blood cells (RBCs): Blood cells that carry oxygen from the lungs to all cells in the body

Red blood cell transfusion: A process that adds red blood cells into the bloodstream

Splenectomy: The removal of your spleen through surgery

Stem cell: All immature cells before they mature and are given a specific role

Stem cell transplant: A type of treatment that replaces your abnormal cells with healthy stem cells from a donor

Symptom: A physical or mental sign of a condition or disease

Transfusions: Procedures that add parts of blood or whole blood into the bloodstream

Transfusion burden: How often you need to have red blood cell (RBC) transfusions