REBLOZYL® (luspatercept-aamt) is a prescription medicine used to treat anemia (low red blood cells) in adults with beta thalassemia who need regular red blood cell (RBC) transfusions.
REBLOZYL is not for use as a substitute for RBC transfusions in people who need immediate treatment for anemia.
It is not known if REBLOZYL is safe or effective in children.
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Our bodies make blood cells in the bone marrow, a soft tissue that is found in the center of most bones.
Erythropoiesis [ih-rith-roh-poi-ee-sis] is the process of immature blood cells maturing into healthy red blood cells (RBCs). When something goes wrong with this process and it doesn’t work, it is called ineffective erythropoiesis (IE).
IE can cause many different symptoms in people living with BT, including:
Anemia (low RBC count)
Higher chance of developing blood clots
Abnormalities in your bones
The bodies of people with BT may not make hemoglobin (Hgb) correctly. Hgb is an important part of an RBC, a type of blood cell, that carries oxygen through your body. Normal, healthy Hgb includes 2 sets of alpha globin and beta (β) globin. While these proteins are different, both are considered building blocks of Hgb. If your body doesn’t make enough of either one of the proteins, RBCs do not form the right way. That means they cannot carry the oxygen your organs need to stay healthy. This could result in anemia (low RBCs), which may be severe enough to require red blood cell (RBC) transfusions.
What it means to have BT may vary from person to person. Because people with BT can’t make enough working RBCs, they may need RBC transfusions regularly to manage their anemia. Transfusions add new, working RBCs to your body from a donor. They temporarily replace RBCs and help increase Hgb, but do not help your body produce more.
RBCs are rich in iron, so repeated transfusions may result in iron collecting abnormally in organs like your heart, liver, spleen, or endocrine glands. This buildup can prevent these organs from working properly, so a treatment called a “chelating agent” is often used to remove this excess iron.
Some types of BT may require RBC transfusions. These include:
People with this type of BT have anemia symptoms that can range from mild to severe. Many people eventually require regular RBC transfusions.
BT major (Cooley’s anemia)
BT major (Cooley’s anemia)
People with this type of BT start showing anemia symptoms as a baby. They need regular RBC transfusions and ongoing medical care.
Based on the severity of a person’s transfusion burden, they may refer to their BT as being either transfusion dependent or non-transfusion dependent.