About β‑thalassemia

β-thalassemia is an inherited blood disorder

β-thalassemia (BAY‑tah THAL‑a‑SEE‑mee‑ah) is an inherited condition, which means it is passed down from parents to children. It is a disorder that can lead to anemia, which is a low number of red blood cells or a low amount of hemoglobin, the oxygen-carrying molecule in red blood cells. Roughly 55% of patients are older than 20 years (average age 23.2 years).

What causes β‑thalassemia?

In people with β‑thalassemia, hemoglobin may not be made correctly. Normal, healthy hemoglobin includes 2 sets of 2 different proteins: an alpha and a beta. In many people with β-thalassemia, “beta globin” proteins are not formed correctly. If your body does not produce enough of either protein, red blood cells do not form properly and cannot carry the oxygen your organs need to stay healthy. This could result in anemia, which may be severe enough to require red blood cell transfusions.

β-thalassemia can cause symptoms of anemia and other conditions

If you have anemia, you may feel tired, weak, or have pale skin. Your doctor will measure hemoglobin to help determine if you have anemia. When you have anemia due to β-thalassemia, there can be complications, such as an enlarged spleen or brittle bones.

  • If anemia causes the spleen to become enlarged, some patients with β‑thalassemia will have it removed through a surgery called a splenectomy

Types of β-thalassemia

Many people with β-thalassemia rely on red blood cell transfusions

Red blood cell transfusions are a primary option for treating anemia in people with β-thalassemia. Transfusions add new, working red blood cells to your body from a donor. They temporarily replace red blood cells and help increase hemoglobin, but do not help your body produce more.

Red blood cells 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.

Types of β-thalassemia that may require red blood cell transfusions

Types of β-thalassemia that may require red blood cell transfusions

Types of β-thalassemia that may require red blood cell transfusions

Understanding anemia

Anemia occurs in patients with β‑thalassemia in 3 ways:

Three ways anemia occurs in patients

Three ways anemia occurs in patients

Why some people with β‑thalassemia have too few mature, working red blood cells

Mature red blood cells are produced in bone marrow through a process called erythropoiesis. In people with β‑thalassemia, not enough immature red blood cells (erythroid cells) are able to mature and leave the bone marrow. These erythroid cells are unable to do the job of fully working, mature red blood cells, which is to carry oxygen throughout the body.

  • When erythroid cells start to pile up in the bone marrow, they can prevent mature, working red blood cells from developing

the term for when red blood cells are unable to fully mature or develop before leaving the bone marrow is

the term for when red blood cells are unable to fully mature or develop before leaving the bone marrow is

What is REBLOZYL® (luspatercept-aamt)?

REBLOZYL 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.
  • myelodysplastic syndromes with ring sideroblasts (MDS-RS) or myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) who need regular RBC transfusions and have not responded well to or cannot receive an erythropoiesis stimulating agent (ESA).

REBLOZYL is a prescription medicine used to treat anemia (low red blood cells) in adults with myelodysplastic syndromes with ring sideroblasts (MDS-RS) or myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) who need regular RBC transfusions and have not responded well to or cannot receive an erythropoiesis stimulating agent (ESA).

REBLOZYL 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.

Before receiving REBLOZYL, tell your healthcare provider about all of your medical conditions, including if you:

  • have or have had blood clots
  • have or have had high blood pressure (hypertension)
  • take hormone replacement therapy or birth control pills (oral contraceptives)
  • have had your spleen removed (splenectomy)
  • smoke
  • are pregnant or plan to become pregnant. REBLOZYL may harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with REBLOZYL.
  • Females who are able to become pregnant:

    • Your healthcare provider should do a pregnancy test before you start treatment with REBLOZYL.
    • You should use effective birth control (contraception) during treatment with REBLOZYL and for at least 3 months after the last dose.
  • are breastfeeding or plan to breastfeed. It is not known if REBLOZYL passes into your breast milk.
    • Do not breastfeed during treatment with REBLOZYL and for 3 months after the last dose. Talk to your healthcare provider about the best way to feed your baby during this time.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of REBLOZYL?

REBLOZYL may cause serious side effects, including:

  • Blood clots. Blood clots in the arteries, veins, brain, and lungs have happened in people with beta thalassemia during treatment with REBLOZYL. The risk of blood clots may be higher in people who have had their spleen removed or who take hormone replacement therapy or birth control (oral contraceptives). Call your healthcare provider or get medical help right away if you have any of these symptoms:
    • chest pain
    • trouble breathing or shortness of breath
    • pain in your leg, with or without swelling
    • a cold or pale arm or leg
    • sudden numbness or weakness that are both short-term or continue to happen over a long period of time, especially on one side of the body
    • severe headache or confusion
    • sudden problems with vision, speech, or balance (such as trouble speaking, difficulty walking, or dizziness)
  • High blood pressure. REBLOZYL may cause an increase in your blood pressure. Your healthcare provider will check your blood pressure before you receive your REBLOZYL dose. Your healthcare provider may prescribe you medicine to treat high blood pressure or increase the dose of medicine you already take to treat high blood pressure, if you develop high blood pressure during treatment with REBLOZYL.

The most common side effects of REBLOZYL include:

  • tiredness
  • headache
  • muscle or bone pain
  • joint pain (arthralgia)
  • dizziness
  • nausea
  • diarrhea
  • cough
  • stomach (abdominal) pain
  • trouble breathing
  • allergic reactions

REBLOZYL may cause fertility problems in females. This could affect your ability to become pregnant. Talk to your healthcare provider if this is a concern for you.

These are not all of the possible side effects of REBLOZYL. Call your doctor for medical advice about side effects. You are encouraged to report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information and Patient Information for REBLOZYL.