How was REBLOZYL studied?

The clinical trial compared REBLOZYL to placebo (no active medication)

  • This trial included adults with ‌β-thalassemia who needed regular red blood cell transfusions of 6 to 20 units every 24 weeks

The main goal of the study was to see if REBLOZYL could reduce transfusion burden by at least one-third (at least 2 red blood cell units) during a set 12-week period (weeks 13–24)

Patients in the clinical trial were divided into 2 groups:

Patients in the clinical trial were divided into 2 groups
Patients in the clinical trial were divided into 2 groups

REBLOZYL was continued for as long as a reduction in transfusions was observed or until unacceptable toxicity.

*Subcutaneous means under the skin.

Patients included in the study needed to:

  • Receive regular red blood cell transfusions (6–20 red blood cell units per 24 weeks) with no transfusion-free period greater than 35 days during that period
  • Be at least 18 years old

Patients could not be a part of the study if they had:

  • Hemoglobin S/β-thalassemia or alpha (α)-thalassemia
  • Major organ damage (liver disease, heart disease, lung disease, poor kidney function)
  • Recent deep vein thrombosis or stroke, or recent use of erythropoiesis-stimulating agent, immunosuppressant, or hydroxyurea therapy

Characteristics of the 336 patients in the clinical trial

Characteristics of the patients in the clinical trial

Characteristics of the patients in the clinical trial

What are the possible benefits of REBLOZYL?

The main goal of the clinical trial

The main goal of the study was to see if REBLOZYL could reduce transfusion burden by at least 
one-third (at least 2 red blood cell units) during a set 12-week period (weeks 13–24).

  • Reducing transfusion burden could mean fewer red blood cell units per transfusion or a reduction in the visits needed for transfusions

Example patient

Results from example patient

Results from example patient

REBLOZYL helped more patients have fewer red blood cell transfusions

Patients who had their red blood cell transfusion burden reduced by one-third during weeks 13 to 24:

Patients who had their red blood cell transfusion burden reduced by one-third

Patients who had their red blood cell transfusion burden reduced by one-third

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 β-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
  • Muscle or bone pain
  • Dizziness
  • Diarrhea
  • Stomach (abdominal) pain
  • Allergic reactions
  • Headache
  • Joint pain (arthralgia)
  • Nausea
  • Cough
  • Trouble breathing

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.

Tell your healthcare team about any side effect(s) you may experience

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.