How was REBLOZYL studied?

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

The main goal of the trial was to eliminate the need for red blood cell transfusions for at least 8 weeks in a row within the first 24 weeks of treatment

The trial included 229 adults with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) or MDS/MPN-RS-T

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
  • Healthcare providers monitored patients for 48 weeks in the trial
  • All patients in both arms were allowed to receive best supportive care, which included red blood cell transfusions as needed

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

*Subcutaneous means under the skin.

Who were the patients in the clinical trial?

Patients included in the study needed to:

  • Receive regular red blood cell transfusions
    • This was defined as receiving 2 or more red blood cell units per 8 weeks

AND

  • Have received prior treatment with an erythropoiesis-stimulating agent (ESA) with a poor response

OR

  • Be unlikely to respond to erythropoiesis-stimulating agents
    • This was defined as having serum erythropoietin levels greater than 200 U/L and not having received an ESA previously

OR

  • Be intolerant of erythropoiesis-stimulating agent treatment

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

  • deletion 5q (del 5q)
  • White blood cell count >13 Gi/L
  • Neutrophils <0.5 Gi/L
  • Platelets <50 Gi/L
  • Prior disease-modifying agent for treatment of MDS

Characteristics of all 229 patients in the clinical trial:

Characteristics of all 229 patients in the clinical trial
Characteristics of all 229 patients in the clinical trial

What are the possible benefits of REBLOZYL?

Almost 4 in 10 patients who received REBLOZYL achieved the primary goal: no red blood cell transfusions for 8 weeks or more within the first 24 weeks of treatment

37.9%
of patients (58/153) receiving
REBLOZYL had no transfusions
versus
13.2%
of patients (10/76) receiving
placebo had no transfusions

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.