About MDS

MDS are a group of disorders that affect the blood and bone marrow

Your bone marrow makes all of your blood cells

Bone marrow produces immature blood cells that typically grow into 3 types of fully functional, mature blood cells. The cell types include:

  • Red blood cells (RBCs), which transport oxygen throughout the body
  • White blood cells (WBCs), which help fight infection
  • Platelets, which help with blood clotting

Bone marrow produces immature blood cells that typically grow into 3 types of fully functional, mature blood cells. The cell types include:

  • Red blood cells (RBCs), which transport oxygen throughout the body
  • White blood cells (WBCs), which help fight infection
  • Platelets, which help with blood clotting

Normal blood cell development

Normal blood cell development
Normal blood cell development

Patients with MDS have different risk levels

Types of MDS vary from very low to high risk based on several factors. Very low- to intermediate-risk MDS is the most common type of MDS, which means that the MDS is at low risk of possibly getting worse. It is based on a prognostic score that your healthcare provider uses to help decide how to manage your MDS.

Certain types of MDS have ring sideroblasts

Ring sideroblasts are a type of immature red blood cells that are ringed with iron deposits. Ring sideroblasts are part of the diagnostic workup taken from your bone marrow or blood. Ring sideroblasts are found in certain types of disease states, including anemia associated with MDS-RS and MDS/MPN-RS-T.

How does anemia in MDS occur?

In MDS, the bone marrow does not make enough mature blood cells

This lack of mature blood cells can occur from a change to DNA in bone marrow cells, called a mutation. In MDS, your bone marrow does not totally stop working. Instead, the blood cells produced by the bone marrow do not develop correctly.

In MDS, not enough immature blood cells become mature or the blood cells may not survive as long. These immature cells are not able to enter the bloodstream. This means you have fewer mature blood cells working in the body, leading to anemia, neutropenia, and/or thrombocytopenia.

Abnormal blood cell development

Abnormal blood cell development

Abnormal blood cell development

Anemia occurs in up to 90% of people who have MDS

Normally, mature red blood cells are produced through a process called erythropoiesis. When this process does not function properly, anemia may occur.

Anemia is the most common type of low blood cell count in people with lower-risk MDS, in which not enough immature red blood cells (called erythroid cells) mature into fully functional red blood cells. If you have anemia, you may feel tired, weak, or have pale skin. Your healthcare provider will measure your hemoglobin levels to help determine if you have anemia. Hemoglobin is the protein in red blood cells that carries oxygen.

Understanding anemia

Anemia occurs in patients with ‌MDS in 3 ways:

Three ways anemia occurs in patients

Three ways anemia occurs in patients

Why people with MDS have too few mature, working red blood cells

As stated earlier, mature red blood cells are produced through a process called erythropoiesis. In people with MDS, not enough 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

How anemia may be managed

If your anemia is severe enough, you may require red blood cell transfusions. Red blood cell transfusions add new red blood cells to your body from a donor. They temporarily replace your missing mature red blood cells and help increase hemoglobin, but do not help your body produce more red blood cells.

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.