INDICATIONS (1 of 2)
REBLOZYL® (luspatercept-aamt) 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 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 under 18 years of age.
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Myelodysplastic syndromes (MDS)
(my-el-o-dis-plastic) and
anemia
Learning about the relationship between MDS and anemia may be helpful to better understand your disease.
What is MDS?
MDS are a group of disorders in which the
bone marrow
fails to make enough healthy
red blood cells (RBCs),
white blood cells (WBCs),
or
platelets
and is considered a blood cancer. Bone marrow is the soft tissue that is found in the center of most bones. Bone marrow is the main place where
blood cells
are made.
What causes MDS?
MDS may be caused by changes to DNA called mutations. These mutations change the way the cells grow and function. With MDS, some newly formed blood cells become abnormal and do not grow (mature) correctly or do not enter the bloodstream to do their job.
Mature
RBC
Abnormal RBC
The abnormal cells take up space needed for normal bone marrow function. This can lead to your body making too few mature blood cells that may not live as long.
Bone Marrow
The pile up in the bone marrow keeps immature RBCs from growing into mature RBCs. Some of these abnormal immature RBCs are called
ring sideroblasts (RS).
Ring Sideroblast
What is the role of RS in MDS?
RS are not fully grown, or immature, RBCs that have a ring of small circular iron clumps around the center structure of the cell where most cells store DNA.
Ring Sideroblasts
How many RS you have is part of diagnosing the type of MDS. You can be diagnosed with
myelodysplastic syndromes with ring sideroblasts (MDS-RS)
if at least 15% (or 15 out of every 100) of your red blood cells are ring sideroblasts.
If you have a specific gene mutation (SF3B1) in your blood cells, an RS percentage of at least 5% is used to diagnose MDS-RS.
RS contribute to having low blood cell counts. That means you have a low number of RBCs.
Ring Sideroblasts
With fewer mature blood cells in your body, you can develop
anemia
(low RBCs). You can also develop other low blood counts.
What are the 3 different types of low blood cell counts (cytopenia)?
Your healthcare team will look to see what type of blood cell count is low.
Anemia: Too few RBCs—these are the blood cells that carry oxygen through your body
Possible symptoms:
- Problems with thinking or memory
- Feeling tired
- Weakness
- Chills
- Dizziness
- Lightheadedness
- Pale or yellowish skin
- Headaches
- Shortness of breath
- Muscle cramps and
bone pains
Neutropenia: Too few WBCs—these are the blood cells that help fight infection
Possible symptoms:
- Frequent infections, fevers, and swollen lymph nodes
Thrombocytopenia: Too few platelets—these are the blood cells that help stop bleeding, like when you get a cut
Possible symptoms:
- Easy bruising and bleeding
- Frequent nosebleeds
- Bleeding gums
Anemia is very common in people with lower-risk MDS. In fact, anemia occurs in up to 9 out of 10 people who have MDS.
How is MDS diagnosed?
Your healthcare team will run certain tests before diagnosing your type of MDS. These tests include taking bone marrow and blood samples to answer the following:
- Do you have cytopenias?
- Do the cells in your bone marrow look normal or abnormal?
- How many abnormal cells are present in the bone marrow?
- How many immature or early forms of blood cells (blast cells) are present in the bone marrow?
- What are the types of blood cells that look abnormal?
- Are there cells that have rings of iron (also called RS)?
- Do the cells in your bone marrow have gene mutations?
Learning you have MDS can be hard. And you may be worried about what your future holds. You may experience a range of thoughts as you come to terms with your MDS diagnosis. Learning about MDS is the first step in taking control.
What happens after an MDS diagnosis?
After your healthcare team has confirmed that you have MDS, they will give you a risk level score. This score helps your healthcare team understand the chances of your MDS progressing. Your risk score is one important factor. However, it’s not the only one. Your doctor will look at other factors, such as your age, your ability to perform daily tasks, and your recent blood test results. If you are concerned about your risk level score, it may help to talk with other people who have MDS, your healthcare team, or even a counselor.
How does my doctor know my risk level score?
Risk level scores are based on a number of factors, including:
- The number of blasts (immature or very early forms of blood cells) in the bone marrow, compared with normal mature cells
- The type and number of mutations in the cells
- The level of RBCs in your blood
- The level of platelets in your blood
- The level of WBCs in your blood
Scores are given to each factor and then added up for an overall risk level score.
Risk level scores can range from “very low” to “very high.” A lower score means a lower risk of your MDS progressing to a more serious condition. That is why it’s important to stay connected with your healthcare team about your risk level score. Even if your score is low, you should still talk to them about your MDS treatment and management.
What are the different treatment approaches for MDS?
Based on your risk level score, your healthcare team will choose a treatment approach. Your doctor will recommend a treatment plan based on the information they have.
There are a number of different ways to treat MDS. Here’s a summary of treatment approaches:
Watch-and-wait method
Watch-and-wait method
Watch and wait means being aware of and monitoring your symptoms and blood work for any changes. You will work closely with your healthcare team and have regular visits with them to discuss any changes. You may find it helpful to track these changes to make sure you don’t miss anything during your visits. Be sure to share any changes with your doctor. If after sharing this information your doctor thinks your MDS is getting worse, you will work together on a treatment option that is best for you. Starting treatment–whether to help with symptoms or MDS directly–can be a little scary. However, it’s important to understand that it’s a step toward managing your MDS.
Supportive care
Supportive care
Focuses on treating your symptoms of MDS. One type of this care is RBC transfusions. RBC transfusions add donated RBCs to your body.
Erythropoiesis stimulating agent (ESA)
Erythropoiesis-stimulating agent (ESA)
An ESA is a man-made
hormone given by injection to raise blood cell counts.
Erythroid maturation agent (EMA)
Erythroid maturation agent (EMA)
An EMA helps by developing more mature, working RBCs.
Active treatments for MDS
Active treatments for MDS
Before using these treatments, your healthcare team must decide that they are right for you. These treatments are available if you are in the low- to very high-risk score range. Examples include medicines such as:
- Chemotherapy: A type of medicine that destroys cancer cells. It works by killing abnormal cells. It can also affect normal cells. There are many different types of chemotherapy drugs
- Immunotherapy: A type of medicine that fights cancer cells by using your immune system
- Immunosuppressive therapy: A medicine that lowers your immune system response and stops it from attacking your bone marrow
- Immunomodulator: A medicine that adjusts different parts of your immune system. This type of therapy boosts the immune system and also helps kill abnormal cells
- Immunosuppressive therapy:
- Stem cell transplant: A process where you receive healthy blood stem cells from a donor to replace your abnormal cells
It’s important to talk openly with your doctor about what you are looking for from a treatment plan. Talk to your healthcare team after starting any treatment. Tell them how you’re feeling and any new or changing symptoms you’ve experienced. Knowing these options can help you talk with your doctor about your plan.
Here are some questions you may want to ask your healthcare team about treating MDS:
Here are some questions you may want to ask your healthcare team about treating MDS:
- What does MDS mean for my future?
- What might my symptoms mean?
- What are the pros and cons of each treatment?
- What are the common side effects of these treatments?
- How will we know if a treatment is working?
- How might not getting treatment impact my symptoms?
- How often should I have appointments with you?
- Are there any other doctors I should see in addition to you?
When it comes to managing your MDS, it’s important for you to feel like an active part of your treatment journey. You are your best advocate. Asking questions along the way to understand your treatment can help you achieve this.
If your doctor chooses RBC transfusions, be sure to write down your hemoglobin (Hgb) levels and track when you are going for your transfusions. You should also write down the number of RBC units in each transfusion. This can help your healthcare team see how your treatment is working. It’s important that you work with your healthcare team along the way. They are there to give you support and to share resources with you that can help manage MDS.