MDS - Treatment (OLD) | Aplastic Anemia and MDS International Foundation

MDS - Treatment (OLD)

MDS treatments focus on increasing the number of healthy blood cells and/or reducing the number of abnormal cells.  When blood counts go up, a patient experiences fewer symptoms and requires less treatment. The only potential cure for MDS is a bone marrow transplant, which is not an option for most MDS patients. All other treatments are designed to treat symptoms and extend life.

 

    Basics

    Your doctor considers these factors to determine the best treatment plan for you:

    • Your symptoms
    • Your age
    • Which subtype of MDS you have 
    • Your IPSS (International Prognostic Scoring System) disease risk score
    • Other serious conditions or diseases you have
    • Whether someone is willing and able to donate matching bone marrow to you (preferably a family member)

    Treatments for MDS have a variety of goals. They may:

    • Help healthy cells mature
    • Increase the number of healthy cells in your blood
    • Kill abnormal bone marrow cells 
    • Reduce the number of blasts (young white blood cells) in your bone marrow that have an abnormal shape, size, or appearance.

    There are 6 general approaches to the treatment of MDS depending on the type you have and the severity of your case:

    1. Wait and watch, which might be suggested if your blood counts are not too low and your symptoms are not too bad
    2. Supportive care to help you manage the symptoms of your MDS
    3. Drug Therapy with medicines specifically approved to treat MDS and stop abnormal cells from growing
    4. Immunosuppressive therapy, which can lower your body's immune response
    5. Chemotherapy, using drugs that kill abnormal cells
    6. Bone marrow transplantation to replace damaged bone marrow stem cells with healthy ones

    Wait and Watch

    Your doctor might suggest the wait and watch approach, also called watchful waiting, if your blood counts are not too low and your symptoms are not too bad. In this approach, your doctor will monitor your blood counts and bone marrow to see how they change over time. As blood counts go lower and bone marrow blast counts rise, your doctor will discuss other treatment options.

    Supportive Care

    Supportive care helps you manage the symptoms of your MDS. Its goals are to increase blood counts, treat infections and treat iron overload. Supportive care is often the best treatment for lower-risk MDS patients who:

    • May or may not need blood transfusions, depending on blood cell counts
    • Have a low or intermediate-1 risk IPSS score, which is between 0 and 1.0
    • Do not usually have an excess of blasts in the bone marrow

    This type of care includes:

    • Blood transfusions (red blood cells and platelets)
    • Iron chelation to treat iron overload
    • Growth factors to increase red blood cell counts
    • Antibiotics to prevent or treat infection

    Blood Transfusions

    A blood transfusion is a safe and common procedure. When you receive a transfusion, the cell parts of blood from a donor are put into your bloodstream. This increases your blood counts and reduces symptoms. Most people who have MDS will receive at least one blood transfusion.

    A transfusion usually involves only certain parts of blood. These might be the red blood cells, platelets or plasma. Rarely is whole blood (red cells, plasma, platelets, and white cells) used for a transfusion. A transfusion for red blood cells takes 2 to 4 hours. A platelet transfusion takes 30 to 60 minutes.

    As with any medical procedure, there are some risks. These include the very small chance of having an allergic reaction and an even smaller chance of getting an infection. Symptoms of an allergic reaction might include bleeding or pain at the IV site, fever, chills and hives. Before you receive a transfusion, your healthcare provider will discuss the risks with you.

    Learn more about blood transfusions

    Iron Chelation Therapy for Iron Overload

    Iron overload occurs when there is too much iron in your body. Red blood cells contain iron, so with each red blood cell transfusion, extra iron is stored in your body. If it builds up in your vital organs, it could damage them over time.

    Doctors use these tests to check iron levels:

    • Serum transferrin saturation measures the amount of iron in your blood.
    • Serum ferritin, a protein inside of cells that stores iron for later use by your body that indicates the total amount of iron being stored in your cells.

    If your doctor thinks you have iron overload, she might recommend iron chelation therapy to remove extra iron from your body. There are three iron chelators that are approved by the U.S. Food and Drug Administration (FDA) for use in the United States:

    • Deferoxamine (Desferal®) is usually administered by subcutaneous (under the skin) infusion using a small portable pump about the size of a CD player.
    • Deferasirox (Exjade®) is a newer iron chelating medication that comes in a tablet form. It must be dissolved in juice or water and taken (by mouth) once a day. Deferasirox is not recommended for people with high-risk MDS.
    • Deferasirox (Jadenu ®) is new tablet formulation of deferasirox approved for use in 2015. It is also taken on an empty stomach or with a light meal once a day with water or other liquids. Deferasirox is not recommended for people with high-risk MDS.

    Learn more about iron chelation therapy

    Red cell growth factors

    Red cell growth factor is called erythropoietin or EPO for short. It is a protein made by your kidneys in response to low oxygen levels in the body caused by low red cell counts and anemia. EPO causes your bone marrow to make more red blood cells.

    If you don't have enough natural EPO or if you have anemia, your doctor may prescribe a pharmaceutical form of EPO, such as:

    • Epoetin Alpha (Procrit®, Epogen®):  Epoetin alpha is typically given as an injection (shot) in the arm, abdomen or thigh.
    • Darbepoetin alpha (Aranesp®):  Darbepoetin alpha may be given as an injection (shot) in the arm, abdomen or thigh or by infusion into a vein (intravenous IV).

    Your doctor may ask you to take a white blood cell growth factor along with EPO. This combination may improve red blood cell counts in certain patients.

    These medicines have some risks, including the chance of getting a blood clot. You and your doctor will decide whether the benefits of these drugs outweigh the risks.

    White Blood Cell Growth Factors

    If you don't have enough healthy white cells in your blood, your doctor may ask you to take a white cell growth factor to increase your count and improve your immune system. The most common ones:

    • G-CSF (granulocyte colony-stimulating factors) is sold under the names Filgrastim® and Neupogen®.
    • GM-CSF (granulocyte macrophage colony-stimulating factors) is sold under the names Leukine® and Sargramostim®.

    Doctors are cautious about giving white cell growth factors to MDS patients. Sometimes, white cell growth factors can increase the growth of blast cells in patients with high-risk MDS. This can be a serious problem. For this reason, the use of these growth factors for MDS patients is often limited to the treatment of infections, along with antibiotics.

    Antibiotics

    Many MDS patients get infections easily and have trouble getting rid of them. That’s because they don’t have enough white blood cells (neutrophils) to fight infection, a condition called  neutropenia. Antibiotics will help, but they must be started quickly for patients with low neutrophil counts and fevers.

    If you have MDS and you get a fever, contact your doctor right away or go to the emergency room.

    Drug Therapy

    The FDA has approved three medicines to specifically treat MDS in the U.S. These include azacitidine (Vidaza®) and decitabine (Dacogen®), both considered hypomethylating agents, and lenalidomide (Revlimid®), which is considered an immunomodulatry agent.

    For patients in Europe, note that the European Agency for the Evaluation of Medicinal Products (EMEA) is reviewing these drugs for potential use.

    Hypomethylating Agents

    Hypomethylating Agents help prevent the growth of abnormal bone marrow stem cells in all types of MDS patients.

    Patients receive hypomethylating agent treatments in a clinic or hospital setting. The medicine is fed slowly into the vein through what is called an IV infusion. There are several different regimens, but the most common one involves getting injections for 7 days in a row every 4 weeks.

    It is important to understand that hypomethylating agents may lower blood counts before they start to go up. It can make the treatment difficult for some patients, but it is how these agents work as they set the stage for healthier stem cell development.

    Immunomodulatory Agents:

    Immunomodulatory agents enhance the activity of immune cells and reduce inflammation. It also stops the effects of "bad" chemicals produced in the bone marrow and increases how well certain white blood cells work. These drugs may also slow growth of new blood vessels that feed the MDS cells so they can’t grow:

    Learn more about MDS drug therapy

    Chemotherapy

    The risk of developing Acute Myelogenous Leukemia (AML) depends on the type of MDS you have. As a preventive measure, your doctor may prescribe intensive chemotherapy to kill bone marrow cells that have an abnormal size, shape or look.

    Chemotherapy for MDS patients involves cytotoxic agents which kill cells. They are divided into low-dose and high-dose treatments.

    How does chemotherapy work?

    Chemotherapy kills healthy cells along with abnormal ones, so you may need to stay in the hospital for a few weeks after treatment to receive transfusions of red blood cells and platelets. You will also be given antibiotics  to fight infection.

    Approximately 40 to 50 out of 100 patients have no MDS symptoms after receiving high-dose chemotherapy. But for nearly 90 out of 100 patients, the disease returns within 5 years, and for most, within 2 years. High-dose chemotherapy rarely provides a cure.

    Learn more about chemotherapy.

    Immunosuppressive Therapy

    With some types of MDS, the immune system may prevent the bone marrow from making normal blood cells. Immunosuppressive therapy uses medicines to stop the immune system from attacking the bone marrow. These medicines include ATG (antithymocyte globulin) and cyclosporine.

    Antithymocyte globulin (ATG)

    ATG helps some MDS patients by attacking specific cells in their immune system called T-lymphocytes. This allows bone marrow to rebuild and start making healthy cells. As with any drug used to treat MDS, the beneficial effects of ATG are temporary.

    Cyclosporine

    Cyclosporine prevents T-lymphocytes from becoming active. Once T-lymphocytes are turned off by the cyclosporine, they stop attacking stem cells in the bone marrow. Then the stem cells may be able to grow back and start making healthy blood cells.

    When used alone, cyclosporine is less effective than ATG. When used with ATG, cyclosporine improves blood counts in about 7 out of 10 cases.

    Learn more about immunosupressive therapy.

    Bone Marrow Transplantation

    Each MDS patient’s situation is unique. For some, a stem cell or bone marrow transplant offers the possibility of a cure. If your doctor thinks you may be a candidate for a bone marrow  transplant, you should start looking for a matched donor right away.

    First check within your immediate family for a sibling match, which has a 1 in 4 chance of being a match. If you don’t have a sibling match, you will need to find an matched unrelated donor (MUD) through a bone marrow donor registry like the one managed by the National Marrow Donor Program. Another source of stem cells considered in some cases is frozen umbilical cord blood.

    Once you have a matched donor, steps will include:

    • Receiving a regimen of chemotherapy and/or radiation. This kills off your current bone marrow cells and disables your immune system to prepare it for the donated stem cells.
    • Getting your donated stem cells through a central line. This means delivering them right into the blood stream where they make their way to your bone marrow. There they begin to grow and produce blood cells in a process called engraftment.
    • Until the donated stem cells engraft, you may need blood and/or platelet transfusions to keep your blood cells numbers up. During this time you are also at high risk for infection until your body starts making new white blood cells on its own. Your healthcare team will take extra precautions to make sure you don’t get an infection.

    Learn more about Bone Marrow and Stem Cell Transplantation.

    Clinical Trials

    Clinical research studies, also called clinical trials, are at the heart of all medical advances. They help identify new ways to prevent, detect or treat disease. If you have MDS, you may want to consider taking part in a research study. They offer good treatment options for some MDS patients.

    Learn more about Clinical Trials