Treatments

MDS treatments focus on increasing the number of healthy blood cells and/or reducing the number of abnormal cells. When blood counts go up, patients feels better and need less treatment. A bone marrow transplant is called an "imperfect cure" for MDS , but it isn't an option for most patients and comes with many risks and possible long-term side effects. All other treatments are designed to treat symptoms and extend life.

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.

    Therapies

    Blood Transfusions

    A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some patients with low blood counts.

    Chemotherapy

    If you have MDS and have a high-risk or intermediate-2 risk IPSS score, you have a higher risk of developing Acute Myelogenous Leukemia (AML). To prevent this from happening, your doctor may prescribe intensive chemotherapy in order to kill bone marrow cells that have an abnormal size, shape, or look.

    Growth Factors

    Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts.

    Immunosuppressive Therapy

    Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts.

    Iron Chelation

    Iron chelation therapy is the main treatment used when you have a condition called iron overload. Iron overload means you have too much iron in your body. This can be a problem for people who get lots of red blood cell transfusions.

    MDS Drug Therapy

    There are three medicines approved in the U.S. to treat MDS. Azacitidine (Vidaza®) and decitabine (Dacogen®) are approved to treat both low- and high-risk patients with all sub-types of MDS. Lenalidomide (Revlimid®) is approved for transfusion-dependent MDS patients with isolated del(5q) and with a low or intermediate-1 risk IPSS score.

    Bone Marrow Transplant

    A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due to the risks and potential long-term side effects as an "imperfect cure".

    Clinical Trials

    Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.