This information represents the views of the doctors and nurses serving on the American Cancer Society's Cancer Information Database Editorial Board. These views are based on their interpretation of studies published in medical journals, as well as their own professional experience.

The treatment information in this document is not official policy of the Society and is not intended as medical advice to replace the expertise and judgment of your cancer care team. It is intended to help you and your family make informed decisions, together with your doctor.

Your doctor may have reasons for suggesting a treatment plan different from these general treatment options. Don't hesitate to ask him or her questions about your treatment options.

Myelodysplastic syndrome (MDS) isn't just one disease; it is a group of diseases. The different types of MDS vary in their outlook and response to treatment. Treatment is based on the type of MDS, as well as the patient's age and health. Treatment is given by a specialist such as a cancer doctor (oncologist) or a doctor who treats blood diseases (hematologist).

Chemotherapy

Chemotherapy (most often called chemo) is the use of drugs, taken by mouth or injected into a vein, to treat cancer. The drugs enter the bloodstream and reach most places in the body. The purpose of the chemo is to kill the abnormal stem cells and allow normal ones to grow back.

While chemo drugs kill cancer cells, they can also damage normal cells, causing side effects. These side effects depend on the type of drugs given, the dose, and how long they are given.

Short-term side effects can include:

• hair loss (the hair grows back once treatment ends)
• mouth sores
• loss of appetite
• nausea
• vomiting
• greater chance of infection (from a shortage of white blood cells)
• easy bruising and bleeding (from low platelet counts)
• tiredness, called fatigue (from a shortage of red blood cells)

The doctor will watch carefully for all side effects and adjust treatment as needed. Your health care team often can suggest ways to lessen side effects. For example, other drugs can be given along with the chemo to prevent or reduce nausea and vomiting.

If a patient's white blood cell counts are very low during treatment, the risk of infection can be reduced by doing certain things. Patients should be very careful about washing their hands and not eating fresh, uncooked fruit and other foods that might carry germs. They might also wear a surgical mask around crowds. The doctor might also suggest giving strong antibiotics even before an infection has started.

If platelet counts are low, patients might have platelet transfusions--or red blood cell transfusions if their red cell counts are low.

Immune treatments

The drugs thalidomide and lenalidomide (Revlimid^®) belong to the class of drugs known as immunomodulating drugs (or IMiDs). Thalidomide was used first in treating MDS. It helped some patients, but many people stopped taking the drug because of side effects. Lenalidomide is a newer drug that has fewer side effects.

Side effects include:

• decreased blood counts (most often the white cell count and platelet count)
• diarrhea or constipation
• feeling tired and weak

Lenalidomide can also increase the risk of serious blood clots that start in the veins in the legs. Part of a clot can break off and travel to the lungs where it can cause problems with breathing or even death. Many experts feel that patients getting this drug should also get some kind of treatment to prevent blood clots.

Both of these drugs are only available through program run by the companies that makes them.

Immunosuppression: Drugs that suppress or weaken the immune system can help some patients with MDS. A drug called anti-thymocyte globulin (ATG) has helped some people, usually younger ones, with MDS. It must be given in the hospital because it can sometimes cause severe allergic reactions leading to low blood pressure and problems breathing. Another drug that works by suppressing the immune system is called cyclosporine. It was first used to block immune responses in people who have had organ or bone marrow transplants, but it has helped some patients with MDS. Side effects of cyclosporine include loss of appetite and kidney damage.

Growth factors

Growth factors are substances that speed up the making of blood cells in the bone marrow. The body itself makes growth factors, but scientists have also learned how to make them in the lab. They can then be given to patients to help their bodies make more blood cells. Patients usually get the growth factors through shots (injections) under the skin. Your health care team can give the injections, or you or your family members can learn to give them.

A shortage of blood cells causes most of the symptoms in people with MDS, so giving growth factors can help some patients. There are several different growth factors that might be used, depending on the patient's situation.

Supportive treatment

For many patients with MDS, the risk of the disease changing into leukemia is low. The main goal of treatment for these patients is to prevent problems from low blood counts. These patients might be very tired because of low red blood cell counts and may need to get many blood transfusions. One problem from a lot of blood transfusions is the build-up of iron in the blood. Too much iron can cause liver and heart damage. There are drugs that can be given to help prevent this.

Patients with low white blood cell counts might get infections which could be very serious. These infections are treated with antibiotics.

Stem cell transplant

A stem cell transplant (SCT) is the only treatment that can cure MDS. In this treatment, the patient gets very strong chemo and perhaps radiation to kill cells in the bone marrow. Once the bone marrow cells are destroyed, the patient gets new blood-forming stem cells. For patients with MDS, these new stem cells come from a donor—often a brother or sister. This is called an allogeneic stem cell transplant. If there is no matching family member to be a donor, then a matched, unrelated donor is used.

How the process works

Most often, stem cells are taken from the donor's blood in a process called apheresis. The stem cells are frozen and stored. The patient then gets high-dose chemo and, often, radiation treatment. This destroys the MDS cells, but it also kills the normal cells in the bone marrow.

The donor's stem cells are then given to the patient as a transfusion. These cells travel through the blood to the bone marrow where they settle and start to grow. After a time they begin making white blood cells, then platelets and, finally, red blood cells.

There is a newer type of transplant, called a mini-transplant, that is now being tried. It differs from the standard approach in that low doses of chemo or radiation are used. Side effects, though, are still a major problem with this low-dose method.

Side effects

This treatment can have serious, even fatal, side effects and is rarely used in older patients. Because of the side effects, many doctors only use SCT in people younger than 50.

The side effects of SCT can be divided into early and long-term effects. Early on, the side effects are the same as those caused by high-dose chemo. Those side effects that occur later can last for a long time. They include:

• radiation damage to the lungs (this is rare)
• damage to the ovaries that can result in infertility, sudden change of life (menopause), and hot flashes
• damage to the thyroid gland
• cataracts (clouding of the eye that can cause vision problems)

The most serious side effect from allogeneic transplant is called graft-versus-host disease (or GVHD). This happens when the new immune cells (from the donor) see the patient's tissues as foreign and so attack them. GVHD can happen early on or it can start later. Symptoms include weakness, tiredness, dry mouth, rashes, infection, diarrhea, and muscle aches. Drugs to suppress the immune system are given as part of the transplant to prevent or treat GVHD.

If you would like more detailed information on SCT, please see the American Cancer Society document Bone Marrow & Peripheral Blood Stem Cell Transplants, available at 1-800-ACS-2345 (1-800-227-2345).

Last Medical Review: 04/15/2009
Last Revised: 04/15/2009