Myelodysplastic Syndromes Treatment (PDQ®)

As a National Cancer Institute (NCI)-designated Comprehensive Cancer Center, a core part of our mission is to educate patients and the community about cancer. The following summary is trusted information from the NCI.

General Information About Myelodysplastic Syndromes

A myelodysplastic syndrome is a type of cancer in which the bone marrow does not make enough healthy blood cells and there are abnormal (blast) cells in the blood and/or bone marrow.

In a healthy person, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time.

Anatomy of the bone. The bone is made up of compact bone, spongy bone, and bone marrow. Compact bone makes up the outer layer of the bone. Spongy bone is found mostly at the ends of bones and contains red marrow. Bone marrow is found in the center of most bones and has many blood vessels. There are two types of bone marrow: red and yellow. Red marrow contains blood stem cells that can become red blood cells, white blood cells, or platelets. Yellow marrow is made mostly of fat.

A blood stem cell may become a lymphoid stem cell or a myeloid stem cell. A lymphoid stem cell becomes a white blood cell. A myeloid stem cell becomes one of three types of mature blood cells:

Blood cell development. A blood stem cell goes through several steps to become a red blood cell, platelet, or white blood cell.

In a patient with a myelodysplastic syndrome, the blood stem cells (immature cells) do not become healthy red blood cells, white blood cells, or platelets. These immature blood cells, called blasts, do not work the way they should and either die in the bone marrow or soon after they go into the blood. This leaves less room for healthy white blood cells, red blood cells, and platelets to form in the bone marrow. When there are fewer healthy blood cells, infection, anemia, or easy bleeding may occur.

The different types of myelodysplastic syndromes are diagnosed based on certain changes in the blood cells and bone marrow.

  • Refractory anemia: There are too few red blood cells in the blood and the patient has anemia. The number of white blood cells and platelets is normal.
  • Refractory anemia with ring sideroblasts: There are too few red blood cells in the blood and the patient has anemia. The red blood cells have too much iron inside the cell. The number of white blood cells and platelets is normal.
  • Refractory anemia with excess blasts: There are too few red blood cells in the blood and the patient has anemia. Five percent to 19% of the cells in the bone marrow are blasts. There also may be changes to the white blood cells and platelets. Refractory anemia with excess blasts may progress to acute myeloid leukemia (AML). See the PDQ Adult Acute Myeloid Leukemia Treatment summary for more information.
  • Refractory cytopenia with multilineage dysplasia: There are too few of at least two types of blood cells (red blood cells, platelets, or white blood cells). Less than 5% of the cells in the bone marrow are blasts and less than 1% of the cells in the blood are blasts. If red blood cells are affected, they may have extra iron. Refractory cytopenia may progress to acute myeloid leukemia (AML).
  • Refractory cytopenia with unilineage dysplasia: There are too few of one type of blood cell (red blood cells, platelets, or white blood cells). There are changes in 10% or more of two other types of blood cells. Less than 5% of the cells in the bone marrow are blasts and less than 1% of the cells in the blood are blasts.
  • Unclassifiable myelodysplastic syndrome: The numbers of blasts in the bone marrow and blood are normal, and the disease is not one of the other myelodysplastic syndromes.
  • Myelodysplastic syndrome associated with an isolated del(5q) chromosome abnormality: There are too few red blood cells in the blood and the patient has anemia. Less than 5% of the cells in the bone marrow and blood are blasts. There is a specific change in the chromosome.
  • Chronic myelomonocytic leukemia (CMML): See the PDQ summary on Myelodysplastic/ Myeloproliferative Neoplasms Treatment for more information.

Age and past treatment with chemotherapy or radiation therapy affect the risk of a myelodysplastic syndrome.

Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get a disease; not having risk factors doesn't mean that you will not get a disease. Talk with your doctor if you think you may be at risk. Risk factors for myelodysplastic syndromes include the following:

The cause of myelodysplastic syndromes in most patients is not known.

Signs and symptoms of a myelodysplastic syndrome include shortness of breath and feeling tired.

Myelodysplastic syndromes often do not cause early signs or symptoms. They may be found during a routine blood test. Signs and symptoms may be caused by myelodysplastic syndromes or by other conditions. Check with your doctor if you have any of the following:

  • Shortness of breath.
  • Weakness or feeling tired.
  • Having skin that is paler than usual.
  • Easy bruising or bleeding.
  • Petechiae (flat, pinpoint spots under the skin caused by bleeding).

Tests that examine the blood and bone marrow are used to detect (find) and diagnose myelodysplastic syndromes.

The following tests and procedures may be used:

Certain factors affect prognosis and treatment options.

The prognosis (chance of recovery) and treatment options depend on the following:

  • The number of blast cells in the bone marrow.
  • Whether one or more types of blood cells are affected.
  • Whether the patient has signs or symptoms of anemia, bleeding, or infection.
  • Whether the patient has a low or high risk of leukemia.
  • Certain changes in the chromosomes.
  • Whether the myelodysplastic syndrome occurred after chemotherapy or radiation therapy for cancer.
  • The age and general health of the patient.

Treatment Option Overview

There are different types of treatment for patients with myelodysplastic syndromes.

Different types of treatment are available for patients with myelodysplastic syndromes. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Treatment for myelodysplastic syndromes includes supportive care, drug therapy, and stem cell transplantation.

Patients with a myelodysplastic syndrome who have symptoms caused by low blood counts are given supportive care to relieve symptoms and improve quality of life. Drug therapy may be used to slow progression of the disease. Certain patients can be cured with aggressive treatment with chemotherapy followed by stem cell transplant using stem cells from a donor.

Three types of standard treatment are used:

Supportive care

Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include the following:

  • Transfusion therapy

    Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or treatment. A red blood cell transfusion is given when the red blood cell count is low and signs or symptoms of anemia, such as shortness of breath or feeling very tired, occur. A platelet transfusion is usually given when the patient is bleeding, is having a procedure that may cause bleeding, or when the platelet count is very low.

    Patients who receive many blood cell transfusions may have tissue and organ damage caused by the buildup of extra iron. These patients may be treated with iron chelation therapy to remove the extra iron from the blood.

  • Erythropoiesis-stimulating agents

    Erythropoiesis-stimulating agents (ESAs) may be given to increase the number of mature red blood cells made by the body and to lessen the effects of anemia. Sometimes granulocyte colony-stimulating factor (G-CSF) is given with ESAs to help the treatment work better.

  • Antibiotic therapy

    Antibiotics may be given to fight infection.

Drug therapy

  • Lenalidomide

    Patients with myelodysplastic syndrome associated with an isolated del(5q) chromosome abnormality who need frequent red blood cell transfusions may be treated with lenalidomide. Lenalidomide is used to lessen the need for red blood cell transfusions.

  • Immunosuppressive therapy

    Antithymocyte globulin (ATG) works to suppress or weaken the immune system. It is used to lessen the need for red blood cell transfusions.

  • Azacitidine and decitabine

    Azacitidine and decitabine are used to treat myelodysplastic syndromes by killing cells that are dividing rapidly. They also help genes that are involved in cell growth to work the way they should. Treatment with azacitidine and decitabine may slow the progression of myelodysplastic syndromes to acute myeloid leukemia.

  • Chemotherapy used in acute myeloid leukemia (AML)

    Patients with a myelodysplastic syndrome and a high number of blasts in their bone marrow have a high risk of acute leukemia. They may be treated with the same chemotherapy regimen used in patients with acute myeloid leukemia.

Chemotherapy with stem cell transplant

Stem cell transplant is a method of giving chemotherapy and replacing blood-forming cells destroyed by the treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of a donor and are frozen for storage. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.

This treatment may not work as well in patients whose myelodysplastic syndrome was caused by past treatment for cancer.

Stem Cell TransplantStem cell transplant (Step 1). Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm.Stem cell transplant (Step 2). The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown).Stem cell transplant (Step 3). The patient receives stem cells through a catheter placed into a blood vessel in the chest.

New types of treatment are being tested in clinical trials.

Information about clinical trials is available from the NCI Web site.

Patients may want to think about taking part in a clinical trial.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.

Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.

Patients can enter clinical trials before, during, or after starting their treatment.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.

Clinical trials are taking place in many parts of the country. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI's listing of clinical trials.

Follow-up tests may be needed.

Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests. This is sometimes called re-staging.

Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.

Treatment Options for Myelodysplastic Syndromes

Standard Treatment Options for Myelodysplastic Syndromes

Standard treatment options for myelodysplastic syndromes include:

Treatment of Therapy-Related Myeloid Neoplasms

Patients who were treated in the past with chemotherapy or radiation therapy may develop myeloid neoplasms related to that therapy. Treatment options are the same as for other myelodysplastic syndromes.

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with adult myelodysplastic syndromes. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI Web site.

Treatment Options for Relapsed or Refractory Myelodysplastic Syndromes

There is no standard treatment for refractory or relapsed myelodysplastic syndromes. Patients whose cancer does not respond to treatment or has come back after treatment may want to take part in a clinical trial.

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with adult myelodysplastic syndromes. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your doctor about clinical trials that may be right for you. General information about clinical trials is available from the NCI Web site.

To Learn More About Myelodysplastic Syndromes

For more information from the National Cancer Institute about myelodysplastic syndromes, see the following:

For general cancer information and other resources from the National Cancer Institute, see the following:



Cancer Care Services

Clinical Trials