Myelodysplastic syndromes are a group of diseases in which the bone marrow does not make enough healthy blood cells.
Myelodysplastic syndromes are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that develop into mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. The lymphoid stem cell develops into a white blood cell. The myeloid stem cell develops into one of three types of mature blood cells:
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In myelodysplastic syndromes, the blood stem cells do not mature into healthy red blood cells, white blood cells, or platelets. The immature blood cells, called blasts, do not function normally and either die in the bone marrow or soon after they enter the blood. This leaves less room for healthy white blood cells, red blood cells, and platelets to develop in the bone marrow. When there are fewer blood cells, infection, anemia, or easy bleeding may occur.
There are several types of myelodysplastic syndromes.
Myelodysplastic syndromes have too few of one or more types of healthy blood cells in the bone marrow or blood. Myelodysplastic syndromes include the following diseases:
See the following PDQ summaries for information about other blood cell diseases:
Age and past treatment with chemotherapy or radiation therapy affect the risk of developing 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. People who think they may be at risk should discuss this with their doctor. Risk factors for myelodysplastic syndromes include the following:
Possible signs of myelodysplastic syndrome include feeling tired and shortness of breath.
Myelodysplastic syndromes often do not cause early symptoms and are sometimes found during a routine blood test. Other conditions may cause the same symptoms. A doctor should be consulted if any of the following problems occur:
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:
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Myelodysplastic syndromes are diagnosed based on certain changes in the blood cells and bone marrow.
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis (chance of recovery) depends on the following:
Treatment options depend on the following:
There is no staging system for myelodysplastic syndromes. Treatment is based on whether the disease developed after the patient was exposed to factors that cause myelodysplastic syndrome or whether the disease was previously treated. Myelodysplastic syndromes are grouped for treatment as follows:
De novo myelodysplastic syndromes
De novo myelodysplastic syndromes develop without any known cause.
Secondary myelodysplastic syndromes
Secondary myelodysplastic syndromes develop after the patient was treated with chemotherapy or radiation therapy for other diseases or after being exposed to radiation or certain chemicals that are linked to the development of myelodysplastic syndromes. Secondary myelodysplastic syndromes may be harder to treat than de novo myelodysplastic syndromes.
Previously treated myelodysplastic syndromes
The myelodysplastic syndrome has been treated but has not gotten better.
There are different types of treatment for patients with myelodysplastic syndromes.
Different types of treatments 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 a myelodysplastic syndrome. 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 aims to relieve symptoms, slow progression, and improve quality of life.
Treatment options for patients with myelodysplastic syndromes range from supportive care that helps relieve symptoms to aggressive treatment that may slow or prevent progression of the disease.
Problems caused by low blood cell counts, such as fatigue and infections, may be treated with transfusions of blood products or the use of growth factors.
Chemotherapy may be used to delay progression of the disease. Other drug therapy may be used to lessen the need for transfusions. Certain patients may benefit from aggressive treatment with chemotherapy followed by stem cell transplant using stem cells from a donor.
Three types of standard treatment are used:
Chemotherapy
In myelodysplastic syndromes, chemotherapy is a treatment that uses drugs to stop the growth of immature blood cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the spinal column, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the disease being treated.
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 (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or treatment. Patients who receive frequent red blood cell transfusions may have their tissues and organs damaged from the buildup of extra iron. Iron chelation therapy is a treatment that uses drugs that attach to the extra iron. The drug and the iron are removed from the body in the urine.
Platelet transfusions are usually given when the patient is bleeding or is having a procedure that may cause bleeding.
Erythropoietin may be given to increase the number of red blood cells and lessen the effects of anemia. Sometimes granulocyte colony-stimulating factor (G-CSF) is given with erythropoietin to help the treatment work better.
Deferoxamine may be used to treat the build-up of too much iron in the blood of patients receiving blood transfusions. It is sometimes given with vitamin C.
Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndrome caused by a specific chromosome change.
Antithymocyte globulin (ATG) may also be used to lessen the need for transfusions in patients with a certain form of myelodysplastic syndrome.
Antibiotics may be given to fight infections.
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.
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 medical research process. Clinical trials are done to find out if new treatments are safe and effective or better than the standard treatment.
Many of today's standard treatments for disease 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 diseases 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 disease has not gotten better. There are also clinical trials that test new ways to stop a disease from recurring (coming back) or reduce the side effects of 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 clinical trials database.
Follow-up tests may be needed.
Some of the tests that were done to diagnose the disease 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.
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 disease has recurred (come back). These tests are sometimes called follow-up tests or check-ups.
A link to a list of current clinical trials is included for each treatment section. Check with your doctor for clinical trials that are not listed here but may be right for you.
Treatment of de novo and secondary myelodysplastic syndromes may include the following:
Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with de novo myelodysplastic syndromes and secondary myelodysplastic syndromes.
Treatment of previously treated myelodysplastic syndromes may include the following:
Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with previously treated myelodysplastic syndromes.
For more information from the National Cancer Institute about myelodysplastic syndromes, see the following:
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Changes were made to this summary to match those made to the health professional version.
2005-02-18 Last Modified: 2008-08-14