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Table of Contents General Information About Myelodysplastic Syndromes
Stages of Myelodysplastic Syndromes
Treatment Option Overview
Treatment Options for Myelodysplastic Syndromes
To Learn More About Myelodysplastic Syndromes
Get More Information From NCI
Changes to This Summary (08/14/2008)
About PDQ
General Information About Myelodysplastic Syndromes
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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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| Blood cell development. A blood stem cell goes through several steps to become a red blood cell, platelet, or white blood cell. |
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:
- Refractory anemia.
- Refractory anemia with ringed sideroblasts.
- Refractory anemia with excess blasts.
- Refractory anemia with excess blasts in transformation.
- Refractory cytopenia with multilineage dysplasia.
- Myelodysplastic syndrome associated with an isolated del(5q) chromosome abnormality.
- Unclassifiable myelodysplastic syndrome.
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:
- 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).
- Fever or frequent infections.
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:
- Physical exam and history: An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient’s health habits and past illnesses and treatments will also be taken.
- Complete blood count (CBC) with differential: A procedure in which a sample of blood is drawn and checked for the following:
- The number of red blood cells and platelets.
- The number and type of white blood cells.
- The amount of hemoglobin (the protein that carries oxygen) in the red blood cells.
- The portion of the blood sample made up of red blood cells.
| Enlarge | | | | Complete blood count (CBC). Blood is collected by inserting a needle into a vein and allowing the blood to flow into a tube. The blood sample is sent to the laboratory and the red blood cells, white blood cells, and platelets are counted. The CBC is used to test for, diagnose, and monitor many different conditions. |
- Peripheral blood smear: A procedure in which a sample of blood is checked for changes in the number, type, shape, and size of blood cells and for too much iron in the red blood cells.
- Cytogenetic analysis: A test in which cells in a sample of blood or bone marrow are viewed under a microscope to look for certain changes in the chromosomes.
- Bone marrow aspiration and biopsy: The removal of bone marrow, blood, and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for abnormal cells.
| Enlarge | | | | Bone marrow aspiration and biopsy. After a small area of skin is numbed, a Jamshidi needle (a long, hollow needle) is inserted into the patient’s hip bone. Samples of blood, bone, and bone marrow are removed for examination under a microscope. |
Myelodysplastic syndromes are diagnosed based on certain changes in the blood cells and bone marrow.
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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.
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Refractory anemia with ringed sideroblasts:
There are too few red blood cells in the blood and the patient has anemia. The red blood cells have too much iron. The number of white blood cells and platelets is normal.
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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 and there are a normal number of blasts found in the blood. There also may be changes to the white blood cells and platelets. Refractory anemia with excess blasts may progress to acute myeloid leukemia. See the PDQ Adult Acute Myeloid Leukemia Treatment summary for more information.
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Refractory anemia with excess blasts in transformation:
There are too few red blood cells, white blood cells, and platelets in the blood and the patient has anemia. Twenty percent to 30% of the cells in the bone marrow are blasts and more than 5% of the cells in the blood are blasts. Refractory anemia with excess blasts in transformation is sometimes called acute myeloid leukemia.
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Refractory cytopenia with multilineage dysplasia:
There are too few of at least two 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. If red blood cells are affected, they may have extra iron. Refractory cytopenia may progress to acute leukemia.
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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.
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Unclassifiable myelodysplastic syndrome:
There are too few of one type of blood cell in the blood. The number of blasts in the bone marrow and blood is normal, and the disease is not one of the other myelodysplastic syndromes.
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis (chance of recovery) depends on the following:
- Whether the myelodysplastic syndrome occurred after chemotherapy or radiation therapy for another disease.
- The number of blast cells in the bone marrow.
- Whether one or more types of blood cells are affected.
- Certain changes in the chromosomes.
Treatment options depend on the following:
- Whether the myelodysplastic syndrome occurred after chemotherapy or radiation therapy for another disease.
- Whether the myelodysplastic syndrome has progressed after being treated.
- The age and general health of the patient.
 Back to Top Stages of Myelodysplastic Syndromes
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.
Back to Top Treatment Option Overview
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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:
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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. 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.
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Growth factor therapy
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.
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Drug therapy
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.
Back to Top Treatment Options for Myelodysplastic Syndromes
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.
De Novo and Secondary Myelodysplastic Syndromes
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.
Previously Treated 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.
Back to Top 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:
Back to Top Get More Information From NCI
Call 1-800-4-CANCER
For more information, U.S. residents may call the National Cancer Institute's (NCI's) Cancer Information Service toll-free at 1-800-4-CANCER (1-800-422-6237) Monday through Friday from 9:00 a.m. to 4:30 p.m. Deaf and hard-of-hearing callers with TTY equipment may call 1-800-332-8615. The call is free and a trained Cancer Information Specialist is available to answer your questions.
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Write to us
For more information from the NCI, please write to this address:
- NCI Public Inquiries Office
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Search the NCI Web site
The NCI Web site provides online access to information on cancer, clinical trials, and other Web sites and organizations that offer support and resources for cancer patients and their families. For a quick search, use our “Best Bets” search box in the upper right hand corner of each Web page. The results that are most closely related to your search term will be listed as Best Bets at the top of the list of search results.
There are also many other places to get materials and information about cancer treatment and services. Hospitals in your area may have information about local and regional agencies that have information on finances, getting to and from treatment, receiving care at home, and dealing with problems related to cancer treatment.
Find Publications
The NCI has booklets and other materials for patients, health professionals, and the public. These publications discuss types of cancer, methods of cancer treatment, coping with cancer, and clinical trials. Some publications provide information on tests for cancer, cancer causes and prevention, cancer statistics, and NCI research activities. NCI materials on these and other topics may be ordered online or printed directly from the NCI Publications Locator. These materials can also be ordered by telephone from the Cancer Information Service toll-free at 1-800-4-CANCER (1-800-422-6237), TTY at 1-800-332-8615.
Back to Top Changes to This Summary (08/14/2008)
The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.
Changes were made to this summary to match those made to the health professional version.
Back to Top About PDQ
PDQ is a comprehensive cancer database available on NCI's Web site.
PDQ is the National Cancer Institute's (NCI's) comprehensive cancer information database. Most of the information contained in PDQ is available online at NCI's Web site. PDQ is provided as a service of the NCI. The NCI is part of the National Institutes of Health, the federal government's focal point for biomedical research.
PDQ contains cancer information summaries.
The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries are available in two versions. The health professional versions provide detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions provide current and accurate cancer information.
The PDQ cancer information summaries are developed by cancer experts and reviewed regularly.
Editorial Boards made up of experts in oncology and related specialties are responsible for writing and maintaining the cancer information summaries. The summaries are reviewed regularly and changes are made as new information becomes available. The date on each summary ("Date Last Modified") indicates the time of the most recent change.
PDQ also contains information on clinical trials.
A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." 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.
Listings of clinical trials are included in PDQ and are available online at NCI's Web site. Descriptions of the trials are available in health professional and patient versions. Many cancer doctors who take part in clinical trials are also listed in PDQ. For more information, call the Cancer Information Service 1-800-4-CANCER (1-800-422-6237); TTY at 1-800-332-8615.
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