ON THIS PAGE: You will learn about the different types of treatments doctors use for people with myelodysplastic syndrome (MDS). Use the menu to see other pages.
This section explains the types of treatments, also known as therapies, that are the standard of care for MDS. “Standard of care” means the best treatments known. Information in this section is based on medical standards of care for MDS in the United States. Treatment options can vary from one place to another.
When making treatment plan decisions, you are encouraged to discuss with your doctor whether clinical trials offer additional options to consider. A clinical trial is a research study that tests a new approach to treatment. Doctors learn through clinical trials whether a new treatment is safe, effective, and possibly better than the standard treatment. Clinical trials can test a new drug, a new combination of standard treatments, or new doses of standard drugs or other treatments. Clinical trials are an option for all stages of cancer. Your doctor can help you consider all your treatment options. To learn more about clinical trials, see the About Clinical Trials and Latest Research sections.
How MDS is treated
For MDS, different types of doctors who specialize in cancer, called oncologists, and blood disorders, called hematologists, often work together to create a patient’s overall treatment plan that combines different types of treatments. This is called a multidisciplinary team. Your health care team may include other health care professionals, such as physician assistants, nurse practitioners, nurses, social workers, pharmacists, counselors, dietitians, physical therapists, occupational therapists, and others. Learn more about the clinicians who provide cancer care.
Treatment options and recommendations depend on several factors, including the subtype and risk score of MDS, the risk of developing AML, possible side effects, and your preferences, age, and overall health. The goal of treatment may differ based on these factors. For lower-risk disease, the goal to bring blood counts back to healthy levels. For higher-risk disease, the goals of treatment are to prevent or slow the disease from developing into AML and control the disease in the bone marrow. As explained in the Subtypes and Classification section, a patient’s risk score and MDS subtype help doctors determine when treatment should begin. It is important to note that the treatments may not be equally effective for every patient.
Take time to learn about all of your treatment options and be sure to ask questions about things that are unclear. Talk with your doctor about the goals of each treatment and what you can expect while receiving the treatment. These types of conversations are called "shared decision-making." Shared decision-making is when you and your doctors work together to choose treatments that fit the goals of your care. Shared decision-making is important for MDS because there are different treatment options. Learn more about making treatment decisions.
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Some people with MDS who do not have any symptoms may only need close monitoring, including measuring blood counts regularly.
Some people with low-risk MDS are treated with hematopoietic growth factors. Many of the symptoms of MDS are caused by a lack of blood cells. Growth factors are a natural substance made by the body that help the bone marrow make red blood cells, white blood cells, and platelets. Growth factors can be made in a pharmaceutical lab. They may be injected beneath the skin or given as oral medications. Examples of growth factors include:
Red blood cell growth factors
White blood cell growth factors, also known as granulocyte colony stimulating factors or G-CSF
Platelet growth factors, also known as TPO-mimetics
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Red blood cell transfusions and platelet transfusions can help relieve the symptoms caused by anemia and thrombocytopenia. Almost everyone with MDS has anemia and common symptoms include fatigue, shortness of breath, and headache. Thrombocytopenia, which is when there are too few platelets in the blood, is also common for people with MDS. Thrombocytopenia can increase a person's risk of bleeding. Most people with MDS will need blood transfusions at some point.
During a blood transfusion, donated blood or blood components are given through a tube that is inserted directly into a vein with a needle. Blood or blood components flow through the tube and into the vein. Blood transfusions are done in an outpatient setting, meaning they are done at the doctor's office or clinic. They may take from 1 to 3 hours to complete.
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Therapies using medication
The treatment plan for MDS may include medications to destroy unhealthy cells. Medication may be given through the bloodstream to reach those cells throughout the body. When a drug is given this way, it is called systemic therapy.
This treatment is generally prescribed by a medical oncologist, a doctor who specializes in treating cancer with medication, or a hematologist, a doctor who specializes in treating blood disorders.
Medications are often given through an intravenous (IV) tube placed into a vein using a needle or as a pill or capsule that is swallowed (orally). If you are given oral medications to take at home, be sure to ask your health care team about how to safely store and handle them.
The types of medications used for MDS include:
Each of these types of therapies is discussed below in more detail. A person may receive a single type of medication at a time, or a combination of medications given at the same time. Medication can also be given as part of a treatment plan that includes other types of therapies.
The medications used to treat MDS are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications.
It is also important to let your doctor know if you are taking any other prescription or over-the-counter medications or supplements. Herbs, supplements, and other drugs can interact with cancer medications, causing unwanted side effects or reduced effectiveness. Unless a vitamin deficiency exists, there is very little evidence that supplemental vitamins are helpful in MDS. Learn more about your prescriptions by using searchable drug databases.
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Chemotherapy is the use of drugs to destroy unhealthy cells, usually by ending those cells’ ability to grow and divide.
A chemotherapy regimen, or schedule, usually consists of a specific number of cycles given over a set period of time. A patient may receive 1 drug at a time or a combination of different drugs given at the same time. Which drug is given often based on information about the MDS subtype and classification.
Common drugs for MDS include:
Both azacitidine and decitabine are approved by the U.S. Food and Drug Administration (FDA) to treat all types of MDS. However, these drugs are used most often for patients with higher IPSS-R scores. Both can be given in the doctor’s office or clinic. Cedazuridine is an oral medication. Patients often need more than 1 round of treatment, given monthly, before it starts improving their health.
Conventional chemotherapy drugs for more intensive treatment
People with high-risk subtypes of MDS who have an increased risk of developing AML may benefit from conventional chemotherapy. Overall, about 30% to 40% of patients may benefit from chemotherapy for MDS. Your doctor will consider factors such as your age and health before making any recommendations for conventional chemotherapy.
Immunomodulatory drugs (IMiD)
Lenalidomide is taken by mouth, or orally. It is very effective when given to patients with low-risk MDS and chromosome 5q abnormalities. Therefore, it is very important for doctors to find out whether a patient has this chromosomal change through testing.
In general, the side effects of drug therapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, hair loss, loss of appetite, and diarrhea. These side effects usually go away after treatment is finished.
Learn more about the basics of chemotherapy.
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Immunotherapy uses the body's natural defenses to fight cancer by improving your immune system's ability to attack cancer cells. Although this is rarely used for MDS, it may be an option for some patients. A type of immunotherapy for MDS is antithymocyte globulin (ATGAM, Thymoglobulin).
Different types of immunotherapy can cause different side effects. Common side effects include skin reactions, flu-like symptoms, diarrhea, and weight changes. Talk with your doctor about possible side effects for the immunotherapy recommended for you. Learn more about the basics of immunotherapy.
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Bone marrow transplantation/stem cell transplantation
High-dose chemotherapy with bone marrow/stem cell transplantation is the only current treatment that can produce a long-term remission. However, transplantation is a higher-risk treatment and may not be recommended for patients who are older and/or have other medical problems. However, for patients ages 50 to 75, an ALLO transplant may be an option after reduced intensity treatment. Before recommending a transplant, your doctor will talk with you about the risks of this treatment. They will also consider several other factors, such as the subtype of MDS, results of any previous treatment, and your general health.
A bone marrow transplant is a medical procedure in which bone marrow that contains cancer is destroyed and replaced by highly specialized normal, healthy cells. These cells, called hematopoietic stem cells, develop into healthy bone marrow. Hematopoietic stem cells are blood-forming cells found both in the bloodstream and in the bone marrow. This procedure is also called a stem cell transplant or hematopoietic stem cell transplant.
There are 2 types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). ALLO uses donated stem cells from a healthy donor, while AUTO uses the patient’s own stem cells. In both types, the goal is to destroy all of the unhealthy cells in the marrow, blood, and other parts of the body using high doses of chemotherapy and/or radiation therapy and then allow replacement blood stem cells to create healthy bone marrow. An ALLO transplant is usually the type recommended for people with MDS.
Side effects depend on the type of transplant, your general health, and other factors. Learn more about the basics of stem cell and bone marrow transplantation.
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Physical, emotional, social, and financial effects of MDS
MDS and its treatment often cause side effects, as well as emotional, social, and financial effects. Managing all of these effects is called palliative and supportive care. It is an important part of your care that is included along with treatments intended to slow, stop, or eliminate MDS.
Palliative and supportive care focuses on improving how you feel during treatment by managing symptoms and supporting patients and their families with other, non-medical needs. Any person, regardless of age or subtype and classification of MDS, may receive this type of care. And it often works best when it is started right after a diagnosis. People who receive palliative and supportive care along with treatment for MDS often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.
Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional support, and other therapies. You may also receive palliative treatments such as chemotherapy. Talk with your doctor about the goals of each treatment in the treatment plan.
Some people with MDS who do not have any symptoms may only need close monitoring, including measuring blood counts, to watch for any symptoms. People with low-risk MDS, meaning those with RA and RARS subtypes, usually receive supportive care to control symptoms and improve quality of life, including growth factors and blood transfusions (see above). Antibiotics may also be given to fight infection, a common side effect of low white blood cell counts.
Before treatment begins, talk with your health care team about the goals of each treatment in the recommended treatment plan. You should also talk about the possible side effects of the specific treatment plan and palliative and supportive care options. Many patients also benefit from talking with a social worker and participating in support groups. Ask your doctor about these resources, too.
Care for MDS is often expensive, and navigating health insurance can be difficult. Ask your doctor or another member of your health care team about talking with a financial navigator or counselor who may be able to help with your financial concerns.
During treatment, your health care team may ask you to answer questions about your symptoms and side effects and to describe each problem. Be sure to tell the health care team if you are experiencing a problem. This helps the health care team treat any symptoms and side effects as quickly as possible. It can also help prevent more serious problems in the future.
Learn more about the importance of tracking side effects in another part of this guide. Learn more about palliative and supportive care in a separate section of this website.
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If MDS worsens
If MDS continues to worsen despite treatment, it is a good idea to talk with doctors who have experience in treating the disease. Doctors can have different opinions about the best standard treatment plan. Clinical trials are also a treatment option. Learn more about getting a second opinion before starting treatment, so you are comfortable with your chosen treatment plan.
Your treatment plan may include a combination of the treatments discussed above. Palliative and supportive care will also be important to help relieve symptoms and side effects.
For most people, worsening MDS is very stressful and difficult. You and your family are encouraged to talk about how you feel with doctors, nurses, social workers, or other members of the health care team. It may also be helpful to talk with other patients, such as through a support group or other peer support program.
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Remission and the chance of recurrence
A remission is when MDS cannot be detected in the body and there are no symptoms. This may also be called having “no evidence of disease” or NED.
A remission may be temporary or permanent. This uncertainty causes many people to worry that the disease will come back. While many remissions are long-lasting, it is important to talk with your doctor about the possibility of the disease returning. Understanding your risk of recurrence and the treatment options may help you feel more prepared if the disease does return. Learn more about coping with the fear of recurrence.
If MDS does return after the original treatment, it is called recurrent MDS. If this happens, a new cycle of testing will begin to learn as much as possible about the recurrence. After this testing is done, you and your doctor will talk about the treatment options. Sometimes, new genetic mutations have appeared that can be targeted by new medications. Often the treatment plan will include the treatments described above, such as chemotherapy and bone marrow transplantation, but they may be used in a different combination or given at a different pace. Your doctor may suggest clinical trials that are studying new ways to treat this type of recurrent disease. Whichever treatment plan you choose, palliative and supportive care will be important for relieving symptoms and side effects.
People with recurrent MDS often experience emotions such as disbelief or fear. You are encouraged to talk with the health care team about these feelings and ask about support services to help you cope. Learn more about dealing with a recurrence.
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If treatment does not work
Recovery from MDS is not always possible. If MDS cannot be cured or controlled, the disease may be called advanced or terminal.
This diagnosis is stressful, and for some people, advanced MDS is difficult to discuss. However, it is important to have open and honest conversations with your health care team to express your feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families. Making sure a person is physically comfortable and free from pain is extremely important.
Planning for your future care and putting your wishes in writing is important, especially at this stage of disease. Then, your health care team and loved ones will know what you want, even if you are unable to make these decisions. Learn more about putting your health care wishes in writing.
People who have advanced disease and who are expected to live less than 6 months may want to consider hospice care. Hospice care is designed to provide the best possible quality of life for people who are near the end of life. You and your family are encouraged to talk with your doctor or a member of your palliative care team about hospice care options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Learn more about advanced care planning.
After the death of a loved one, many people need support to help them cope with the loss. Learn more about grief and loss.
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The next section in this guide is About Clinical Trials. It offers more information about research studies that are focused on finding better ways to care for people with MDS. Use the menu to choose a different section to read in this guide.