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This section outlines treatments that are the standard of care (the best proven treatments available) for this specific disease. When making treatment plan decisions, patients are also encouraged to consider clinical trials as an option. A clinical trial is a research study to test a new treatment to evaluate whether it is safe, effective, and possibly better than standard treatment. Your doctor can help you review all treatment options. For more information, see the Clinical Trials and Current Research sections.
For MDS, different types of doctors often work together to create a patient’s overall treatment plan that combines different types of treatments. This is called a multidisciplinary team.
Descriptions of the most common treatment options for MDS are listed below. The goal of treatment is to bring about a remission and to bring blood counts back to normal. Treatment options and recommendations depend on several factors, including the subtype and IPSS score of MDS, the risk of developing AML, possible side effects, and the patient’s preferences, age, and overall health. As explained in the Subtypes section, a patient’s IPSS score and MDS subtype help doctors determine the most effective treatment, but it is important to note that the treatments may not be equally effective for every patient.
Learn more about making treatment decisions.
MDS and its treatment often cause side effects. In addition to treatment for MDS, an important part of care is relieving a person’s symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.
Palliative care can help a person at any stage of illness. People often receive treatment for MDS and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.
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 (RA and RARS subtypes) usually receive supportive care to control symptoms and improve quality of life. Supportive care may include:
- Use of growth factors that help cells mature, such as epoetin (Epogen, Eprex, Procrit), a red blood cell growth factor and filgrastim (Neupogen), a white blood cell growth factor
- Antibiotics to fight infection, a common side effect of low white blood cell counts
Before treatment begins, talk with your health care team about the possible side effects of your specific treatment plan and supportive care options. And during and after treatment, be sure to tell your doctor or another health care team member if you are experiencing a problem, so it is addressed as quickly as possible. Learn more about palliative care.
Chemotherapy is the use of drugs to kill cancer cells, usually by stopping the cancer cells’ ability to grow and divide. Systemic chemotherapy is delivered through the bloodstream to reach cancer cells throughout the body. Chemotherapy is given by a medical oncologist, a doctor who specializes in treating cancer with medication, or by a hematologist, a doctor who specializes in treating blood disorders. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time. A patient may receive one drug at a time or combinations of different drugs at the same time.
Chemotherapy used for MDS includes the following drugs:
- Lenalidomide (Revlimid)
- Decitabine (Dacogen)
- Azacitidine (Vidaza)
- Cytarabine (Cytosar-U)
- Idarubicin (Idamycin)
- Daunorubicin (Cerubidine)
People with high-risk subtypes of MDS who have an increased risk of developing AML may benefit from chemotherapy. Medications that are used to treat AML, such as cytarabine, idarubicin or daunorubicin, are also sometimes considered, particularly if the MDS has turned into AML. Overall, 30 to 40% of patients may benefit from chemotherapy for MDS. Factors such as the patient’s age and medical condition are evaluated before starting chemotherapy. Supportive care (see above) is an alternative treatment option for patients when the side effects of chemotherapy are too severe.
Lenalidomide is an oral (taken by mouth) medication that is very effective when given to patients with low-risk MDS and 5q chromosomal abnormalities (See Subtypes). Therefore, it is very important for doctors to find out whether a patient has this chromosomal change.
Both decitabine and azacitidine are approved by the U.S. Food and Drug Administration (FDA) to treat all types of MDS, although they are used most often for patients with higher IPSS scores. Both can be given in the doctor’s office or clinic, and patients often need more than one round of treatment given monthly before it starts improving a patient’s condition.
The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, loss of appetite, and diarrhea. These side effects usually go away once treatment is finished.
Learn more about chemotherapy and preparing for treatment. The medications used to treat cancer 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. Learn more about your prescriptions by using searchable drug databases.
Stem cell transplantation/bone marrow transplantation
High-dose chemotherapy with stem cell/bone marrow 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 or have other medical problems. Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the subtype of MDS, results of any previous treatment, and patient’s age and general health.
A stem cell transplant is a medical procedure in which diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because it is the blood stem cells that are typically being transplanted, not the actual bone marrow tissue.
There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). AUTO transplants are not often used for patients with MDS.
In both types, the goal of transplantation is to destroy disease cells in the marrow, blood, and other parts of the body and allow replacement blood stem cells to create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible. Learn more about bone marrow and stem cell transplantation.
A remission is when MDS cannot be detected in the body and there are no symptoms. This may also be called “no evidence of disease” or NED.
A remission can be temporary or permanent. This uncertainty leads to many survivors feeling worried or anxious that the disease will come back. While many remissions are permanent, it is important to talk with your doctor about the possibility of the disease returning. Understanding the 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. When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, you and your doctor will talk about your treatment options. Often the treatment plan will include the therapies described above (chemotherapy and stem cell transplantation) but may be used in a different combination or given at a different pace. Your doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent disease.
People with recurrent MDS often experience emotions such as disbelief or fear. Patients are encouraged to talk with their health care team about these feelings and ask about support services to help them cope. Learn more about dealing with a recurrence.
If MDS worsens
If MDS continues to worsen despite treatment, patients are encouraged to talk with doctors who are experienced in treating this disease because there can be different opinions about the best treatment plan. Learn more about seeking a second opinion before starting treatment, so you are comfortable with the treatment plan chosen. This discussion may include clinical trials studying new treatments.
Your health care team may recommend a treatment plan that includes a combination of the treatments discussed above. Supportive care will also be important to help relieve symptoms and side effects.
For many patients, worsening MDS can be very stressful and, at times, difficult to bear. Patients and their families are encouraged to talk about the way they are feeling with doctors, nurses, social workers, or other members of the health care team. It may also be helpful to talk with other patients, including through a support group.
If treatment fails
Recovery from MDS is not always possible. If treatment is not successful, the disease may be called advanced or terminal disease.
This diagnosis is stressful, and this is difficult to discuss for many people. However, it is important to have open and honest conversations with your doctor and 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.
Palliative care given toward the end of a person’s life is called hospice care. You and your family are encouraged to think about where you would be most comfortable: at home, in the hospital, or in a hospice environment. Nursing care and special equipment can make staying at home a workable alternative for many families. Learn more about advanced care planning.
After the death of a loved one, many people need support to help cope with the loss. Learn more about grief and bereavement.
Find out more about common terms used during treatment.