Myelodysplastic Syndromes

Treatment Options

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.

Treatment overview

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.

Palliative/supportive care

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:

Transfusions to treat anemia and thrombocytopenia (low numbers of platelets)

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. 

Chemotherapy

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. 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.

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.  

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.

Recurrent MDS

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. 

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.

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. 

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.

Clinical Trials

Doctors and scientists are always looking for better ways to treat patients with MDS. To make scientific advances, doctors create research studies involving people, called clinical trials.

Many clinical trials are focused on new treatments, evaluating whether a new treatment is safe, effective, and possibly better than the current (standard) treatment. These types of studies evaluate new drugs, different combinations of existing treatments, new approaches to radiation therapy or surgery, and new methods of treatment. Patients who participate in clinical trials are often among the first to receive new treatments before they are widely available. However, there is no guarantee that the new treatment will be safe, effective, or better than a standard treatment.

There are also clinical trials that study new ways to ease symptoms and side effects during treatment and manage the late effects that may occur after treatment. Talk with your doctor about clinical trials regarding side effects. In addition, there are ongoing studies about ways to prevent the disease.

Patients decide to participate in clinical trials for many reasons. For some patients, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Other patients volunteer for clinical trials because they know that these studies are the only way to make progress in treating MDS. Even if they do not benefit directly from the clinical trial, their participation may benefit future patients with MDS.

Sometimes people have concerns that, by participating in a clinical trial, they may receive no treatment by being given a placebo or a “sugar pill.” The use of placebos in cancer clinical trials is rare. When a placebo is used in a study, it is done with the full knowledge of the participants. 

To join a clinical trial, patients must participate in a process known as informed consent. During informed consent, the doctor should list all of the patient’s options, so that the person understands how the new treatment differs from the standard treatment. The doctor must also list all of the risks of the new treatment, which may or may not be different from the risks of standard treatment. Finally, the doctor must explain what will be required of each patient in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.

Patients who participate in a clinical trial may stop participating at any time for any personal or medical reason. This may include that the new treatment is not working or there are serious side effects. Clinical trials are also closely monitored by experts who watch for any problems with each study. It is important that patients participating in a clinical trial talk with their doctor and researchers about who will be providing their treatment and care during the clinical trial, after the clinical trial ends, and/or if the patient chooses to leave the clinical trials before it ends.

Coping with Side Effects

Fear of treatment side effects is common after a diagnosis of MDS, but it may help to know that preventing and controlling side effects is a major focus of your health care team. This is called palliative or supportive care, and it is an important part of the overall treatment plan, regardless of the stage of disease.

Common side effects from each treatment option for MDS are described in detail within the Treatment section.  Side effects depend on a variety of factors, including the subtype of MDS, the length and dosage of treatment(s), and your overall health.

Before treatment begins, talk with your doctor about possible side effects of each type of treatment you will be receiving. Ask which side effects are most likely to happen, when they are likely to occur, and what can be done to prevent or relieve them. And, ask about the level of caregiving you may need during treatment and recovery, as family members and friends often play an important role in the care of a person with MDS. 

In addition to physical side effects, there may be psychosocial (emotional and social) effects as well. Patients and their families are encouraged to share their feelings with a member of their health care team, who can help with coping strategies.

During and after treatment, be sure to tell the health care team about the side effects you experience, even if you feel they are not serious. Sometimes, side effects can last beyond the treatment period, called a long-term side effect. A side effect that occurs months or years after treatment is called a late effect. Treatment of both types of effects is an important part of survivorship care.

After Treatment

After treatment for MDS, talk with your doctor about developing a follow-up care plan. This plan may include regular physical examinations and/or medical tests for the coming months and years. People treated for MDS need ongoing tests for blood counts and bone marrow function to check if or how the disease changes, how well current treatment is working, and whether additional treatment is needed. People who received a stem cell/bone marrow transplant need follow-up care more often; this includes watching for symptoms and signs of graft-versus-host disease, a possible side effect of an ALLO transplantation .

People treated for MDS are encouraged to follow established guidelines for good health, such as maintaining a healthy weight, not smoking, eating a balanced diet, and receiving appropriate screening for other types of cancer beyond AML. Talk with your doctor to develop a plan that is best for your needs. Moderate physical activity can help rebuild your strength and energy level after receiving more intensive treatments. Your doctor can help you create an appropriate exercise plan based upon your needs, physical abilities, and fitness level.  

Latest Research

Doctors are working to learn more about MDS, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your doctor about the diagnostic and treatment options best for you.

New drugs and drug combinations. Researchers are looking at treatments with the following:

Arsenic trioxide (Trisenox)

ATG (anti-thymocyte globulin)

Azacitidine in combination with other drugs

Decitabine in combination with other drugs

Lenalidomide in patients who do not have abnormalities of chromosome 5

Histone deacetylase inhibitors

Different approaches to stem cell transplantation

Some of these medications, such as azacitidine, decitabine, and lenalidomide, have been approved by the FDA for the treatment of MDS and are now being evaluated in different doses and schedules and in combination with other drugs.

Supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current MDS treatments in order to improve patients’ comfort and quality of life.