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The treatment options for people with myelodysplastic syndromes (MDS) depend on a number of factors, such as:

• The type of MDS a person has
• The prognostic score (risk group) of the MDS
• What symptoms a person is having (if any)
• A person’s age, overall health, and preferences

In general, doctors divide MDS into 2 main groups when thinking about treatment options:

• Lower-risk MDS
• Higher-risk MDS

For more on these groupings, see Myelodysplastic Syndrome Prognostic Scores (Risk Groups).

Treating lower-risk MDS

Lower-risk MDS is less likely to progress to acute myeloid leukemia (AML) or to cause other serious health problems. But it’s important to know that each person’s outlook is unique.

In people without symptoms

Some people with lower-risk MDS don’t have very low blood cell counts or bothersome symptoms when the MDS is first found.

These people might not need to be treated right away. Instead, they can often be watched closely, with regular doctor visits and blood tests to monitor blood cell counts. How often this needs to be done can differ from person to person. This can be continued for as long as the MDS isn’t causing problems, which might be many years for some people.

Most doctors favor this approach, because it’s not clear that treating lower-risk MDS in people without symptoms right away helps them live any longer, but it can still cause side effects.

If the MDS starts to cause symptoms or leads to very low blood cell counts at some point, treatment can then be started (see below).

Treating people with symptoms or with very low blood cell counts

There is no single best treatment for all people with lower-risk MDS who need to be treated. Treatment is tailored to each person’s situation, based on their blood counts and symptoms, as well as other factors.

Supportive therapies, medicines to treat MDS directly, or both might be used. Examples of treatment options might include:

• Blood transfusions, growth factors, and/or other medicines to raise red blood cell counts
• Platelet transfusions or medicines to raise platelet counts
• Antibiotics to treat infections and/or growth factors to raise white blood cell counts
• Lower-intensity chemo with a hypomethylating agent (azacitidine or decitabine)
• Medicines that affect the immune system, such as lenalidomide or anti-thymocyte globulin (ATG)
• Other targeted medicines, if the MDS cells have certain gene or chromosome changes

More intense treatments, like those used to treat acute myeloid leukemia (AML) (such as high-dose chemo, possibly with a stem cell transplant), are usually reserved for people with higher-risk MDS. However, they might be an option for some people with lower-risk MDS, especially those who are younger, in good health, and who want to try to put the MDS into long-term remission. Even then, many doctors would advise that treatment be done as part of a clinical trial.

Treating higher-risk MDS

People with higher-risk MDS generally need treatment right away, as their MDS is more likely to progress and cause serious problems. Still, it’s important to keep in mind that the MDS risk groups can’t be used to predict any person’s outlook for sure.

Often the first step before deciding on treatment is determining if the person is a good candidate for (and is willing to get) a stem cell transplant (SCT), also known as a bone marrow transplant (BMT). This treatment offers the best chance to put the MDS in long-term remission, and possibly even cure it. But it’s a very intense treatment, so it might not be a good option for many people with higher-risk MDS, especially if they are older or have other major health problems.

For people who can have a stem cell transplant

There’s no set age cutoff for a stem cell transplant, but it’s important that people who are getting one can tolerate (are strong enough for) the treatment. For some people who are slightly older or who have less serious health conditions, a reduced intensity transplant (also known as a non-myeloablative transplant or a mini-transplant) might be an option.

Some people might be able to get a SCT as their first treatment, especially if they have a matched stem cell donor available. For other people, including those who don’t have an available matched donor, other treatments might be used first. Examples include:

• Lower-intensity chemo with a hypomethylating agent (azacitidine or decitabine)
• Higher-intensity chemo, like that used to treat AML 
• Targeted therapy or other drugs, if the MDS cells have certain gene or chromosome changes

While these treatments are less likely to result in long-term remissions on their own than a SCT, they might help keep the MDS in check until a SCT can be done.

Regardless of which approach is used, people can also get supportive therapies to help prevent or relieve symptoms from the MDS. Supportive care is important no matter what other treatments a person is getting.

Careful general medical care and measures to prevent and treat infections are also very important.

For people who can’t have (or don’t want) a stem cell transplant

When a stem cell transplant isn’t a good option, other treatments can still be helpful in people with higher-risk MDS. Treatment can still help prevent or relieve symptoms, improve a person’s quality of life, and possibly help them live longer as well.

Most people who aren’t candidates for a SCT most likely wouldn’t be able to tolerate high-dose chemotherapy either, so this isn’t used often.

Examples of treatments that might be used include:

• Lower-intensity chemo with a hypomethylating agent (azacitidine or decitabine)
• Targeted therapy or other drugs, if the MDS cells have certain gene or chromosome changes
• Supportive care as needed, with treatments such as blood transfusions, growth factors, or other medicines

Careful general medical care and measures to prevent and treat infections are also very important.

Treating MDS that progresses or doesn’t respond to treatment

If one type of treatment for MDS doesn’t work (or if it stops working), another one might still be helpful. A person’s options will depend on the type and risk group of the MDS, the person’s health and preferences, and which treatment(s) they’ve already had.

At some point if the MDS progresses or returns after treatment, the doctor might want to get new blood and/or bone marrow tests to see if the MDS cells have changed. This might affect treatment options. For example, new gene changes might appear in the MDS cells, which might mean that the MDS can be treated with newer medicines.

Whichever treatment plan you choose, supportive care is still important to help with symptoms.

Many new medicines to treat MDS are also being studied in clinical trials. Because the best options to treat MDS aren’t always clear, and because MDS often becomes harder to treat over time, taking part in a clinical trial might be a good option at some point. Talk to your health care team to learn more about clinical trials that might be right for you.