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The treatment approach for myelodysplastic syndromes (MDS) depends on a number of factors, such as:

• The type of MDS
• The prognostic score (risk group) of the MDS
• A person’s age, overall health, and preferences

Some people with MDS who don’t have very low blood cell counts or bothersome symptoms might not need to be treated right away. 

If treatment is needed, a stem cell transplant (SCT) is usually considered the only way to potentially cure MDS, so it may be the treatment of choice for younger, relatively healthy patients if a matched donor is available. Unfortunately, many people with MDS are older or in poor health and might not be good candidates for a SCT.

When SCT is not an option, MDS is very unlikely to be cured, but it can often still be treated. The main goal of treatment is to relieve symptoms and avoid complications and side effects.

If low blood counts are causing problems, supportive care treatments such as transfusions or blood cell growth factors may be helpful. Careful general medical care and measures to prevent and treat infections are also very important. Supportive care is important regardless of whether a person is getting other treatments for MDS.

If other treatment is needed, a chemotherapy drug such as azacitidine (Vidaza) or decitabine (Dacogen) is often the first choice, especially for patients with lower-risk forms of MDS. These drugs can often improve blood counts, and many patients need fewer transfusions and have a better quality of life, with less fatigue. These drugs can also help some people live longer. Another option for some people might be medicines to suppress the immune system, such as ATG and cyclosporine.

If a person has the del(5q) type of MDS (where the cells are missing part of chromosome 5), lenalidomide (Revlimid) is often used as the first treatment. If this drug doesn’t help, treatment with azacitidine or decitabine is often the next option.

For some patients with more advanced MDS, such as those whose prognostic scores are high risk or higher, or those whose MDS looks like it is becoming acute myeloid leukemia (AML), standard chemotherapy drugs might be an option. Unfortunately, this treatment can often be too toxic for patients who are elderly or who have many other medical problems. For young and healthy patients, though, the treatment is similar to treatment for AML.

If one type of treatment doesn’t work (or if it stops working), another one might be tried. Many new medicines to treat MDS are also being studied in clinical trials. Because the best options to treat MDS aren’t clear, and because MDS often becomes hard 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.