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Drug therapy is one of many methods doctors use to treat MDS. Three single-drug approaches have been approved by the US Food and Drug Administration (FDA) for treatment of MDS.

  • azacitidine
  • decitabine
  • lenalidomide


Azacitidine (Vidaza®) is a member of a class of drugs called hypomethylating or demethylating agents. It's used to treat both low- and high-risk patients. It kills abnormal cells and appears to help bone marrow function more normally.

Your doctor injects azacitidine under your skin (subcutaneous injection). (Another form of azacitidine that can be swallowed is being studied in clinical trials.)

About 40 percent of MDS patients respond to azacitidine, improving quality of life and reducing the need for blood transfusions. However, it can cause such side effects as nausea, vomiting and diarrhea. It also temporarily reduces blood counts, but they return to their previous levels before each series of injections.


Decitabine (Dacogen®) is also a hypomethylating (demethylating) agent. It's used to treat both low- and high-risk patients. Like azacitidine, it kills abnormal cells and appears to help bone marrow function more normally.

Decitabine is injected into a vein (intravenous injection). It reduces the need for blood transfusions and has been shown to improve blood counts in 30 percent to 40 percent of patients. Researchers are continuing to study the most effective doses of decitabine.


Lenalidomide (Revlimid®) is an immunomodulatory drug. It's the preferred treatment for patients who (1) fall into the low or intermediate-1 risk category of the International Prognostic Scoring System (IPSS), (2) have transfusion-dependent anemia and (3) have a deletion of chromosome 5q (5q-) with or without other chromosomal abnormalities. It's estimated that 20 percent to 30 percent of MDS patients have a 5q-.

Lenalidomide can reduce the need for red cell blood transfusions in most low-risk patients with 5q- for about two years. It also reduces the need for red cell transfusions in low-risk patients without 5q- but not as effectively as in patients with 5q-. More studies are needed to understand lenalidomide's effect on life expectancy and its benefits for patients in the intermediate-2 or high-risk categories of the IPSS, with or without 5q-.

If you would like to read about these drugs individually, including information about side effects, click here.

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last updated on Wednesday, April 24, 2013

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