Medicines Approved by the U.S. Food and Drug Administration (FDA) to Treat MDS

The FDA has approved three medicines to treat MDS:

  • Azacitidine (Vidaza®) for both low- and high-risk patients with all sub-types of MDS
  • Decitabine (Dacogen®) for both low- and high-risk patients with all sub-types of MDS
  • Lenalidomide (Revlimid®) for transfusion-dependent MDS patients with isolated del(5q) and with a low or intermediate-1 risk IPSS score

For those living in Europe, these medicines are now under review by the European Agency for the Evaluation of Medicinal Products (EMEA).

Azacitidine (Vidaza®)

Azacitidine was the first medicine approved specifically to treat MDS. It can be used by both low- and high-risk patients with all subtypes of MDS. This medicine is in a new class of medicines called demethylating agents. Drugs in this class help prevent the growth of abnormal bone marrow stem cells.

Scientists did a study that compared patients taking azacitidine with patients receiving standard care, including supportive care and chemotherapy. They found that patients who took azacytidine:

  • Generally had a better quality of life
  • Lived longer
  • Needed fewer transfusions
  • Were more likely to have improved red blood cell counts
  • Were much less likely to develop acute leukemia or die

Decitabine (Dacogen®)

Like azacytidine, decitabine is a demethylating agent that can be used by low- and high-risk patients with all subtypes of MDS.

Lenalidomide (Revlimid®)

Lenalidomide is approved for treating transfusion-dependent MDS patients with isolated del(5q) MDS with a low or intermediate-1 risk IPSS (International Prognostic Scoring System) score.  Lenalidomide is considered an immunomodulatory agent, as it stops the effects of "bad" chemicals produced in the bone marrow. It also slows the growth of new blood vessels that feed the MDS cells, which makes it an anti-angiogenic agent. Lenalidomide also kills abnormal cells in the bone marrow that are then replaced by normal looking cells. For some MDS patients, this drug can improve anemia and either reduce or eliminate the need for blood transfusions.

Lenalidomide may also lower your white blood cell and platelet counts before raising them. So you should have your blood counts checked once a week for the first eight weeks of treatment, and at least once a month after that.

Lenalidomide may cause birth defects. This drug is similar to the drug thalidomide, which is known to cause life-threatening birth defects in babies. For this reason, it is only available through a special program called RevAssist. Doctors, pharmacists, and patients are all required by the FDA to participate in RevAssist.

Women who are pregnant or plan to become pregnant during treatment, should not take lenalidomide. The RevAssist program teaches you about the best methods of birth control to use in order to avoid becoming pregnant at this time.

Your doctor will give you information on how to enroll in RevAssist if the doctor is going to prescribe this drug. You may also want to call the manufacturer, toll free, at (888) 423-5436.

Men who are taking lenalidomide must take special care when having sex with women who are of childbearing age. This includes using a condom (even if you have had a vasectomy) each and every time you have intercourse.