MDS Drug Therapy

There are three medicines approved in the U.S. to treat MDS. Azacitidine (Vidaza®) and decitabine (Dacogen®) are approved to treat both low- and high-risk patients with all sub-types of MDS. Lenalidomide (Revlimid®) is approved for transfusion-dependent MDS patients with isolated del(5q) and with a low or intermediate-1 risk IPSS score.

The U.S. Food and Drug Administration (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

Azacitidine (Vidaza®)

Azacitidine is in a class of medicines called hypomethylating agents. Drugs in this class help prevent the growth of abnormal bone marrow stem cells.

Azacitidine was the first medicine approved by the U.S. Food and Drug Administration (FDA) specifically to treat MDS. It is approved to treat both low- and high-risk patients with all subtypes of MDS.

Azacitidine is given by injection under the skin or as an intravenous infusion (IV) where it is injected into your vein. It is given in a clinic or hospital setting.

Azacitidine is usually given once a day for five to seven days, followed by 21 days without treatment. This 28-day cycle is then repeated. Treatment regimens vary with different treatment centers and physicians. Some patients respond in as little as two cycles. Others need more than six. Experts recommend having at least six treatment cycles to see whether the drug is working for you. The treatment may be continued for as long as the patient continues to benefit.

How well does it work?

Scientists did a study in higher-risk MDS patients comparing those taking azacitidine to those getting conventional care, including supportive care and chemotherapy. They found that about 5 in 10 patients had some positive response to treatment, meaning that blood counts went up. The percentage of patients still surviving after two years doubled in those taking azacitidine compared to those getting conventional care.

The researchers found that patients who took azacitidine:

  • Generally had a better quality of life

Lived longer

  • Needed fewer transfusions
  • Were more likely to have improved red blood cell counts
  • Experienced a delay in MDS progressing to acute leukemia

What are common side effects?

Some common side effects of azacitidine include:

  • Nausea
  • Vomiting
  • Diarrhea or constipation
  • Tiredness and weakness
  • Low blood counts (most often the white blood cells or platelets)

It is important for you to know that azacitidine may lower your blood counts before raising them. Your counts may be back to normal or better than normal before the next treatment cycle begins. This can be difficult for some patients and can sometimes reduce the willingness to continue treatment.

Decitabine (Dacogen®)

Decitabine is in a class of medicines called hypomethylating agents. Drugs in this class help prevent the growth of abnormal bone marrow stem cells.

Decitabine, like azacitidine, is approved by the U.S. Food and Drug Administration (FDA) specifically to treat MDS. It is approved to treat both low- and high-risk patients with all subtypes of MDS.

Decitabine is given as an intravenous infusion (IV) in a clinic or hospital.

There are two main regimens.

  1. The infusion is given every eight hours for three days. This three-day treatment is usually repeated every six weeks.
  2. The infusion is given one time per day for five days in a row. This five-day treatment is repeated every four weeks (28 days).

Experts recommend having at least six treatment cycles to see whether the drug is working for you. The treatment may be continued for as long as the patient continues to benefit.

How well does it work?

Researchers did a large study comparing patients taking decitabine or conventional care regimens, including best supportive care and chemotherapy. In this study about 3 in 10 patients had a positive response, with a complete and partial remission rate of 17 in 100 patients. However, no overall survival advantage was shown with decitabine compared to best supportive care.

What are common side effects?

Some common side effects of decitabine include:

  • Nausea
  • Vomiting
  • Diarrhea or constipation
  • Tiredness and weakness
  • Low blood counts (most often the white blood cells or platelets)

It is important for you to know that decitabine may lower your blood counts before raising them. Your counts may be back to normal or better than normal before the next treatment cycle begins. This can be difficult, and can sometimes reduce the willingness to continue treatment.

Lenalidomide (Revlimid®)

Lenalidomide is considered an immunomodulatory agent. This means it is able to enhance the activity of immune cells and reduce inflammation. This slows the growth of abnormal bone marrow cells. In addition, it improves the way certain white blood cells work and is thought to slow the growth of new blood vessels that feed the MDS cells.

Lenalidomide (Revlimid®) is approved for treating MDS patients with isolated del(5q) MDS subtyped who are transfusion dependent (still need blood transfusions) and have a low or intermediate-1 risk IPSS (International Prognostic Scoring System) score. Research studies are currently looking at how well lenalidomide works for patients with other MDS subtypes.

Lenalidomide is a pill taken by mouth. It is typically taken once per day for 3 weeks with a 1 week rest period. This period of 4 weeks is known as a treatment cycle. It can take 4 or more cycles to know if this drug is working for you.

Most MDS patients who respond to lenalidomide do so for 1 or 2 cycles (1 to 2 months). Response means that you have become transfusion independent (i.e., no longer need blood transfusions).

Experts recommend staying on this drug for at least 4 cycles (4 months) to see if it is working for you. It can take up to 12 cycles for some people to respond.

How well does it work?

In one large clinical study more than 6 in 10 patients with isolated del(5q) MDS responded. The length of response varied, but on average participants were transfusion independent for more than 40 weeks.

What are common side effects?

Some common side effects include:

  • Decreased blood counts (most often the white cell count and platelet count)
  • Diarrhea or constipation
  • Tiredness and weakness
  • Dry or itchy skin
  • Muscle or joint pain
  • Headache