Chemotherapy for Myelodysplastic Syndromes

Chemotherapy (chemo) is the use of drugs for treating a disease such as cancer. Some chemo drugs can be swallowed as pills, while others are injected by needle into a vein or muscle. These drugs are considered systemic treatment because they enter the bloodstream and reach most areas of the body. This type of treatment is useful for diseases such as myelodysplastic syndrome (MDS) that are not only in one part of the body. The purpose of the chemo is to kill the abnormal stem cells and allow normal ones to grow back.

Hypomethylating agents

These types of chemo drugs affect the way certain genes inside a cell are controlled. These drugs activate some genes that help cell mature. They also kill cells that are dividing rapidly. Examples of this type of drug include:

  • Azacitidine (Vidaza)
  • Decitabine (Dacogen)

In some MDS patients, using one of these drugs can often improve blood counts (sometimes enough so that blood transfusions aren’t needed), improve quality of life, lower the chance of getting leukemia, and even help a person live longer.

Azacitidine can be injected under the skin or into a vein (IV), often for 7 days in a row each month. Decitabine is often injected into a vein (IV) over 3 hours every 8 hours for 3 days. This is repeated every 6 weeks. Decitabine can also be given by IV over an hour, each day for 5 days in a row, and repeated every 4 weeks.

These drugs can have some of the same side effects as standard chemo drugs (see below), but these side effects are usually milder. A major side effect of these drugs is usually an early drop in blood cell counts, which tends to get better as the drug begins to work. Other side ffects can include:

  • Fever
  • Nausea/vomiting
  • Diarrhea or constipation
  • Fatigue and weakness

Standard chemotherapy drugs

Standard chemo drugs are less useful for MDS than the hypomethylating agents, so they are not used often. But higher-risk MDS is more likely to progress to acute myeloid leukemia (AML), so some patients with these types of MDS may receive the same chemotherapy treatment as AML patients.

The chemo drug most often used for MDS is called cytarabine (ara-C). It can be given by itself at a low-dose, which can often help control the disease, but doesn’t often put it into remission.

Another option is to give the same, intense type of chemo that is used for younger patients with AML. This means giving cytarabine at a higher dose along with other chemo drugs. This is more often used in younger, healthier patients with higher-risk forms of MDS (like MDS with excess blasts). Some of the chemo drugs that can be combined with cytarabine are:

  • Idarubicin
  • Daunorubicin

Other chemo drugs might be used as well.

Patients who get the higher dose treatment are more likely to go into remission, but they can also have more severe, even life-threatening side effects, so this treatment is typically given in the hostpital. Still, this treatment may be an option for some patients with advanced MDS.

Chemo drugs can cause many side effects. These depend on the type and dose of the drugs that are given and how long they are taken. Common side effects include:

  • Hair loss
  • Mouth sores
  • Loss of appetite
  • Nausea and vomiting
  • Low blood counts

MDS patients already have low blood counts, which often become even worse for a time before they get better.

  • Low white blood cell counts lead to an increased risk of serious infections.
  • Low platelet counts can lead to problems with easy bruising and can have serious bleeding, including bleeding into the brain or the intestine.
  • Low red blood cell counts (or anemia) can lead to fatigue and shortness of breath. In people with heart problems, severe anemia can lead to a heart attack.

If a patient's blood cell counts become too low, they may need supportive therapy (including transfusions) or growth factors to help prevent or treat serious side effects. 

Most side effects from chemo will go away after treatment is finished. Your health care team can often suggest ways to lessen side effects. For example, drugs can be given to help prevent or reduce nausea and vomiting.

Chemo drugs can also affect other organs. For example:

  • Idarubicin and daunorubicin can damage the heart, so they are often not given to patients who already have heart problems.
  • Cytarabine can affect the brain and cause balance problems, sleepiness, and confusion. This is more common with higher doses.

If serious side effects occur, the chemo treatments may have to be reduced or stopped, at least temporarily. It's important to carefully monitor and adjust drug doses, because some of these side effects can be permanent.

Immune treatments

Immunomodulating drugs (IMiDs)

Lenalidomide (Revlimid) belongs to a class of drugs known as immunomodulating drugs (IMiDs). It seems to work well in low-grade MDS, often eliminating the need for blood transfusions, at least for a time. The drug seems to work best in people whose MDS cells are missing a part of chromosome 5 (MDS-del(5q)). But it can also help some MDS patients that do not have this abnormal chromosome.

Side effects can include:

  • Lowered blood counts (most often the white cell count and platelet count)
  • Diarrhea or constipation
  • Fatigue and weakness

This drug can also increase the risk of serious blood clots that start in the veins in the legs (called a deep vein thrombosis, or DVT). Sometimes, part of a DVT can break off and travel to the lungs (called a pulmonary embolus or PE), where it can cause breathing problems or even death.

This drug might also cause serious birth defects if given to pregnant women. Because of this, it's only available through a special program by the drug company.

Immune system suppression

Drugs that suppress the immune system can help some patients with lower-risk MDS. These drugs are most helpful for people with low numbers of cells in the bone marrow (called hypocellular bone marrow).

Anti-thymocyte globulin (ATG) is an antibody against a type of white blood cell called the T-lymphocyte, which helps control immune reactions. For some patients with MDS, T-lymphocytes interfere with normal blood cell production, so ATG can be helpful. ATG is given as an infusion through a vein. It must be given in the hospital because it can sometimes cause severe allergic reactions leading to low blood pressure and problems breathing.

Cyclosporine is another drug that can suppress the immune system. It can be used along with ATG to help some patients with MDS. Side effects of cyclosporine can include loss of appetite and kidney damage.

The American Cancer Society medical and editorial content team
Our team is made up of doctors and master's-prepared nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Estey EH, Schrier SL. Management of complications of the myelodysplastic syndromes. UpToDate. 2017. Accessed at management-of-the-complications-of-the-myelodysplastic-syndromes on October 12, 2017.

Estey EH, Schrier SL. Treatment of high or very high risk myelodysplastic syndromes. UpToDate. 2017. on October 12, 2017.

National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Myelodysplastic Syndromes. V.1.2018. Accessed at on October 12, 2017.

Last Medical Review: January 22, 2018 Last Revised: January 22, 2018

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