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Chemotherapy for Myelodysplastic Syndrome (MDS)

At this time, traditional chemotherapy protocols are not effective for treating MDS. High-dose chemotherapy may be used before a stem cell transplant to rid the body of cancer cells. It may also be used for MDS that has progressed to acute myeloid leukemia or to relieve symptoms associated with the disease. If chemotherapy is used, it does not offer a cure.

Chemotherapy Drugs and Delivery

There are three combinations of chemotherapy drugs used to treat MDS. These combinations include:

  • Cytarabine and idarubicin
  • Cytarabine and topotecan
  • Cytarabine and fludarabine

Chemotherapy for MDS is usually given through an IV, but some forms can be injected under the skin. It is delivered in cycles over a set period of time. A medical oncologist will determine how many cycles of chemotherapy are needed and what combination of drugs will work best. It is not unusual for a relapse to occur after treatment. If so, the combination of chemotherapy drugs be changed.

Side Effects and Management

Though the drugs are targeted to cancer cells, they can affect healthy cells as well. The death of cancer cells and impact on healthy cells can cause a range of side effects. A medical oncologist will work to find the best drug combination and dosage to have the most impact on the cancer cells and minimal side effects on healthy tissue. Side effects or complications from chemotherapy may include:

  • Low red or white blood cell, or platelet counts
  • Nausea or vomiting
  • Loss of appetite
  • Hair loss
  • Mouth sores
  • Fever
  • In high doses, brain dysfunction

A variety of treatments are available to help manage side effects including medication, lifestyle changes, and alternative treatments. In some cases, the chemotherapy regimen may be adjusted to reduce severe side effects. The earlier the side effects are addressed, the more likely they will be controlled with a minimum of discomfort.


Chemotherapy for myelodysplastic syndromes. American Cancer Society website. Available at: Updated February 10, 2014. Accessed June 30, 2016.

Cytarabine. EBSCO DynaMed Plus website. Available at: Updated June 16, 2017. Accessed June 30, 2017.

Myelodysplastic syndrome. EBSCO DynaMed Plus website. Available at: Updated January 13, 2017. Accessed June 30, 2017.

Treatment option overview. National Cancer Institute website. Available at: Updated August 12, 2015. Accessed June 30, 2017.