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Grand Strand Medical Center
South Strand Medical Center

Chemotherapy for Myelodysplastic Syndrome (MDS)

Chemotherapy is the use of drugs to kill cancer cells. Unlike radiation therapy and surgery, which are localized treatments, chemotherapy is a systemic treatment, meaning the drugs travel throughout the entire body. This means chemotherapy may reach every cell in the body.

Many people with MDS do not require chemotherapy treatment other than supportive treatments, such as intermittent transfusions. However, at times chemotherapy may be required if no other options exist.

Chemotherapy Drugs and Delivery

Standard Chemotherapy

There are 2 combinations of chemotherapy drugs used to treat MDS. These combinations include:

  • Cytarabine and idarubicin
  • Cytarabine and topotecan
  • Cytarabine and fludarabine
Hypomethylating Agents

Hypomethylating agents are drugs that slow down the growth of cells and include:

  • Decitabine
  • Azacitidine
Immunomodulating Therapy

Immunomodulating drugs change the immune system and include:

  • Thalidomide
  • Lenalidomide
Immunosuppression Agents

Immunosuppression agents suppress the immune system and include:

  • Anti-thymocyte globulin (ATG)
  • Cyclosporine

These agents have been used alone, in combination with other anticancer drugs, and with drugs in other categories, such as hematopoietic growth factors.

Thalidomide was initially used to treat MDS but has been replaced with lenalidomide because of fewer side effects. Lenalidomide has been successful in reducing the number of transfusions needed by patients with low-grade MDS.

Effectiveness of Chemotherapy

The only cure for MDS is a stem cell transplant (SCT). A SCT starts with high-dose chemotherapy to kill the bone marrow and is then followed by an injection of healthy stem cells. The stem cells used can come from the blood or bone marrow.

There are 2 types of SCT. The less common one is called an autologous SCT, and occurs when the patient is injected with his own cells after high-dose chemotherapy. The more common method is called allogeneic and occurs when the patient gets donor cells after high-dose chemotherapy.

Low-dose chemotherapy treatment may briefly benefit certain classes of patients, such as the elderly, by exposing them to lower risk.

Side Effects and Possible Complications

Side effects of chemotherapy for MDS are similar to those seen in leukemia and other forms of cancer. The only unique aspect of treating MDS is the advanced age of the patients, which makes many side effects more likely and more severe.

Revision Information

  • Castro-Malaspina H, O’Reilly RJ. Aplastic anemia and the myelodysplastic syndromes. In Harrison's Principles of Internal Medicine. 14th ed. New York, NY: McGraw-Hill; 1998.

  • Chiodi S, Spinelli S, Ravera G, et al. Quality of life in 244 recipients of allogeneic bone marrow transplantation. Br J Haematol. 2000;110(3):614-619.

  • Drug Facts and Comparisons. 56th ed. St. Louis, MO: Facts & Comparisons; 2001.

  • Léger CS, Nevill TJ. Hematopoietic stem cell transplantation: a primer for the primary care physician. CMAJ. 2004;170(10):1569-1577.

  • Myelodysplastic syndromes. American Cancer Society website. Available at: Accessed December 7, 2015.