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Treatments for Myelodysplastic Syndrome (MDS)

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While standard protocols have been established for the treatment of virtually all cancers, doctors will often modify them for their individual patients. These modifications are based on many factors including the patient’s age, general health, desired results, and the specific characteristics of his cancer.

There is only one generally recognized curative treatment for MDS—allogenic stem cell transplant (SCT), or bone marrow transplant (BMT). SCT may be done in otherwise healthy patients.

Chemotherapy is used to treat advanced MDS. There are three standard combinations of chemotherapy drugs used. These combinations include cytarabine and idarubicin , cytarabine and topotecan , and cytarabine and fludarabine . This treatment can have serious side effects and may not be an option for all patients, especially those who are elderly.

Certain medications have also helped treat the disease. A drug called a hypomethylating agent has shown positive results in treating MDS. Examples include azacitidine (Vidaza) and decitabine (Dacogen). These drugs work by slowing down cell growth. Some patients have shown improved blood counts, reduced risk of leukemia, and a longer life with these drugs. Immunomodulating drugs are also used to treat MDS. These drugs alter the immune system and include thalidomide and lenalidomide (Revlimid). Lenalidomide is generally preferred over thalidomide because of fewer side effects. Immunosuppressant drugs are used to treat certain types of MDS. Anti-thymocyte globulin (ATG) and cyclosporine are both immunosuppressants used in the treatment of MDS.

All other interventions are supportive and depend on which family of blood cells is involved. The mainstays of treatment have been blood component transfusions to replace the deficient cell types and antibiotics to treat the infections. Radiation therapy is not used for the treatment of MDS.

Clinical Trials

Treatment protocols have been established and continue to be modified through clinical trials. The research studies are essential to determine whether or not new treatments are both safe and effective. Since highly effective treatments for many cancers remain unknown, numerous clinical trials are always underway around the world. You may wish to ask your doctor if you should consider participating in a clinical trial. You can find out about clinical trials at the government website .

Revision Information

  • American Cancer Society website. Available at: .

  • 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.

  • Children’s Hospital Boston website. Available at: .

  • Detailed guide: myelodysplastic syndrome. American Cancer Society website. Available at: . Accessed April 2, 2009.

  • Lewis R, Silverman MD. Myelodysplastic syndrome. American Cancer Society website. Available at: . Accessed November 2002.