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The best treatment for a person with myelodysplastic syndromes (MDS) depends on the type of MDS, risk score, age, overall health, and his or her own preferences. In addition to transplant, other treatment options include:

Supportive care

Patients with mild disease may receive supportive care. In this “watch and wait” situation, doctors will do frequent blood tests to check for signs that the MDS is becoming worse. Some patients may get platelet or red blood cell transfusions, or both. After many red blood cell transfusions, however, iron builds up in the body, which can cause organ damage. If this happens, patients need additional treatment to remove iron from the body. This is called iron chelation therapy.

Medications to improve marrow function

Some patients may also take medicines, such as G-CSF or erythropoietin, which stimulate the marrow to make more white cells and red blood cells, respectively.

There are other medicines, called hypomethylating agents, which are now being used to treat MDS. Doctors use these drugs to help MDS patients achieve a long-term remission of their disease and to extend their lives.

Sometimes, these medications are used to lessen symptoms and/or the need for transfusions until a patient can have a transplant. Although these medicines can help many patients have a higher quality of life and a longer life, they cannot cure MDS.

Chemotherapy

Chemotherapy is a treatment that uses a group of medicines that destroy diseased cells or stop them from growing. A treatment option for some people with severe MDS is induction chemotherapy. Induction chemotherapy is very intense. The goal is to bring the disease into remission (no more signs of disease).

Induction chemotherapy may be an option for patients in the High or Very High risk categories who are in good health, but do not have a matched donor for an allogeneic transplant. It is also sometimes used to bring a patient with MDS into remission before he or she has a transplant.