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Myelodysplastic Syndromes Treatment (PDQ®)
Patient VersionHealth Professional VersionEn españolLast Modified: 02/22/2008



General Information About Myelodysplastic Syndromes






Stages of Myelodysplastic Syndromes







Treatment Option Overview






Treatment Options for Myelodysplastic Syndromes






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Changes to This Summary (02/22/2008)






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Key Points for This Section


There are different types of treatment for patients with myelodysplastic syndromes.

Different types of treatments are available for patients with myelodysplastic syndromes. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Before starting treatment, patients may want to think about taking part in a clinical trial. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with a myelodysplastic syndrome. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment.

Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI Web site. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team.

Treatment for myelodysplastic syndromes aims to relieve symptoms, slow progression, and improve quality of life.

Treatment options for patients with myelodysplastic syndromes range from supportive care that helps relieve symptoms to aggressive treatment that may slow or prevent progression of the disease.

Problems caused by low blood cell counts, such as fatigue and infections, may be treated with transfusions of blood products or the use of growth factors.

Chemotherapy may be used to delay progression of the disease. Other drug therapy may be used to lessen the need for transfusions. Certain patients may benefit from aggressive treatment with chemotherapy followed by stem cell transplant using stem cells from a donor.

Three types of standard treatment are used:

Chemotherapy

Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the spinal column, an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the type and stage of the cancer being treated.

Supportive care

Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include the following:

  • Transfusion therapy

    Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or treatment. Patients who receive frequent red blood cell transfusions may have their tissues and organs damaged from the buildup of extra iron. Iron chelation therapy is a treatment that uses drugs that attach to the extra iron. The drug and the iron are removed from the body in the urine.

    Platelet transfusions are usually given when the patient is bleeding or is having a procedure that may cause bleeding.



  • Growth factor therapy

    Erythropoietin may be given to increase the number of red blood cells and lessen the effects of anemia. Sometimes granulocyte colony-stimulating factor (G-CSF) is given with erythropoietin to help the treatment work better.



  • Drug therapy

    Deferoxamine may be used to treat the build-up of too much iron in the blood of patients receiving blood transfusions. It is sometimes given with vitamin C.

    Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndrome caused by a specific chromosome change.

    Antithymocyte globulin (ATG) may also be used to lessen the need for transfusions in patients with a certain form of myelodysplastic syndrome.

    Antibiotics may be given to fight infections.



Chemotherapy with stem cell transplant

Stem cell transplant is a method of giving chemotherapy and replacing blood-forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of a donor and are frozen for storage. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.

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