Hematologic Malignancies Program

Myelodysplastic Syndromes (MDS)

Treatment Options

In this group of diseases, those at the chronic and stable end of the spectrum are often not treated.

In patients with more troublesome decreases in blood cell counts, drugs that can stimulate blood cell production may be useful.

There are treatments for all patients with myelodysplastic syndromes. The choice of treatment depends on the type of myelodysplastic syndrome, and the patient's age and general health.

Often the main treatment is giving red blood cells or platelets by a needle in a vein (transfusion) to control anemia or bleeding. Vitamins or other drugs may also be given to treat anemia.

Various treatment options include:

Chemotherapy and biological therapies are being tested in clinical trials. Chemotherapy uses drugs to treat disease. Chemotherapy may be taken by pill, or it may be put into the body by a needle in the vein or muscle. Chemotherapy enters the bloodstream, travels through the body, and affects cells throughout the body. Biological therapy tries to get the body to fight disease. It uses materials made by the body or made in a laboratory to boost, direct, or restore the body's natural defenses against disease.
Stem cell/bone marrow transplantation is a newer type of treatment that uses high doses of chemotherapy and/or radiation therapy (high doses of X-rays or other high-energy rays) to destroy all of the bone marrow in the body, then transplants healthy bone marrow back into the body. Healthy marrow is then taken from another person (a donor) whose tissue is the same or almost the same as the patient's. The donor may be a twin (the best match), a brother, sister, or other relative, or another person not related. The healthy marrow is given to the patient through a needle in the vein, and the marrow replaces the marrow that was destroyed. A bone marrow transplant using marrow from a relative or person not related to the patient is called an allogenic bone marrow transplant.

In an autologous stem cell transplant, a patient's own stem cells are removed from his or her bone marrow or bloodstream. This type of transplant is used in treating some types of cancer but is not used for patients with MDS because it is not possible to get normal stem cells from the patient that are not MDS cells.
Growth factors: Erythropoietin, a growth factor that promotes red blood cell production, can help avoid red blood cell transfusions in some patients. Recently it has been found that combining erythropoietin with G-CSF improves the patient's response to the erythropoietin. Research on a new growth factor called thrombopoietin, which promotes platelet production, is in progress.

Differentiation agents: Differentiation agents cause bone marrow blasts to develop into mature blood cells. These drugs have shown promising results in studies of bone marrow cells grown in laboratory dishes; however, their value in patients with MDS is less clear. Several differentiation agents, including retinoids (chemicals related to vitamin A) and vitamin D, are being tested in clinical trials to find the best way of combining these agents together or with other treatments.
Supportive therapy: For many patients with forms of MDS such as RA (refractory anemia) and RARS (refractory anemia with ringed sideroblasts), as well as other patients with favorable prognostic factors, the risk of progression to acute leukemia is low. The main goal of treatment is to prevent symptoms and complications of low blood cell counts. For example, low red blood cell counts (anemia) can cause severe fatigue. Patients with MDS and anemia often benefit from receiving red blood cell transfusions.

This page was last updated on: August 6, 2008.