Standard Therapy

In the last few years, we have had 3 new drugs approved for the treatment of this disease. We have many options for treatment and decide on treatment based on the risk classification of the disease. The main goal of MDS treatment is to increase the number of healthy cells in your blood (blood count). Another goal in patients with higher risk disease is to prevent the transformation of MDS to leukemia.

There are many approaches used in the treatment of MDS depending on the type MDS you have and severity of your case. For each of our MDS patients, our physicians devise a personalized course of treatment that takes a number of factors into consideration the severity of the disease and your age and overall health status.

Supportive Care

Supportive care — the goal of which is to help alleviate or prevent symptoms from low blood counts ¬†and all patients with MDS eventually require supportive care. Even patients receieving other treatments for MDS receive supportive care.

Supportive care includes the use of blood and platelet transfusions for patients with dangerously low red blood cell and platelet counts, as well as antibiotics to treat infections. Patients may also benefit from taking injections such as erythropoietin (ProcritTM) and darbepoetin (AranespTM), which stimulate the bone marrow to produce red blood cells. Injections of other medications that stimulate the bone marrow to produce more white blood cells include granulocyte colony-stimulating factor (NeupogenTM) and pegfilgrastim (NeulastaTM).

These treatments may help to improve the blood counts temporarily and to reduce or eliminate symptoms from the low blood counts, but they do not fix the underlying cause.

Other Treatment Options


Also known as VidazaTM, 5-azacytidine was approved by the FDA in 2004 for the treatment of MDS. The drug improves bone marrow function by freeing up the genes your body needs to make normal blood cells. When the bone marrow function improves, blood counts increase, and in some patients the blast count in the bone marrow goes back to normal levels. When bone marrow function improves, the development of leukemia is lessened or delayed, and the survival of MDS patients can be prolonged. Physicians generally prescribe this drug for patients who have significantly low blood counts (cytopenias including neutropenia, thrombocytopenia, and transfusion-dependent anemia), and/or an increased number of bone marrow blasts.


Decitabine is another drug that is FDA approved for MDS. It is similar to 5-azacytidine in many ways, including the way it works, and its ability to improve bone marrow function, increase blood counts, and decrease bone marrow blast counts. Decitabine can also delay the development of leukemia in patients with MDS. Decitabine and 5-azacytidine have similar side effects, including a temporary lowering of blood counts.


Lenalidomide (RevlimidTM) is also FDA approved for MDS, but only for a relatively rare subtype of MDS with a specific chromosome 5 abnormality known as deletion 5q abnormality. When MDS patients have the deletion 5q chromosome abnormality, there is a very high chance that lenalidomide will improve the hemoglobin levels in anemic MDS patients. Lenalidomide can work in some patients who do not have the deletion 5q abnormality, but not as well as with those with the deletion 5q subtype. Lenalidomide has to be used very carefully, because even if it improves the hemoglobin levels (the anemia), it will actually decrease the neutrophils, which are the body’s first line of defense against most infections, as well as the platelet count. These lowered counts will increase the risk of bleeding and infection.

Stem Cell Transplantation

MDS patients may be able to receive a stem cell transplant, which has the potential to be curative. Stem cell transplants may be considered in patients up to the age of 70 who are otherwise healthy. In order to receive a stem cell transplant, patients must have a stem cell donor (a family member or an unrelated volunteer stem cell donor). In addition, patients may need MDS treatment before a stem cell transplant.


Another treatment approach, called immunosuppression, uses antithymocyte globulin (ATG) to suppress the activity of white cells that, in some cases of MDS, interfere with normal blood cell production. This approach works temporarily in less than half of patients.

Leukemia Therapy

For patients whose bone marrow has a large number of blast cells, physicians may use therapy for acute leukemia, particularly if the goal is to decrease the bone marrow blast count in preparation for a stem cell transplant procedure.

Investigational Approaches

Lastly, researchers are continually exploring new treatment options for MDS.

Click here to continue reading about ongoing clinical trials.