Types of Treatments

 For each of our MDS patients, we 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. There are a number of treatment options:

  • 5-azacytidine was approved by the Food and Drug Administration (FDA in 2004 for the treatment of MDS. The drug (also known as Vidaza™) improves bone marrow function by freeing up genes the 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 also 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 (Revlimid™) is FDA-approved for 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 is an option for MDS patients that 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.

  • 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 is an option for patients whose bone marrow has a large number of blast cells, particularly if the goal is to decrease the bone marrow blast count in preparation for a stem cell transplant procedure.

  • Investigational Approaches available through clinical trials are an option as well. Medical researchers are continually exploring new treatment options for MDS and offering patients access.

Patients may also benefit from additional treatments, such as erythropoietin (Procrit™) and darbepoetin (Aranesp™). Both options 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 (Neupogen™) and pegfilgrastim (Neulasta™).