Myelodysplastic Syndrome: Treatment

For each patient with myelodysplastic syndrome (MDS), Memorial Sloan Kettering physicians devise a course of treatment that takes a number of factors into consideration, including:

  • the severity of the disease, including the IPSS score and the FAB or WHO category (see Classification & Staging)
  • any prior treatments
  • likelihood of the patient being a candidate for stem cell transplant
  • patient's age and overall health

Stem Cell Transplantation

People with MDS may be able to receive a stem cell transplant, which has the potential to cure MDS. Learn more about our transplantation program.

Stem cell transplants may be considered as a treatment option if you are below the age of 70 and are otherwise healthy. In order to receive a stem cell transplant, you must have a stem cell donor (a family member or an unrelated volunteer stem cell donor). In addition, you may need to undergo treatment for MDS before a stem cell transplant.

Supportive Care

Supportive care — the goal of which is to help alleviate or prevent symptoms from low blood counts — is the cornerstone of all MDS treatments. All patients with MDS eventually require supportive care. Even if you are receiving chemotherapy for MDS  or a stem cell transplant, you will receive supportive care.

Supportive care includes the use of blood and platelet transfusions for people 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 (Procrit®) and darbepoetin (Aranesp®), which stimulate the bone marrow to produce red blood cells. Injections of other medications that coax the bone marrow to produce more white blood cells include granulocyte colony-stimulating factor (Neupogen®) and pegfilgrastim (Neulasta®).

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 of MDS.

Other Treatment Options

For patients who are not eligible for a stem cell transplant, Memorial Sloan Kettering offers the following treatment options:

5-azacytidine

Also known as Vidaza®, 5-azacytidine was approved by the US Food and Drug Administration 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.

The development of leukemia is also 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

Decitabine is another drug that is FDA approved to treat 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 and prolong the survival of patients with MDS. Decitabine and 5-azacytidine have similar side effects, including a temporary lowering of blood counts.

Lenalidomide

Lenalidomide (Revlimid®) 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.

Immunosuppression

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

Researchers are continually testing new treatments for MDS, and many of these studies originate at Memorial Sloan Kettering. These investigational approaches are offered to eligible patients here through the clinical trial process, which is designed to advance the current standard of care for MDS.

By treating patients with MDS in clinical trials, Memorial Sloan Kettering physicians have shown that 5-azacytidine, decitabine, and lenalidomide are safe and effective treatments for MDS. These clinical trials led to FDA approval of these drugs, and in this same way, we hope to develop even better treatments for MDS.

As with all cancer treatment at Memorial Sloan Kettering, clinical trials involve a team approach, in which physicians, scientists, and pathologists work together to care for each patient. We are expert at determining which patients will derive the most benefit from which trial.