Treatment Options

Myelodysplastic syndrome (MDS) may cause only mild symptoms, with little effect on health, or may be very severe. Your doctor will base your treatment plan on the severity of your MDS and the subtype that you have.

Your treatment may also depend on these factors:

• Your age and overall health
• Whether your MDS developed after you received chemotherapy or radiation for another disease or were exposed to other known MDS risk factors (called secondary MDS)
• Whether you’ve already received treatment for MDS and the disease has not improved

Treatment can vary greatly from person to person. Your plan will be customized to you.

Watchful Waiting
 

If your disease is mild and interferes only a little or not at all with your usual activities, your doctor may recommend “watchful waiting.” This means you receive no treatment, but you visit your doctor on a regular basis (or if you notice changes) to check your health. Some people with MDS go for several years with no major health changes.

Supportive Care

Another approach to MDS treatment is supportive care—measures to help reduce symptoms. For MDS, these measures may include medicines and transfusions.

Medicines used in supportive care for MDS include the following:

• The growth factor erythropoietin (EPO) to increase your red blood cell count
• The growth factor granulocyte colony-stimulating factor (G-CSF) or granulocyte macrophage colony-stimulating factor (GM-CSF) to increase your white blood cell count
• Medicines to manipulate your immune system
• Medicines that may reduce your need for transfusions, such as lenalidomide (Revlimid) and antithymocyte globulin (ATG)
• Antibiotics and antiviral drugs to fight infections that your white blood cells cannot fight on their own

Transfusions are used to add red blood cells or platelets to your bloodstream if medicines do not increase your levels enough. Frequent transfusions of RBCs may cause too much iron to build up in your body. If this happens, you will need chelation therapy, which means taking medicines (deferoxamine or deferasirox) that help your body expel excess iron through your urine.

Disease Treatment
 

If you have more severe symptoms, your doctor is likely to recommend treatment that goes beyond supportive care. This may include drug treatment; induction chemotherapy, which can lower blood cells counts at first but may lead to remission (a period of no detectable disease activity); bone marrow transplantation; or other newer treatments. You may need a combination of supportive measures to reduce symptoms and treatments aimed at controlling your underlying disease.

Most patients with MDS receive treatment through clinical research studies, or clinical trials, because studies offer a way to get the best and latest treatments. Studies also help improve care for all patients. Your doctor can identify studies that match your circumstances and talk with you about whether and how to participate. If you are eligible for a study, we strongly recommend you consider this option for yourself. Read more about participating in a study.

Drug Treatment

In addition to lenalidomide (used to reduce the need for transfusions), two other drugs are approved by the U.S. Food & Drug Administration to treat MDS. These are azacitidine (Vidaza) and decitabine (Dacogen). Azacitidine, given as an injection, may help improve RBC, WBC and platelet counts, and reduce blasts. Decitabine, also given as an injection, may be toxic to abnormal MDS cells and promote growth of normal blood cells. Many questions remain as to how azacitidine and decitabine can be used most effectively. Research studies addressing those questions are ongoing.

Induction Chemotherapy

Doctors may use intensive anti-cancer drugs, or chemotherapy, like that used for leukemia, to control MDS by killing myelodysplastic cells. Chemotherapy is given intravenously, by mouth or directly into the spinal fluid (if you need treatment in your central nervous system, which is rare in patients with MDS). These medicines are distributed throughout the body through the bloodstream. They can help control your body’s production of abnormal blood cells.

Side effects are an important concern for many people receiving chemotherapy. Besides killing cancer cells, chemotherapy can damage healthy tissue and cause side effects like fatigue, hair loss, digestive problems, mouth sores, skin irritation, and low levels of healthy blood cells. Your doctor will talk with you about the specific side effects you might develop and what to watch for. We will help you take the best possible care of yourself to prevent, minimize, or relieve side effects so you can focus your energy on your recovery.

Transplantation

Patients with severe or advanced MDS often are treated with a hematopoietic cell transplant (a bone marrow transplant or a transplant using peripheral blood stem cells, those circulating in the blood). This is currently the only type of treatment that has the potential of curing MDS. In some patient groups, the success rate (meaning how many patients are cured of their disease) is as high as 70 to 75 percent. Some of these patients have now been followed for 20 years or longer by their health care teams, with no sign that the disease has returned.

Depending on their situation, patients with MDS may receive either an autologous transplant (using their own stem cells) or an allogeneic transplant (using stem cells from a donor.

Patients having an autologous transplant first undergo intensive chemotherapy designed to kill diseased stem cells in the marrow. This type of pre-transplant conditioning regimen is called myeloablative. It completely destroys the patient’s own bone marrow and immune system.

Patients having an allogeneic transplant also undergo conditioning. Some have myeloablative conditioning; others have lower-intensity conditioning, called nonmyeloablative. (You may also have heard this called mini-transplant.) This type of regimen destroys some of the bone marrow cells. The intent is to suppress the immune system enough so that the donor cells are not rejected and can help to kill remaining abnormal cells in the marrow by an immune attack on those cells.

A transplant is not appropriate for all patients. For instance, a transplant may be too challenging for older individuals, especially if they have other health problems besides MDS, as many older people do. Though some patients have had successful transplants at age 70 years, it is not the standard treatment in this age group. For some patients who cannot undergo a myeloablative transplant, a nonmyeloablative transplant may be an option. Read more about our Transplant Program.

New Treatments

Doctors continue to search for new treatments and possible cures for MDS.

In addition to the treatments described above, we offer numerous agents that are not commercially available but have shown promising results in pilot studies and are being tested in clinical trials. Examples include clofarabine (Clofarex, Clolar) given by mouth, SB1518 (a tyrosine kinase inhibitor), azacitidine (Vidaza) given by mouth (the commercially available drug is given by frequent injection), aurora kinase inhibitors, pomalidomide and others.

One research path has been to investigate how MDS develops—its pathophysiology, or what goes wrong in the bone marrow. By learning more about the chain of events that leads to severe cases of MDS, doctors hope to be able to block one or more of these events and at least stabilize the disease.

For instance, we have been studying azacitidine in combination with another drug called etanercept (Enbrel).  This combination has resulted in improved blood cell counts in almost 70 percent of patients. Responses occurred faster than with azacitidine alone, and in a proportion of patients even the bone marrow became normal. Several of the patients treated this way have now been followed for more than two-and-a-half years, still maintaining their counts and not requiring transfusions.

Among the other drugs being studied for use in MDS are arsenic trioxide (Trisenox), lenalidomide (Revlimid), tipifarnib (Zarnestra), decitabine (Dacogen), AMG-531, vorinostat (Zolinza), and others. Additional studies are trying to determine whether the patient’s immune cells can be modified so that they will attack the MDS cells and eliminate them, a type of treatment called immunotherapy.

Researchers at the Fred Hutchinson Cancer Research Center and SCCA are also working on a number of other topics related to MDS, including the following:

• If doctors can identify subtypes of MDS more precisely based on what they see in affected cells, they hope to come up with more specific treatments. They know that what we call MDS is actually a collection of several different diseases with some important distinctions. Ideally, doctors will be able to match each patient with the most effective drug, or combination of drugs, for his or her subtype and severity of MDS.
• Several studies are underway to figure out the best approach for patients with MDS who receive a hematopoietic (stem) cell transplant. Researchers are investigating questions such as these:
  • Which intensity of chemotherapy leads to better results with fewer side effects depending on a patient’s situation?
  • At which risk level and which age should patients be considered for transplantation?
  • Which other factors about the patient or the disease can help doctors determine whether and when to offer a transplant?
• To help prevent MDS in the future or diagnose and treat it early, researchers are trying to understand what causes MDS, including whether factors in the environment play a role.