Treatment Options
Myelodysplastic syndrome (MDS) may cause only mild symptoms, with little effect on health, or may be very severe. Your doctor will base treatment decisions on the type and severity of your MDS.
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.
Another approach to MDS treatment is supportive care—measures to help reduce symptoms. This may include:
- Medicines—including growth factors to increase your red blood cell, white blood cell or platelet counts, and medicines to manipulate your immune system
- Transfusions—to add red blood cells or platelets to your bloodstream if medicines do not increase your levels enough
- Antibiotics—to fight bacterial or fungal infections that your white blood cells cannot fight on their own
- Antiviral drugs—to fight certain viral infections that respond to these medicines
If you have more severe symptoms, your doctor is likely to recommend treatment that goes beyond supportive care. This may include chemotherapy, which can lower blood cells counts at first but may lead to remission (a period of no detectable disease activity), stem cell transplantation or other newer treatments. You will find information in this section about each of these forms of treatment.
Clinical Trials
While there are exceptions, most patients with MDS should be treated in clinical trials. Information in this section explains why, and there are links here to help you learn more about clinical trials. If you are eligible for such a trial, we strongly recommend that you consider this option for yourself because trials offer a way to get the best and latest treatments and also contribute to further improvements in care for all patients.
More Information
Chemotherapy
Doctors may use chemotherapy for people with myelodysplastic syndrome (MDS). 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 kill abnormal cells that are in the blood and bone marrow as well as those that may have spread to other tissues in the body. Chemotherapy is one way doctors can use to help control your body’s production of abnormal bone marrow cells.
For patients who are having a stem cell transplant, doctors typically use a special chemotherapy regimen. You can read more about this in our detailed adult transplantation section.
Seattle Cancer Care Alliance (SCCA) patients who have MDS receive chemotherapy at the SCCA facility near downtown Seattle.
Transplantation
Patients with severe or advanced MDS often are treated with a hematopoietic stem cell transplant (marrow or peripheral blood), currently the only type of treatment that has the potential of curing the disease. 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.
The chemotherapy given before a transplant and the transplant itself are intensive treatments designed to kill diseased stem cells in the marrow. The most intensive type of pre-transplant conditioning regimen is called myeloablative. This type of regimen completely destroys the patient’s own bone marrow and immune system. A newer approach, using less intensive regimens, is called nonmyeloablative or reduced-intensity conditioning. (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 any remaining abnormal cells in the marrow by an immune attack on those cells.
Donor cells are infused following the conditioning regimen. This is the actual transplant. The intent is that the donor stem cells will settle into the patient’s bone marrow, a process called engraftment. There they will begin producing healthy blood cells and immune cells. This new immune system will then recognize any remaining diseased cells and eliminate them.
Such aggressive treatment 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 reduced-intensity regimen as described above may be an option.
Doctors continue to look for other treatment options for patients who cannot have a transplant at all.
New Treatments
Doctors continue to search for new treatments and possible cures for myelodysplastic syndrome (MDS). 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, at Seattle Cancer Care Alliance (SCCA) doctors have been studying a drug called azacitidine (Vidaza) in combination with another drug called etanercept (Enbrel). Several patients taking part in this research have now been followed for two years and still continue to do well and not to require transfusions. However, eventually, the disease is expected to advance in all patients. More research needs to be done to find out when and how to use these drugs for the best results. Other drugs that are being studied include arsenic trioxide (Trisenox), lenalidomide (Revlimid), tipifarnib (Zarnestra), decitabine (Dacogen), AMG-531, clofarabine (Clofarex, Clolar) and others.
Researchers at the Fred Hutchinson Cancer Research Center and the SCCA are also working on a number of other topics related to MDS, including these:
- 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 refer to as MDS is actually a collection of different diseases with some important distinctions. Ideally, doctors will be able to match patients 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 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 a transplant?
- 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.