Myelodysplastic Syndromes

Treatment

No known drugs cure myelodysplastic syndromes (MDS), and age or serious medical conditions may keep many MDS patients from receiving aggressive chemotherapy treatments. Hematologists at Mayo Clinic work hard to help each MDS patient manage his or her disease with the best treatment possible.

Supportive Care with Growth Factors and Transfusions

Most MDS patients who suffer from anemic symptoms can try the drugs erythropoietin or darbepoetin -- growth factors that promote red cell production. Unfortunately, not all patients respond to these medicines. In rare circumstances, growth factors that promote white cell or platelet production may be tried, but these are generally not very effective with MDS. Red blood cell transfusions may be necessary to keep levels high enough to avoid anemic symptoms such as shortness of breath, chest pain and fatigue. Antibiotics may be needed to treat infections, and platelet transfusions may be required to prevent or stop bleeding. Some patients with milder forms of MDS may be able to survive for many years with this type of "supportive care," using growth factors, transfusions and antibiotics as needed.

Chemotherapy and Bone Marrow Transplant

For young and healthy MDS patients, especially those with aggressive forms of the disease, chemotherapy may be used. For some patients, very high doses of chemotherapy and radiation designed to destroy bone marrow cells followed by an infusion of blood or marrow stem cells from a matched family member or unrelated donor can sometimes cure the disease. However, the traditional form of bone marrow transplant, known as a "myeloablative" transplant, is associated with a very high risk of life-threatening side effects and is not appropriate for many MDS patients.

To reduce the risks of life-threatening side effects while still taking advantage of transplantation, doctors at Mayo Clinic in Rochester are testing a "non-myeloablative" approach. This approach involves administering low doses of chemotherapy. Patients tolerate this approach better and can then receive an infusion of peripheral blood stem cells from a matched (related or unrelated) donor. Non-myeloablative transplant has been termed "mini-transplant." While still associated with side effects, physicians hope that this approach will be safer and as effective as myeloablative transplant.

Mayo Clinic has long been a world leader in the treatment of patients with myeloid disorders and has an active transplant program that has been performing bone marrow transplants (BMT or bone and marrow transplants) for over 20 years.

Medical Therapy and Clinical Trials

The U.S. Food and Drug Administration has not approved any drugs designed specifically to treat MDS. Thalidomide pills will improve a few patients' blood counts but may cause significant side effects including rash, nerve damage, constipation and sedation. An intravenous drug called 5-azacytidine has recently been shown in a controlled study to improve quality of life, decrease risk of developing leukemia and slightly improve survival in MDS patients. This drug is currently only available on a limited basis through a program run by the National Cancer Institute. Several hematologists at Mayo are registered investigators with the National Cancer Institute and can help appropriate patients obtain the drug. Patients with sideroblastic anemia (a subtype of MDS) usually try pyridoxine (vitamin B6) for a time to see if it helps. How and when these and other therapies should be used must be decided after detailed discussion between the patient and an experienced hematologist.