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 strive to provide treatments that help MDS patients best manage their disease.

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. Not all patients respond to these medicines. In rare circumstances, growth factors that promote white blood cell or platelet production may be tried, but these are generally not very effective with MDS. Red blood cell transfusions may be necessary to avoid symptoms of anemia, 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 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 that involves low doses of chemotherapy. Patients may 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 the transplant still has side effects, physicians hope this approach will be safer and as effective as myeloablative transplant for AML.

Mayo Clinic is a world leader in treating patients who have myeloid disorders and has been performing bone and marrow transplants for over 20 years.

Other Medications

The Food and Drug Administration has approved several drugs specifically for the treatment of myelodysplastic syndromes. These medications may improve blood counts and reduce the need for transfusions:

• Azacitidine (Vidaza) and decitabine (Dacogen) are injectable drugs which may improve quality of life and help delay progression to acute myeloid leukemia. Azacitidine has been shown to improve survival in patients with higher-risk myelodysplastic syndromes.
• Lenalidomide (Revlimid), which is taken in pill form, is sometimes called immune modulating therapy. It has been most helpful to those who have acquired abnormalities of chromosome 5.