Treatment of MDS depends on several factors, including stage of the disease, age and overall health of the individual, as well as prognosis and likely response. At this time, bone marrow stem cell transplants are the only treatment available that will likely cure MDS. They are often the recommended treatment for children and are increasingly being used in adults with MDS also. Without a successful stem cell transplant, MDS is incurable and the focus of treatment is to alleviate symptoms and prevent complications or progression to acute myeloid leukemia. Treatment needs often change over time.
If anemia is present, then blood transfusions may be necessary. Repeated blood transfusions may lead to the buildup of excess iron in the body and the need for iron chelation therapies. Patients with low blood erythropoietin levels (e.g., <500 IU/L) may benefit from treatment with recombinant erythropoietin, which stimulate red blood cell production.
Platelet transfusions may be necessary to control excessive bleeding. Drugs that stimulate bone marrow platelet production may also be prescribed (e.g., eltrombopag).
Antibiotics may be required when white blood cell (WBC) counts are low and a person experiences recurrent infections. Growth factors such as granulocyte colony stimulating factor (G-CSF) and granulocyte macrophage-colony stimulating factor (GM-CSF) may be given to stimulate WBC production.
Some patients with MDS may be given chemotherapy agents such as 5-Azacitidine and Decitabine, immunomodulating drugs such as Lenalidomide (especially those who have a del(5q) chromosome alteration), or immune suppressants.
New therapies for MDS continue to emerge from research and clinical trials. Individuals should talk to their healthcare providers about the treatments that are best for their condition.