How Are Myelodysplastic Syndromes Treated?

This content has been reviewed and approved by

Hagop M. Kantarjian, MD
Chairman & Professor, Leukemia Department
MD Anderson Cancer Center
University of Texas

Treatment of myelodysplastic syndromes (MDS) are individualized and depend on two main factors: 

• The severity of low blood counts 
• The risk of progression to AML 

Other factors that influence treatment decisions include the patient's age, other medical conditions, and the severity of the MDS. 

The potential treatment options for MDS include the following: 

1. Observation 
2. Transfusions and growth factors 
3. Immune therapy with ATG, cyclosporine, and/or steroids 
4. Hypomethylating agents, such as Dacogen^TM (decitabine) or Vidaza® (azacitidine) 
5. Revlimid® (lenalidomide) – approved by FDA for low risk MDS, with 5q- cytogenetic abnormality (defect in chromosome 5) and transfusion dependence 
6. Intensive chemotherapy 
7. Allogeneic stem cell transplant 
8. Iron chelation therapy 
9. Gleevec® (imatinib mesylate) for patients with chromosome abnormalities involving a translocation at 5q33 e.g. t(5;12) 
10. Investigational therapies 

The table below outlines the treatment options according to your risk category. 

Currently, only stem cell transplants using cells from a donor—called an allogeneic transplant—can consistently cure patients with MDS. But it is available to less than 10 percent to 20 percent of patients because of their age, other existing illnesses and availability of donors. Other therapies are directed at prolonging survival and decreasing the symptoms from these diseases. 

Dacogen^® (decitabine) and Vidaza^® (5-azacitidine) are FDA approved for the treatment of MDS and chronic myelomonocytic leukemia (CMML). Revlimid^® (lenalidomide) is FDA approved for the treatment of lower risk MDS with 5q abnormality and red cell transfusion dependence.

In some cases, participation in a clinical trial using new, innovative therapies may provide the most promising treatment. Information about treatments for MDS that are being evaluated in clinical trials is discussed under Strategies to Improve Treatment. 

Circumstances unique to each patient's situation may influence how these general treatment principles are applied. The potential benefits of multimodality care, participation in a clinical trial, or standard treatment must be carefully balanced with the potential risks. The information on this website is intended to help educate patients about their treatment options and to facilitate a mutual or shared decision-making process with their treating cancer physician.