Stem cell transplant (SCT) is usually considered the only curative option for patients with MDS, and may be the treatment of choice for younger patients when a matched donor is available. This is the recommended treatment for nearly all children. For older patients, either the high-dose or low-dose approach can be used. For either of these options, it appears best to wait until the disease is advanced before performing the SCT.

When SCT is not an option, MDS is not considered curable. In that case, the goal is to relieve symptoms and avoid complications and side effects of treatment. Some of the treatments that can help with MDS symptoms include transfusions, blood cell growth factors, and possibly hormones.

If a person has the 5q- type of MDS, then lenalidomide is often used as the first treatment. If this drug doesn't help, treatment with azacytidine or decitabine is often the next option.

Treatment with azacytidine or decitabine is often the first choice for MDS without the 5q- chromosome problem. The drug is injected under the skin for 7 consecutive days every month. The major side effect is the early drop in blood counts seen with most chemotherapy drugs. If the drug is successful, the blood counts recover to levels above those seen before chemotherapy was started.

A major benefit for patients receiving azacytidine or decitabine is that they need fewer transfusions and have a better quality of life. In particular, if they respond, they have less fatigue and are able to function more normally. Finally, the drug may increase life span, although this isn’t certain.

Other drugs such as those mentioned previously have also helped some patients. It may be worth joining a clinical trial or receiving these agents outside a trial if none is available.

Careful general medical care and measures to prevent and treat infections are very important. Patients should consider participating in clinical trials of new treatments.