Stem cell transplant (SCT) is usually considered the only curative option for patients with myelodysplastic syndrome (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. Patients with mild low blood counts and few symptoms may be carefully watched without treatment for a while. If low blood counts are causing problems, supportive care treatments such as transfusions, blood cell growth factors, and possibly androgens may be helpful.
If a person has the 5q- type of MDS, then lenalidomide (Revlimid) is often used as the first treatment. If this drug doesn’t help, treatment with azacytidine (Vidaza) or decitabine (Dacogen) is often the next option.
Treatment with azacytidine or decitabine is often the first choice for patients with MDS without the 5q- chromosome problem. Azacytidine can be injected under the skin, often for 7 consecutive days every month. The standard dosing of decitabine is to inject it into a vein (IV) every 8 hours for 3 days every 6 weeks. Since this means that the patient has to stay in the hospital for treatment, studies were done to see if the drug would still work if given on a different schedule. One option that seems to work well is to give the drug IV daily for 5 days every 4 weeks. This allows it to be given in an outpatient clinic. The major side effect of these drugs is an early drop in blood counts, as seen with most chemotherapy drugs. If the drug is successful, blood counts will improve to levels that are better than those seen before treatment 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, these drugs can increase life span in some patients.
Other drugs, such as those mentioned previously, have 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 think about taking part in clinical trials of new treatments.
Conventional chemotherapy is an option for some patients with more advanced MDS, such as those whose prognostic scores are high risk or higher (discussed in the staging section), or those whose MDS looks like it is becoming an acute leukemia. Unfortunately, this treatment is too toxic for patients who are elderly or who have many other medical problems and in these patients it can shorten their lives. For young and healthy patients, though, the treatment is similar to treatment for acute myeloid leukemia.
Last Revised: 07/02/2015