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The physician may also decide that the patient with myelodysplastic syndromes (MDS) should receive AML-like chemotherapy. This approach can also cure some patients with MDS. However, as is the case with transplantation, this approach usually works best for younger patients who have no chromosome abnormalities.

The main goal of intensive chemotherapy is to eradicate the myelodysplastic clone and restore the ability of the bone marrow to produce normal cells. Intensive chemotherapy has been reported to produce remission in up to 64% of patients with RAEB or RAEB-T. Unfortunately, these remissions are typically short in duration and, in most studies, the median duration of complete remission in high-risk MDS patients was less than 12 months.

Factors that impact the success of intensive chemotherapy include the patient's age (younger patients may have a better prognosis), RAEB-T subtype, whether the patient has primary or secondary MDS, the time that has elapsed from diagnosis to treatment, and karyotype assessment. Another factor is that some patients experience multi-drug resistance.

In addition to killing abnormal cells, chemotherapy may also affect healthy bone marrow cells. The side effects of chemotherapy can include:

• Hair loss
• Mouth sores
• Decreased appetite
• Nausea
• Vomiting
• Increased susceptibility to infections

The side-effects of chemotherapy usually cease with the completion of treatment.

Chemotherapeutic agents that may be used for the treatment of myelodysplastic syndromes include:

• 5-azacytidine

  • 5-azacytidine was recently approved by the U.S. Food and Drug Administration (FDA) for treatment of MDS.
  • in a clinical trial in which patients were assigned to receive either no treatment or treatment with 5-azacytidine, this drug was shown to delay progression to AML and probably improve survival, although the median improvements in survival were only in the range of 1-2 years.

• Decitabine

  • decitabine may prove to be more effective than 5-azacytidine for the treatment of MDS and is currently being investigated in clinical trials.

• Amifostine

  • amifostine is a drug that originally appeared to be effective for MDS but now seems much less so.

• Arsenic trioxide

  • arsenic trioxide is another drug that is under investigation for the treatment of MDS.