Chemotherapy is the use of drugs to kill cancer cells. Unlike radiation and surgery, which are localized treatments, chemotherapy is a systemic treatment, meaning the drugs travel throughout the entire body. This means chemotherapy may reach every cell in the body.

Many people with MDS do not require chemotherapy treatment other than supportive treatments such as intermittent transfusions. However, at times chemotherapy may be required if no other options exist.

Chemotherapy Drugs Used for MDS

  • Cytosine arabinoside (Cytarabine)
  • Azacytidine (Ladakamycin)
  • Topotecan (Hycamtin)
  • Fludarabine (Fludara)
  • Idarubicin (Idamycin)

These agents have been used alone, in combination with other anticancer drugs, and with drugs in other categories, such as hematopoietic growth factors. No consensus has been achieved and no improvement in the ultimate outcome has been realized except with a new experimental drug called AzaC.

Recently, thalidomide has been used in people with MDS. One study has suggested that transfusion requirements may diminish in patients with certain subtypes of MDS treated with thalidomide.

Effectiveness of Chemotherapy

A number of anti-leukemia chemotherapy regimens have induced short-term remissions in as many as 60% of people with MDS, but at substantial risk. The length of these intensive treatment regimens may run between six and nine months. Aggressive anti-leukemia treatment schedules produce a mortality rate up to 30%. Less aggressive programs are suitable for some patients, but again the responses are brief.

This form of treatment may benefit patients who are candidates for bone marrow transplant (BMT). Only BMT and AzaC appear to improve overall survival or change the natural history of the disease at this time.

Low-dose chemotherapy treatment may briefly benefit certain classes of patients, such as the elderly, by exposing them to lower risk.

Side Effects and Possible Complications

Side effects of chemotherapy for MDS are identical to those seen in leukemia and other forms of cancer. The only unique aspect of treating MDS is the advanced age of the patients, which makes many side effects more likely and more dangerous.