Chemotherapy without SCT for MDS


Patients with MDS who are unable or unwilling to undergo a stem cell transplant using donor cells or those planning to have their own stem cells collected for a future transplant can receive conventional chemotherapy without stem cell support. While many patients have a remission with this type of treatment, most ultimately experience disease progression. While some patients may experience a long remission following therapy, remissions after conventional chemotherapy typically average less than 12 months.

Because most patients with MDS are over 65 years of age and cannot tolerate the side effects of conventional chemotherapy, lower doses of chemotherapy may be beneficial.

Vidaza® (azacitadine): In May of 2004, the U.S. Food and Drug Administration (FDA) approved Vidaza for the treatment of MDS. Vidaza is the first drug to be approved specifically for the treatment of MDS.

Results of a clinical trial that compared Vidaza to supportive care in the treatment of 191 patients with MDS demonstrated that the patients treated with Vidaza experienced more anticancer responses and were less likely to develop AML compared to patients who received supportive care. 1

Among patients treated with Vidaza, 15.7% had an anticancer response while none of the patients who received supportive care had a response. Following treatment with Vidaza, all patients who responded to treatment and had previously required blood transfusions no longer required the transfusions to maintain appropriate hemoglobin levels. 2

Patients who received Vidaza experienced an improved quality of life compared to those receiving supportive care. 3

Fludarabine plus cytarabine: Physicians from Italy have reported that the chemotherapy regimen consisting of fludarabine and cytarabine with the white blood cell growth factor, Neupogen® (filgrastim), produced remission in three-quarters of patients with advanced MDS. The 42 patients involved in this study survived 13 months, on average. Nine percent of patients died from complications of the treatment. 4

To learn about other chemotherapy treatment options for MDS, go to Treatment for Acute Myeloid Leukemia.

Strategies to Improve Chemotherapy without Stem Cell Transplant for MDS

The development of more effective cancer treatments requires that new and innovative therapies be evaluated with cancer patients. Clinical trials are studies that evaluate the effectiveness of new drugs or treatment strategies. Future progress in the treatment of MDS will result from the continued evaluation of new treatments in clinical trials. Participation in a clinical trial may offer patients access to better treatments and advance the existing knowledge about treatment of this cancer. Patients who are interested in participating in a clinical trial should discuss the risks and benefits of clinical trials with their physician. Areas of active investigation aimed at improving the treatment of MDS include the following:

  • Dacogen (decitabine)
  • Hycamtin® (topotecan)

Dacogen (decitabine): A clinical trial has shown that patients treated with Dacogen experienced significantly higher response rates and longer time to disease progression—defined as transformation AML or death—compared to patients who received supportive care. Patients who had not received prior treatment and those with intermediate 2 to high-risk disease received the most benefit from Dacogen. 5

Patients in this study only received three courses of Dacogen. More recent findings, however, suggest that a better response may be possible with multiple courses of therapy.

Topotecan/cytarabine may be more tolerable than standard idarubicin/cytarabine: A clinical study that involved 510 patients and compared four different chemotherapy combinations has revealed that anticancer responses and duration of survival were the same for all four treatments. However, treatment with topotecan/cytarabine resulted in fewer deaths than other regimens and may be considered an alternative to the standard idarubicin/cytarabine combination for the treatment of patients with progressive or high-risk MDS. 6

To learn more about treatment techniques that are being evaluated in clinical trials, go to Strategies to Improve Treatment of MDS.


1Silverman LR, Demakos EP, Peterson BL, et al. Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. Journal of Clinical Oncology. 2002;20(10):2429-2440.

2 Pharmion. Vidaza Prescribing Information. Available at: Accessed March 2006.

3Kornblith AB , Herndon JE, silverman LR, et al. Impact of azacytidine on the quality of life of patients with myelodysplastic syndrome treated in a randomized phase III trial:Cancer and Leukemia Group B Study. Journal of Clinical Oncology. 2002;20(10):2441-2452.

4 Felicetto Ferrara, Franco Leoni, Antonio Pinto, et al. Fludarabine, cytarabine, and granulocyte-colony stimulating factor for the treatment of high risk myelodysplastic syndromes. Cancer.1999;86:2006-2013.

5 Saba H, Rosenfeld C, Issa JP, et al. Clinical benefit and survival endpoints from a phase III trial comparing decitabine (DAC) vs supportive care (SC) in patients with advanced myelodysplastic syndromes (MDS). Proceedings of the 41st Annual Meeting of the American Society of Clinical Oncology. Orlando FL. 2005; Abstract #6543.

6 Kantarjian H, Beran M, Cortes J, et al. Long-term follow-up results of the combination of topotecan and cytarabine and other intensive chemotherapy regimens in myelodysplastic syndrome. Cancer. 2006;106: 1099–1109.


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