© 2005-2012 American Society of Clinical Oncology (ASCO). All rights reserved worldwide.
From the May 15, 2002 Issue of Journal of Clinical OncologyMyelodysplastic Syndrome, or MDS, is a group of disorders resulting in the failure of bone marrow to adequately produce blood cells. People with MDS often have low blood counts, which increase their risk of developing potentially serious or fatal infections or episodes of bleeding. Most patients with high-risk MDS - whose disease has progressed - experience bone marrow failure causing hemorrhage or infection. Approximately one-third of patients with MDS eventually develop acute myelogenous leukemia, or AML.Currently, most patients with MDS are treated with supportive care tailored to their disease symptoms. For example, patients with low red blood cell counts receive blood transfusions, patients with infections receive antibiotics, and patients with low platelet counts receive platelet transfusions. While these treatments are helpful for some patients, they do not repair the problems within the bone marrow. However, according to a new study led by Dr. Lewis Silverman at the Mount Sinai School of Medicine, azacitidine, or Aza C, offers new hope for patients with MDS. Aza C reverses the damaged function of the bone marrow and increases the production of blood cells in patients with MDS. According to the study, performed by the Cancer and Leukemia Group B and funded by the National Cancer Institute, 60 percent of patients who received Aza C responded to the drug. Patients taking Aza C reported an improvement in their symptoms and better quality of life than patients receiving supportive care. Aza C also significantly prevented or delayed the development of acute leukemia. The most common side effects of Aza C are nausea and vomiting, which can be managed through medication. Patients do not need to be hospitalized to receive Aza C, which is not yet approved by the FDA. Patients treated in the study had either advanced stages of MDS or early forms with low blood counts and symptoms.