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Categories: Myelodysplastic Syndromes
February 2005 Volume 3, Issue 2
Joan M. Latsko, MSN, CRNP, OCN, Richard Stone, MD, Richard K. Shadduck, MD, and Cheryl Breed, NP, MSN
The United States Food and Drug Administration approved azacitidine in May 2004 for the treatment of patients with the following subtypes of myelodysplastic syndrome (MDS): refractory anemia; refractory anemia with ringed sideroblasts accompanied by neutropenia or thrombocytopenia requiring transfusions; refractory anemia with excess blasts; refractory anemia with excess blasts in transformation; and chronic myelomonocytic leukemia. Azacitidine, the first drug approved for the treatment of MDS, belongs to the class of compounds called demethylation agents. Azacitidine induces DNA hypomethylation, which may restore normal function to genes that are critical to differentiation and proliferation. It is also directly cytotoxic to abnormal hematopoietic cells in bone marrow, which causes the death of rapidly dividing cells including cancer cells that are no longer responsive to normal growth control mechanisms. This monograph addresses important issues pertaining to the use of azacitidine, including dosing, administration, and the management of side effects. Practical guidelines and suggestions are provided for managing the 7-days-per-week dosing regimen, as well as for determining when to stop therapy and other issues that clinicians are likely to face as they begin to use azacitidine in their practices.