Azacitidine is a pyrimidine nucleoside analogue with anti-neoplastic activity. It differs from cytosine by the presence of nitrogen in the C5-position, key in its hypomethylating activity. Two main mechanisms of action have been proposed for azacitidine. One of them is the induction of cytotoxicity. As an analogue of cytidine, it is able to incorporate into RNA and DNA, disrupting RNA metabolism and inhibiting protein and DNA synthesis. The other one is through the inhibition of DNA methyltransferase, impairing DNA methylation. Due to its anti-neoplastic activity and its ability to inhibit methylation in replicating DNA, azacytidine has been used mainly used in the treatment of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), two types of cancer characterized by the presence of aberrant DNA methylation.
In May 2004, the FDA approved the use of azacitidine administered subcutaneously for the treatment of MDS of all French-American-British (FAB) subtypes. In January 2007, the FDA approved the intravenous administration of azacitidine. The use of oral azacitidine for the treatment of AML in patients in complete remission was approved by the FDA in September 2020.
Azacitidine (for subcutaneous or intravenous use) is indicated for the treatment of adult patients with the following French-American-British (FAB) myelodysplastic syndrome (MDS) subtypes: refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL). Azacitidine is also indicated for the treatment of pediatric patients aged 1 month and older with newly diagnosed Juvenile Myelomonocytic Leukemia (JMML).
Azacitidine (for oral use) is indicated for continued treatment of adult patients with acute myeloid leukemia (AML) who achieved first complete remission or complete remission with incomplete blood count recovery following intensive induction chemotherapy and are not able to complete intensive curative therapy.
Georegetown University, Washington, D.C., District of Columbia, United States
Mayo Clinic, Rochester, Minnesota, United States
St. Francis Hospital, Greenville, South Carolina, United States
Universitair Ziekenhuis Gent, Gent, Belgium
Universitair Ziekenhuis Antwerpen, Edegem, Belgium
Universitair Ziekenhuis Brussel, Jette, Belgium
Albert Einstein College of Medicine, Bronx, New York, United States
USC Norris Comprehensive Cancer Center, Los Angeles, California, United States
Northwestern University, Chicago, Illinois, United States
MD Anderson Cancer Center, Houston, Texas, United States
University of Texas MD Anderson Cancer Center, Houston, Texas, United States
1230.33.33002 Boehringer Ingelheim Investigational Site, Marseille, France
1230.33.33001 Boehringer Ingelheim Investigational Site, Paris, France
1230.33.49002 Boehringer Ingelheim Investigational Site, Dresden, Germany
USC Norris Oncology/Hematology-Newport Beach, Newport Beach, California, United States
Medical University of South Carolina, Charleston, South Carolina, United States
Johns Hopkins Bayview Medical Center, Baltimore, Maryland, United States
University of Southern California, Los Angeles, California, United States
UPMC Cancer Center- Hillman Cancer Center, Pittsburgh, Pennsylvania, United States
Sibley Memorial Hospital, Washington, District of Columbia, United States
Desert Hematology Oncology Medical Group, Inc., Rancho Mirage, California, United States
The University of Texas MD Anderson Cancer Center, Houston, Texas, United States
Mount Sinai Medical Center, New York, New York, United States
Washington University School of Medicine, Saint Louis, Missouri, United States
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