A Phase II Study of Intravenous Azacitidine Alone in Patients With Myelodysplastic Syndromes

Inclusion Criteria

• Pathological MDS either de novo or secondary, fitting any of the FAB classifications, confirmed by institutional pathologist within 2 weeks prior to start of treatment. Patients with 5% bone marrow blasts must also meet one of the following criteria:
• Symptomatic anemia with either hemoglobin less than 10.0 g/dL or requiring RBC transfusion
• Thrombocytopenia with a history of two or more platelet counts < 50,000 / µL or a significant hemorrhage requiring platelet transfusions, or
• Neutropenia with two or more absolute neutrophil counts less than 1,000 /µL.
• ECOG performance status of 0-2.
• Must give written informed consent indicating their awareness of the investigational nature of this study and its potential hazards.
• Adequate renal and hepatic function (creatinine ≤ 150% of institutional upper limit of normal, total bilirubin ≤ 150% institutional upper limit of normal, AST ≤ 200% institutional upper limit of normal).
• Life expectancy of at least 12 weeks.
• Have not received any chemotherapy within 4 weeks of study enrollment and must have recovered from any treatment-related toxicities.
• Women of childbearing age must have a negative serum pregnancy test prior to initiating therapy.
• Sexually active women of childbearing potential must use effective birth control during the trial and for an appropriate period after the trial.
• Men must be willing to avoid fathering a new child while receiving therapy with azacitidine.
• ≥18 years, no upper age limit
• Individuals who are candidates for hematopoietic stem cell transplantation and who meet all other study criteria may participate in the study and receive intravenous azacitidine alone as a treatment prior to transplantation.

Exclusion Criteria

• Known CNS leukemia.
• Previously received Azacitidine (Vidaza®, Pharmion Corp., Boulder CO) or decitabine (Dacogen®, MGI Pharma Inc. Bloomington, MN).
• Known or suspected hypersensitivity to azacitidine or mannitol.
• Receiving any other investigational agents within 30 days of first dose of study drug.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, congestive heart failure of NYHA class 3 or 4, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situation that would limit compliance with study requirements.
• Known positive serology for HIV.
• Had radiotherapy within 14 days prior to study enrollment.
• Known presence of hepatic tumors.
• <18 years of age
• Exclude women who are pregnant or breast feeding.