Background: Although originally developed as a cytarabine analog more than 40 years ago, azacitidine has been the subject of renewed interest in the era of cancer epigenetics.
Objective: What is the history of the clinical development of azacitidine and how has it been applied successfully to the treatment of myelodysplastic syndromes (MDS)?
Methods: We review the evolution of the use of azacitidine for the therapy of human disease and review the major studies that have laid the groundwork for its current clinical indication.
Conclusion: The use of azacitidine has changed the approach to the treatment of MDS and has resulted in improved outcomes for patients.