A heterogeneous collection of clonal myeloid cancerous growths make up the myelodysplastic syndromes (MDS). It is distinguished by repetitive cytogenetic abnormalities, multilineage cytopenias, and the likelihood of acute myeloid leukemia (AML) progressing. Acute myeloid leukemia can happen from the beginning or it can be diagnosed secondary to myelodysplastic syndromes. The malignant clones of myeloid lineage in the bone marrow and peripheral blood, with circulation into the tissues helps distinguish AML. The U.S. has approved cytidine nucleoside analog and epigenetic modifier azacitidine to treat all American-British-French subtypes of myelodysplastic syndromes. As per the World Health Organization classification, several countries have also approved the two drugs to treat multilineage dysplasia and acute myeloid leukemia with 20% to 30% blasts.

The phase III clinical trial demonstrated the advantages of using azacitidine to treat patients with AML with more than 30% blast. The oral pills are likely to boost patients’ convenience, eradicate reactions in the injection sites, allow long-term treatment plans, and allow dosage and schedule to be altered according to convenience. Phase I demonstrated the effects of oral azacitidine (CC-486), clinical responses, and acceptability in patients with AML and MDS. 

A prolonged dosage schedule is presently being administered as frontline therapy for patients with lower-risk MDS. It is also being administered as maintenance therapy for patients with AML, who are not eligible for stem cell transplant. In addition, patients with AML or MDS following stem cell transplant are also receiving the said dosage schedule as maintenance therapy. The researchers are assessing the viability of the prolonged dosage schedule of oral azacitidine for a period of two weeks or three weeks on a 28-days cycle. Clinical data provided in the review supports injectable azacitidine in patients with AML and MDS. It also assessed the grounds for and results of developing oral azacitidine.