A Comprehensive Overview of the Role of Azacitidine in the Management of Myelodysplastic Syndromes and Acute Myeloid Leukaemia

Abstract

Myelodysplastic syndromes (MDS) are a heterogeneous group of haematological disorders marked by progressive cytopenias and risk of transformation to acute myeloid leukaemia (AML). Best supportive care (BSC) remains the mainstay of therapy. The pyrimidine analogue azacitidine was the first drug to demonstrate that a change in the natural history of MDS is possible and was the first chemotherapeutic agent approved by the US Food and Drug Administration for the treatment of MDS. Azacitidine, a hypomethylating agent, has shown to reduce proliferation and induce apoptosis of dysplastic cells. The Cancer and Leukemia Group B (CALGB) 9221 and AZA-001 trials demonstrated superiority of azacitidine over BSC and conventional care regimen, respectively. Various studies have demonstrated that azacitidine has better tolerability with comparable efficacy in terms of conventional therapy in older AML patients. Azacitidine is well tolerated even in the elderly patients. Cytopenias were the most commonly occurring haematological adverse events. Gastrointestinal adverse events (AEs) (e.g. nausea, vomiting and diarrhoea) and injection-site reactions were the most commonly occurring nonhaematological AEs. The review summarises the current role of azacitidine in the management of MDS and AML.