Azacitidine for myelodysplastic patients aged > 65 years: a review of clinical efficacy.

INTRODUCTION: Therapeutic strategies for elderly patients affected by myelodysplastic syndromes (MDS) are scarce and only few patients have an advantage in performing allogeneic bone marrow transplant. AREAS COVERED: Primary endpoints for treatment of elderly MDS patients were not curative, but rather allowing to maintain a good quality of life through prolongation of overall survival. In this context, azacitidine showed to improve responses in this subset of patients compared to conventional established regimens, such as intensive or low-dose chemotherapy and best supportive care. Good safety profile of the drug was reported either when it was used inside or outside clinical trials. Improved quality of response was observed when the drug was administered beyond the first response, and it is now usually recommended to continue it at the same dose and schedule in responding patients. EXPERT OPINION: Evaluation of baseline prognostic factors and comorbidities may help to identify patients who can benefit from the prolonged administration of the drug. Real life data regarding efficacy and safety of azacitidine in MDS elderly patients are required in order to confirm the results of clinical trials.