Efficacy and Safety of a 5-Day Regimen of Azacitidine for Patients with High-Risk Myelodysplastic Syndromes.

Although a 7-day (d) regimen of azacitidine (AZA) is the standard treatment of high-risk myelodysplastic syndromes (MDS), AZA is difficult to administer during weekends in an outpatient setting. We retrospectively investigated the outcome of a 5-d regimen of AZA in patients with high-risk MDS. High-risk MDS was defined as MDS with intermediate-2- or high-risk MDS according to the International Prognostic Scoring System. Every months AZA was given at 75 mg/m(2) per day for 5-7 d in hospital for first cycle and 5 d in outpatient for second cycle and later. Between April 2011 and December 2013, AZA treatment was initiated in 25 patients (men, 22; women, 3; median age, 75 yr; age range, 59-86 yr). The median number of AZA cycles was 10 (range, 1-24). Twenty patients received more than three cycles of AZA and 13 (52%) achieved any hematological improvement (HI). The median time to first response was two cycles (1-3). The most common non-hematological adverse events were neutropenia in 21 patients and thrombocytopenia in 17 patients. Nineteen patients died. The main cause of death was disease progression (five patients) and infectious complications (11 patients). The median overall survival was 13.2 months. The 5-d AZA regimen showed a good continuation rate of more than three cycles and an equivalent HI with the 7-d regimen.