Background Azacitidine is a drug used in the treatment of adult patients with myelodysplastic syndrome (MDS), chronic myelomonocytic leukaemia (CMML) and acute myeloid leukaemia (AML). The recommended starting dose for the first treatment cycle is 75 mg/m² of body surface area, injected subcutaneously, daily for 7 days, followed by a rest period of 21 days. The results of a preliminary survey conducted in leading Italian hospitals on the use of the drug revealed a major case of inappropriate prescribing.

Purpose To evaluate the efficacy in terms of Overall Survival (OS) of the main regimens used in hospitals involved in the study.

Materials and methods The project involved five hospitals. We included all patients treated with azacitidine from 01 May 2010 to 30 March 2012 with follow up of at least 1 year (30 March 2013). The following information was recorded from each patient: age, sex, indication, dosing regimen, number of cycles, patient’s status at 30 March 2013 (alive, dead or lost to follow-up). Survival was calculated using the Kaplan-Meier method.

Results 121 patients were treated (55.5% male, mean age 69.7 years ± 9.6 SD), of whom 59 had MDS, 12 had CMML and 46 had AML. The following treatment regimens were adopted: for 50% of the patients treatment was scheduled for a period of 5 consecutive days, for 47% there was an “on/off” treatment schedule with discontinuation of treatment (5 days of treatment, 2 of interruption and other 2/3 days treatment) and for 3% of the patients treatment was scheduled for a period of 7 consecutive days. The statistical analysis revealed a statistically insignificant difference in terms of OS between the two regimens mostly employed (p = 0.32).

Conclusions The small numbers of patients who in clinical practice receive azacitidine for 7 consecutive days does not allow us to determine whether the alternative schedules actually used are as effective as the standard treatment. Until efficacy data become available, it would be desirable to put in place pathways to guarantee patients standard treatment with azacitidine.

No conflict of interest.