Effectiveness and safety of 5-azacitidine in three patients with myelodysplastic syndromes

Abstract

Objective

To assess the effectiveness and safety of using 5-azacitidine to treat myelodysplastic syndromes.

Methods

Review of medical records of patients who received 5-azacitidine 75 mg/m² subcutaneously for during 7 days every 28 days in twelve cycles as compassionate use. We evaluated the objective response, clinical improvement and time to disease progression. We recorded adverse reactions described in the medical history.

Results

Six patients were candidates for treatment with 5-azacitidine. Three cases were evaluated over the study period. Most remained in partial response or better after the study, and no longer needed transfusions. In one patient, the treatment appeared to delay progression to leukaemia.

Conclusions

5-Azacitidine might be considered an effective and relatively safe drug, and may have contributed to controlling peripheral cytopenias, improving the quality of life and delaying progression to leukaemia. Additional studies with more patients are needed to support these results.