The efficacy and safety of
azacitidine in elderly patients (aged >65 years) with
myelodysplastic syndromes (MDS) treated outside clinical trials are reported. Thirty-eight patients with MDS received
azacitidine (75 mg/m(2), schedule 5+2 +2): seven patients were classified as having refractory
cytopenia with multilineage dysplasia (RCMD), nine patients with
refractory anemia with excess of blasts (
RAEB) type 1, 18 patients with
RAEB type 2 and four patients with
chronic myelomonocytic leukemia type 2 (CMML-2). According to International Working Group (IWG) 2006 criteria, after the first four cycles we detected complete remission in seven patients (CR, 18%), improvement of bone marrow dysplasia and reduction of blast percentage in seven patients (partial response, 18%), stable disease in 20 patients (53%) and progression to acute
leukemia in four patients (10%). Median overall survival for all patients treated was 16.4 months. Only mild non-hematologic toxicity was detected (grade 1-2
nausea and
pruritus), whereas 55% of patients experienced hematologic side effects (25% grade 3-4
thrombocytopenia and 30% grade 3-4
neutropenia). Our results suggest that advanced age should not preclude effective treatment with
azacitidine in non-selected elderly patients wih MDS.