The hypomethylating agents
decitabine and
azacitidine have been found to improve the outcome of patients with
myelodysplastic syndrome (MDS); however, the clinical choice between them is controversial. Therefore, this meta-analysis was performed to compare the efficacy, toxicity, and survival advantage of
decitabine and
azacitidine in patients with MDS. Eleven trials with a total of 1392 patients with MDS (
decitabine, n = 768;
azacitidine, n = 624) were included for analysis. The pooled estimates of partial response, hematologic improvement, and overall response rates for
azacitidine were significantly higher than for
decitabine. There were no differences between these 2 drugs regarding complete response,
red blood cell transfusion-independent rates, and grade 3 or 4 hematologic toxicity. When compared with best supportive care,
azacitidine significantly improved overall survival (hazard ratio [HR], 0.69; 95% CI, 0.54-0.87) and time to
acute myeloid leukemia transformation (HR, 0.51; 95% CI, 0.35-0.74). But these benefits were not found with
decitabine. Among patients with higher risk (International Prognostic Scoring System value of 3) or older than 75 years, treatment with
azacitidine was a favorable factor, whereas
decitabine showed no advantage. Therefore, with higher overall response rates and better survival benefits,
azacitidine is recommended as the first-line hypomethylating agent for MDS, especially in elderly patients or those with high risk.