AML-266: Azacitidine or Decitabine Monotherapy for the Treatment of AML: A Comparative Systematic Review and Meta-Analysis

Abstract

<h3>Context:</h3> Decitabine and azacitidine have been increasingly used to treat patients with acute myeloid leukemia (AML) who are elderly or not suitable for intensive chemotherapy. Despite their widespread use, there is no consensus on their efficacy, with considerable variability between studies. <h3>Objective:</h3> Our aim was to analyze and compare the efficacy of azacitidine and decitabine for the treatment of AML in elderly patients and/or patients not suitable for intensive chemotherapy. <h3>Design:</h3> A systematic review and meta-analysis was conducted. <h3>Patients or Other Participants:</h3> Trials were selected if performed on patients not eligible for intensive chemotherapy. The meta-analysis only includes data from the azacitidine or decitabine monotherapy arms; data from experimental arms were excluded from the analysis. <h3>Interventions:</h3> The results were summarized using a point estimate and 95% confidence interval (CI) for the means of the different outcomes between studies. <h3>Main Outcome Measures:</h3> Studies had to report at least one of the following outcomes: mortality, overall survival (OS), complete remission, complete remission with incomplete hematological recovery, or partial response. <h3>Results:</h3> The search strategy revealed 681 citations before duplicates were removed. Finally, 20 articles were included after analysis of abstracts and full text. A total of 3,000 patients from 23 cohorts were analyzed (12 cohorts of azacitidine and 11 of decitabine). A trend toward lower response rates was observed for azacitidine (31%, 95%CI: 24%–37%) compared to decitabine (40%, 95%CI: 31%–49%o, p=0.081). However, there was no significant difference in OS between azacitidine (9.99 months, 95%CI: 8.17–11.82) and decitabine (8.88 months, 95%CI: 7.67–10.08, p=0.550). Lower 1-year mortality with standard 7-day 75 mg/m² azacytidine treatment was observed compared to other azacitidine schedules (51%, 95%CI: 46%-57% <i>vs</i> 72%, 95%CI: 61%-82%, p=0.001). Moreover, OS was lower when administered for 5 days (6.28 months, 95%CI: 4.23-8.32) <i>versus</i> 7 days (10.90 months, 95%CI: 8.92–12.89, p=0.002). However, there was no significant difference in response rates, mortality, and OS when comparing the 5-day <i>vs</i> 10-day decitabine regimens. <h3>Conclusions:</h3> There were no significant differences in 1-year mortality or OS for azacitidine and decitabine (roughly 9 months). These results exploring single-HMA regimens could be useful for the design and use of new HMA-based combination schedules.