Long-term survival with oral azacitidine: An update from the QUAZAR AML-001 trial

The QUAZAR AML-001 trial has been extensively covered on our hub, with key results demonstrating that maintenance with oral azacitidine (AZA) improves overall survival (OS) and relapse-free survival (RFS) when compared with placebo in elderly patients with acute myeloid leukemia (AML) in first complete remission. At the European Hematology Association (EHA) 2022 Congress, Andrew Wei presented updated results for long-term survival with oral azacitidine from this trial,¹ which we are pleased to summarize here.

Methods

• Long-term survival was defined as surviving ≥3 years from randomization, with these patients grouped into a “long-term survival (LTS)” cohort
• In the non-LTS cohort, patients either died within 3 years from randomization, withdrew consent, or were lost during follow-up

Results

• Overall, 29.7% (140/472) of patients were included in the LTS cohort, including 34.9% (83/238) of patients receiving oral azacitidine, and 24.4% (57/234) of patients receiving placebo
• A total of 6.3% (15/238) of patients receiving oral azacitidine received allogeneic hematopoietic stem cell transplant (allo-HSCT)
  • Seven of these patients were in the non-LTS cohort, and eight were in the LTS cohort
  • In the placebo arm, 13.7% (n = 32) of patients received allo-HSCT after treatment discontinuation, and 16 of whom were in the LTS cohort

Multivariable analysis

• In a multivariable analysis controlling for all other covariates, oral azacitidine was significantly associated with improved LTS compared to placebo (p = 0.010)
  • Other variables significantly associated with LTS were measurable residual disease (MRD) negativity at baseline (p = 0.002), presence of the NPM1 mutation at diagnosis (p < 0.001), and intermediate versus poor cytogenetic risk at diagnosis (p = 0.007)

In summary, updated results from the QUAZAR AML-001 trial demonstrated that treatment with oral azacitidine led to improved LTS outcomes compared with placebo (34.9% vs 24.4%). Oral azacitidine was also significantly associated with improved LTS in a multivariable analysis.