Modified azacitidine plus cytarabine versus traditional cytarabine in patients with newly diagnosed acute myeloid leukaemia: a retrospective and propensity score matched analysis

Azacitidine (AZA) is a hypomethylating agent with well-known antileukaemic activity. The addition of AZA to intermediate-dose cytarabine (IDAra-c) plus idarubicin (Ida) or etoposide (Eto) during consolidation may improve outcomes in acute myeloid leukaemia (AML) patients and reduce side effects. We retrospectively evaluated whether the addition of AZA to IDAra-c plus Ida or Eto during consolidation could improve outcomes and reduce toxicity in newly diagnosed AML patients who achieved complete remission (CR) or partial remission (PR) after induction therapy. Propensity score matching was performed between patients in the AZA+ IDAra-c+ Ida/Eto group and patients who received IDAra-c as consolidation in the corresponding period at a 1:1 ratio according to age at diagnosis, sex, Eastern Cooperative Oncology Group performance status, European Leukaemia Net 2022 risk stratification and induction therapy classification. Forty-eight patients treated with AZA+IDAra-c+Ida/Eto were matched with 48 patients receiving IDAra-c alone. AZA+ IDAra-c+ Ida/Eto was associated with longer overall survival (OS, 89.58 vs. 53.49%, p <0.01) and event-free survival (EFS, 80.92 vs. 48.16%, p <0.01) at 40 months. Subgroup analysis revealed that patients who were ≤40 years old, who achieved MRD negativity after induction therapy, who were diagnosed with adverse risk or who carried methylation-associated mutations may have potentially improved survival when AZA+ IDAra-c+ Ida/Eto was chosen as consolidation treatment. Myelosuppression was present in both groups, but the affected cells differed. AZA+IDAra-c+Ida/Eto consolidation was associated with improved long-term survival and comparable toxicity versus IDAra-c alone in newly diagnosed AML patients.