BACKGROUND: After pharmaceutical benefits scheme approval of midostaurin for fms-like tyrosine kinase 3 (FLT3)-mutated acute myeloid leukaemia (AML) in 2018, the Australasian Leukaemia & Lymphoma Group (ALLG) proposed a consensus approach to AML induction with 7+3 chemotherapy (7 days of infusional cytarabine with three doses of anthracycline) to align with future clinical trial protocols.

AIMS: To determine the efficacy and safety of idarubicin-based 7+3 induction ± midostaurin (per ALLG recommendations) in a real-world, tertiary hospital setting.

METHODS: Data were prospectively collected for all patients assessed for front-line AML treatment. Disease risk and response assessments were defined by European LeukaemiaNet 2017 guidelines. Efficacy and safety endpoints included complete remission (CR) rates, composite CR rates, event-free survival (EFS), overall survival (OS), induction mortality, duration of cytopenias and intensive care unit (ICU) utilisation. Analysis was planned following completion of ≥50 inductions and 5-year aggregated experience.

RESULTS: Between 2018 and 2023, 58 patients (median age 49 years) received 7+3 induction with CR and induction mortality rates of 88% (95% confidence interval (95% CI): 77-95%) and 1.7% (95% CI: 0-9%) respectively. At a median of 24.6 months of follow-up, median OS was 17.6 months for adverse-risk versus not reached for non-adverse-risk patients (P = 0.03). FLT3-mutated patients demonstrated an 89% CR rate (95% CI: 67%-99%) with comparable 4-year EFS (65%) and OS (68%) to FLT3-wild-type patients. Safety across 58 induction and 139 consolidation cycles was acceptable, with a single death and a 21% ICU admission rate (95% CI: 11%-33%) during induction.

CONCLUSIONS: Idarubicin-based 7+3 induction with contemporary supportive care yields good safety and CR rates, including in midostaurin-treated FLT3-mutated patients. Survival outcomes for adverse-risk AML patients remain suboptimal.