Retrospective cohort study demonstrates improved survival with midostaurin plus intensive chemotherapy in FLT3-mutated AML, supporting its frontline standard-of-care status.
Key Points
To evaluate real-world clinical outcomes and survival benefits of adding midostaurin to intensive chemotherapy in patients across the adult age spectrum with newly diagnosed FLT3-mutated acute myeloid leukemia.
Retrospective registry analysis of 1,658 adults aged 14–85 years with newly diagnosed FLT3-mutated acute myeloid leukemia across 129 PETHEMA registry centers.
Compared outcomes between patients receiving intensive chemotherapy plus midostaurin (n = 469) versus intensive chemotherapy alone (n = 1,189), including multivariable and 261-pair propensity score–matched analyses.
Intensive chemotherapy plus midostaurin yielded higher composite complete remission (81.4% vs. 71.7%; p < 0.001) and lower Day 30 mortality (2.1% vs. 7.1%; p < 0.001) compared to chemotherapy alone.
Midostaurin significantly extended median overall survival to 47.2 months versus 19.3 months with chemotherapy alone (HR 0.64; 95% CI, 0.53–0.76; p < 0.001; adjusted HR 0.73; p = 0.017).
In propensity score–matched pairs, midostaurin maintained significant event-free survival benefit (HR 0.77; p = 0.029) and trended toward improved overall survival (HR 0.77; p = 0.06), with consistent effects across FLT3 mutation types and allelic burdens.