ABSTRACT The addition of midostaurin (MIDO) to intensive chemotherapy (IC) improves survival in younger adults with FLT3 ‐mutated acute myeloid leukemia (AML); however, real‐world data in elderly patients (≥ 60 years) are limited. This large, retrospective, multicenter study from three European registries (PETHEMA, FILO, DATAML) evaluated MIDO+IC ( n = 194) versus IC alone ( n = 371) in 565 patients with FLT3 ‐mutated AML aged ≥ 60 years (median age 67.5 years; 35.6% ≥ 70 years). MIDO+IC was associated with lower day‐60 early death (8.2% vs. 21.4%, p < 0.0001) and higher composite complete remission (CRc) rates (78.9% vs. 63.1%, p < 0.0001). After a median follow‐up of 46.0 months, median overall survival (OS) was 24.2 months for MIDO+IC versus 8.7 months for IC ( p < 0.0001), with 5‐year OS rates of 40.6% vs. 12.9%, respectively. Event‐free survival (EFS; median 13.5 vs. 4.6 months; 5‐year EFS: 36.0% vs. 10.1%) and relapse‐free survival (RFS; median 20.2 vs. 8.0 months; 5‐year RFS: 45.4% vs. 15.7%) were also significantly improved (both p < 0.0001). The 5‐year cumulative incidence of relapse was lower with MIDO+IC (47.8% vs. 67.1%, p < 0.001). In multivariate analyses, midostaurin was an independent favorable prognostic factor for CRc (aOR 1.97 95% CI: 1.29–2.98), OS (aHR 0.46 95% CI: 0.36–0.58), EFS (aHR 0.49 95% CI: 0.39–0.60), and RFS (aHR 0.47 CI: 0.36–0.62) (all p ≤ 0.002). These benefits were confirmed by propensity score matching. This large real‐world study demonstrates that combining midostaurin with IC significantly improves remission rates and survival outcomes in elderly patients with FLT3 ‐mutated AML, supporting its consideration in this population.
Requena et al. (Wed,) studied this question.