IC ± midostaurin in adults aged ≥60 years with ND FLT3-mutated AML: A real-world study

Results from a multicenter registry analysis, evaluating real-world outcomes of intensive chemotherapy (IC) with or without midostaurin (Mido) in adults aged ≥60 years with newly diagnosed (ND) FLT3-mutated acute myeloid leukemia (AML; N = 565), were published in the American Journal of Hematology by Requena et al. Patients were identified from three registries – the Spanish Programa Español de Tratamientos en Hematología (PETHEMA) registry (NCT02607059), the French Innovative Leukemia Organization (FILO) registry, and the French Toulouse-Bordeaux (DATAML) registry – between January 1, 2005, and August 31, 2023.

Key data: With a median follow-up of 46.0 months, median overall survival (OS) was 24.2 months with IC + Mido (n = 194) vs 8.7 months with IC alone (n = 371; p < 0.0001); 5‑year OS rates were 40.6% vs 12.9%, respectively. Median event-free survival (EFS) was also superior with IC + Mido (13.5 vs 4.6 months; p < 0.0001); a similar benefit was seen for median relapse-free survival (RFS; 20.2 vs 8.0 months; p < 0.0001). The 5‑year cumulative incidence of relapse (CIR) was 47.8% with IC + Mido vs 67.1% with IC alone (p < 0.001). In multivariate analyses, Mido was independently associated with favorable composite complete remission (CRc; adjusted odds ratio [aOR], 1.97), OS (adjusted hazard ratio [aHR], 0.46), EFS (aHR, 0.49), and RFS (aHR, 0.47; all p ≤ 0.002); these benefits were confirmed by propensity score matching (PSM).

Key learning: In this large registry analysis, the addition of Mido to IC was associated with improved remission and survival outcomes vs IC alone in adults aged ≥60 years with ND FLT3-mutated AML, supporting its potential role in this setting.