Intensive Chemotherapy With or Without Midostaurin in Adults ≥ 60 Years Old With <scp>FLT3</scp> ‐Mutated <scp>AML</scp> : A <scp>FILO</scp> ‐ <scp>DATAML</scp> ‐ <scp>PETHEMA</scp> Real‐World Study

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.

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Journal
American Journal of Hematology
Published
2026-02-11
DOI
https://doi.org/10.1002/ajh.70233
Primary Topic
Acute Myeloid Leukemia Research
Type
article
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article

Intensive Chemotherapy With or Without Midostaurin in Adults ≥ 60 Years Old With FLT3 ‐Mutated AML : A FILO ‐ DATAML ‐ PETHEMA Real‐World Study

Laura Torres, David Martinez‐Cuadrón, Lauren Véronèse, Adrien Contejean et al.
American Journal of Hematology
Acute Myeloid Leukemia Research
article

Intensive Chemotherapy With or Without Midostaurin in Adults ≥ 60 Years Old With FLT3 ‐Mutated AML : A FILO ‐ DATAML ‐ PETHEMA Real‐World Study

Laura Torres, David Martinez‐Cuadrón, Lauren Véronèse, Adrien Contejean, Cristina Gil, C. Recher, E. Berard, Eduardo Rodríguez‐Arbolí, Maria Jose Sayas Lloris, Ariane Mineur, María García‐Fortes, Corentin Orvain, R Birsen, Josefina Serrano, P Dumas, Romain Guièze, Carlos Rodríguez‐Medina, María Luz Amigo, Lorenzo Algarra Algarra, Rebeca Rodríguez‐Veiga, Pierre Peterlin, Sarah Bertoli, Arnaud Pigneux, Mar Tormo, Celestine Simand, Juan Miguel Bergua‐Burgues, Pau Montesinos, Emmanuelle Tavernier, Martin Carré, Gaspar Aspas Requena
article en

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.

American Journal of Hematology
Université Toulouse III - Paul Sabatier (FR), Inserm (FR), Université Fédérale de Toulouse Midi-Pyrénées (FR), Université de Montpellier (FR), Université Paris Cité (FR), Centre Hospitalier Universitaire de Grenoble (FR), Centre Hospitalier Universitaire de Bordeaux (FR), Hospital Universitari i Politècnic La Fe (ES), Institut de Cancérologie Strasbourg (FR), Hospital General Universitario Morales Meseguer (ES), Hôpital Cochin (FR), Centre Hospitalier Universitaire de Montpellier (FR), Hospital Clínico Universitario de Valencia (ES), Maternité Port Royal (FR), Hospital Universitario de Gran Canaria Doctor Negrín (ES), Institut universitaire du cancer de Toulouse Oncopole (FR), Hospital San Pedro de Alcántara (ES), Centre Hospitalier Universitaire d'Angers (FR), Hospital Universitario Reina Sofía (ES), Hospital Universitario Doctor Peset (ES), Centre Hospitalier Universitaire de Clermont-Ferrand (FR), Instituto de Biomedicina de Sevilla (ES), Centre Hospitalier Annecy Genevois (FR), Hospital Universitario Central de Asturias (ES), Hospital General Universitario de Alicante Doctor Balmis (ES), Hospital General Universitario de Albacete (ES), Centre Hospitalier Universitaire de Saint-Étienne (FR), Hospital Clínico Universitario Virgen de la Victoria (ES), INCLIVA Health Research Institute (ES), Université d'Angers (FR), University of Córdoba (ES), Nantes Université (FR)
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