Real-World Outcomes of Midostaurin Plus Intensive Chemotherapy in FLT3-Mutated AML: The PETHRATIFY Study.
СтатьяНазвание и аннотация: Перевод в очереди
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Romero Riquelme Mónica Alejandra, Aspas Requena Gaspar, Lloret-Madrid Pilar, Gil Cristina, Aguiar Eliana, Tormo Mar, Del Bernal Del Castillo Teresa, Rodríguez Arbolí Eduardo, Serrano Josefina, Sánchez-García Joaquín, Rodríguez-Medina Carlos, Mariz José Mário, Ayala Rosa, Martínez-López Joaquín, Amigo María Luz, Vives Susana, Algarra Algarra Lorenzo, García Boyero Raimundo, Brioso Joana, Castaño Tamara, Bergua Burgués Juan M, Salamero Olga, Bass Maturana María Francisca, Colorado Mercedes, García Fortes María, Sayas Lloris María José, Vicente Belén Vidriales, Chillón Carmen, Pérez Santaolalla Esther, Madrigal María Dolores, Labrador Gómez Jorge, García Pérez María José, Casado Calderón Soledad, Pérez Encinas Manuel, Balerdi Malcorra Amaia, Rodríguez María Solé, Gómez García Lara María, Herrera Puente Pilar, Arce Fernández Olga, Barrios García Manuel, Noriega Víctor, Fernández Rosa, Mateos Mamen, Lacalzada Carolina, Almela Gallego Ágata, García Garay María Carmen, Couto Carmen, Pérez-Simón José Antonio, Barragán González Eva, Montesinos Pau
Аннотация
Mutations in FLT3 are present in approximately 30% of patients with AML. The addition of midostaurin (MIDO) to intensive chemotherapy (IC) became standard of care following the RATIFY trial, but comprehensive real-world data spanning the full adult age spectrum and including both FLT3-ITD and FLT3-TKD mutations remain limited. We retrospectively analyzed 1658 adults aged 14-85 years with newly diagnosed FLT3-mutated AML from 129 PETHEMA registry centers: 469 received IC + MIDO and 1189 IC alone. Composite complete remission was higher with IC + MIDO than IC (81.4% vs. 71.7%; p < 0.001) and Day 30 mortality was substantially lower (2.1% vs. 7.1%; p < 0.001). Median overall survival was 47.2 versus 19.3 months (HR 0.64; 95% CI, 0.53-0.76; p < 0.001), and the benefit was sustained after multivariable adjustment (HR 0.73; p = 0.017). In 261 propensity score-matched pairs, the effect was attenuated, remaining significant for event-free survival (HR 0.77; p = 0.029) and showing a nonsignificant trend for OS (HR 0.77; p = 0.06). Allogeneic hematopoietic stem cell transplantation in first remission was more frequent in the IC + MIDO cohort (48.9% vs. 41.3%; p = 0.023). Time-dependent analyses showed the largest MIDO effect among autologous and non-transplanted patients (OS HR 0.45; p = 0.138, and HR 0.69; p = 0.014, respectively). The benefit of MIDO was consistent irrespective of FLT3 mutation type, FLT3-ITD allelic burden, cytogenetic risk, and gender, while less improvement occurred among NPM1 wild type and secondary AML patients. This large real-world cohort confirms the survival benefit of MIDO plus IC across the full adult age spectrum, supporting its standard-of-care status in FLT3-mutated AML.