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    The results from the RATIFY trial ( NCT00651261; CALGB 10603) showed that midostaurin combined with standard chemotherapy significantly improved outcomes in patients with FMS-like tyrosine kinase 3 (FLT3)-mutated acute myeloid leukemia (AML), compared with placebo. In this post hoc subgroup analysis from the trial, we evaluated the impact of midostaurin in 163 patients with FLT3-tyrosine kinase domain (TKD) mutations. At a median follow-up of 60.7 months (95% CI, 55.0-70.8), the 5-year event-free survival (EFS) rate was significantly higher in patients treated with midostaurin than in those treated with placebo (45.2% vs 30.1%; P = .044). A trend toward improved disease-free survival was also observed with midostaurin (67.3% vs 53.4%; P = .089), whereas overall survival (OS) was similar in the 2 groups. Patients with AML and NPM1mut/FLT3-TKDmut or core binding factor (CBF)-rearranged/FLT3-TKDmut genotypes had significantly prolonged OS with or without censoring at hematopoietic cell transplantation (HCT), compared with NPM1WT/CBF-negative AMLs. The multivariable model for OS and EFS adjusted for allogeneic HCT in first complete remission as a time-dependent covariable, revealed NPM1 mutations and CBF rearrangements as significant favorable factors. These data show that NPM1 mutations or CBF rearrangements identify favorable prognostic groups in patients with FLT3-TKD AMLs, independent of other factors, also in the context of midostaurin treatment. © 2020 by The American Society of Hematology.


    Maria Teresa Voso, Richard A Larson, Dan Jones, Guido Marcucci, Thomas Prior, Jürgen Krauter, Michael Heuser, Serena Lavorgna, Josep Nomdedeu, Susan M Geyer, Alison Walker, Andrew H Wei, Jorge Sierra, Miguel A Sanz, Joseph M Brandwein, Theo M de Witte, Joop H Jansen, Dietger Niederwieser, Frederick R Appelbaum, Bruno C Medeiros, Martin S Tallman, Richard F Schlenk, Arnold Ganser, Sergio Amadori, Yuan Cheng, YinMiao Chen, Celine Pallaud, Ling Du, Alfonso Piciocchi, Gerhard Ehninger, John Byrd, Christian Thiede, Konstanze Döhner, Richard M Stone, Hartmut Döhner, Clara D Bloomfield, Francesco Lo-Coco. Midostaurin in patients with acute myeloid leukemia and FLT3-TKD mutations: a subanalysis from the RATIFY trial. Blood advances. 2020 Oct 13;4(19):4945-4954

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    PMID: 33049054

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