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The phase randomized III RATIFY trial (NCT00651261) assessed the effect of midostaurin, a multi-targeted tyrosine kinase inhibitor, in combination with standard induction and consolidation chemotherapy in Fms-Like Tyrosine Kinase 3 (FLT3)-mutated Acute Myeloid Leukemia (AML) patients. In this study, 717 FLT3 mutated AML patients were randomly assigned to receive either placebo or midostaurin 50 mg orally twice daily on Days 8–21 of each cycle of induction and consolidation chemotherapy followed by continuous daily midostaurin for up to 12 cycles. Published data from this study showed that midostaurin in combination with standard induction and consolidation therapy prolonged the Overall Survival (OS) of newly diagnosed FLT3-mutated AML patients.1 More results of this study were reported on the AML Global Portal (AGP) in July 2017.
At the 59th American Society of Hematology (ASH) Annual Meeting, Atlanta, GA, Konstanze Döhner, MD, from the University Hospital of Ulm, Ulm, Germany, presented data from a post-hoc analysis, which aimed to evaluate the clinical impact in a post-hoc analysis of Nucleophosmin 1 (NPM1)/ FLT3- Internal Tandem Duplication (FLT3-ITD) genotypes as defined by the 2017 European LeukemiaNet (ELN) recommendations, in patients treated within the phase III randomized RATIFY trial (NCT00651261). The post-hoc analysis also aimed to evaluate the potential impact of midostaurin in distinct NPM1/FLT3-ITD genotypes.2
In this post-hoc analysis, 428 patients (median age = 47 years) of 717 patients enrolled in the RATIFY study with biomarker analysis were categorized into one of the four ELN NPM1/FLT3 subgroup including NPM1mut/FLT3-ITDlow (n = 85), NPM1mut/FLT3-ITDhigh (n = 159), NPM1wt/FLT3-ITDlow (n = 75) and NPM1wt/FLT3-ITDhigh (n = 109). The median follow-up time was 59 months (range, 42–81)
Konstanze Döhner concluded by noting that the findings of this post-hoc analysis suggests a “high prognostic value of the NPM1/FLT3-ITD genotypes” which provides support for the 2017 ELN risk stratification that includes FLT3-ITD allelic burden.
Furthermore, an advantageous effect for midostaurin was more prominent in patients with NPM1wt/FLT3-ITDhigh genotype although these patients might not benefit from allo-HCT.
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