Gilteritinib as post-transplant maintenance for acute myeloid leukemia with FLT3 internal tandem repeat mutation.

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In this randomized clinical trial, patients with acute myeloid leukemia (AML) who had FLT3 ITD mutations in first remission after stem cell transplantation were randomly assigned to receive maintenance therapy with placebo or gilteritinib for 24 months. Although gilteritinib showed an improvement in relapse-free survival (primary endpoint), the difference did not reach statistical significance compared with the placebo group. However, in a prespecified subgroup analysis, gilteritinib showed clear benefits in patients with measurable residual disease (MRD) around the time of transplant. In contrast, patients who tested negative for MRD did not obtain a significant benefit from gilteritinib.

These results suggest that maintenance treatment with gilteritinib for 24 months may improve relapse-free survival in chronic myeloid leukemia patients who have FLT3 ITD mutations and are MRD positive around the time of transplantation.



Allogeneic hematopoietic cell transplantation (HCT) improves outcomes for patients with acute myeloid leukemia (AML) who have FLT3 internal tandem duplication mutation (FLT3-ITD) AML. These patients are routinely treated with an FLT3 inhibitor after HCC, but there is limited evidence to support this. Therefore, we conducted a randomized clinical trial of post-HCT maintenance with the FLT3 inhibitor gilteritinib ( Identifier: NCT02997202) to determine whether all these patients benefit or whether detection of measurable residual disease (MRD) can identify those who could benefit. .


Adults with FLT3-ITD AML in first remission underwent HSCT and were randomized to placebo or gilteritinib 120 mg once daily for 24 months after HSCT. The primary endpoint was relapse-free survival (RFS). Secondary endpoints included overall survival (OS) and the effect of pre- and post-HSCT MRD on RFS and OS.

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Three hundred fifty-six participants were randomized after HSCT to receive gilteritinib or placebo. Although relapse-free survival was higher in the gilteritinib group, the difference was not statistically significant (hazard ratio [HR]0.679 [IC del 95%, 0.459 a 1.005]; Two-sided P = .0518). However, 50.5% of participants had detectable MRD before or after HSCT, and in a prespecified subgroup analysis, gilteritinib was beneficial in this population (HR, 0.515). [IC del 95%, 0.316 a 0.838]; P = .0065). Those with no detectable MRD showed no benefit (HR, 1.213.1). [IC del 95%, 0.616 a 2.387]; F = .575).


Although the overall improvement in relapse-free survival was not statistically significant, relapse-free survival was greater for participants with detectable FLT3-ITD MRD before or after HSCT who received treatment with gilteritinib. To our knowledge, these data are among the first to support the efficacy of MRD-based post-HSCT therapy.

Source: Biopress

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