Multinational randomized trial of gilterinib as post-transplant maintenance for FLT3-ITD AML

Allogeneic hematopoietic cell transplantation (HCT) improves outcomes for patients with FLT3-ITD AML. NCCN guidelines recommend post-HCT maintenance with FLT3 inhibitors to reduce the risk of relapse, but there is limited evidence to support this. A randomized trial at 122 centers in 16 countries compared post-HCT gilteritinib (FLT3 inhibitor) maintenance versus placebo among these patients. Although relapse-free survival was higher in the gilteritinib group, the difference was not statistically significant (HR 0.679; p=0.0518). However, 50.5% of participants had measurable residual disease (MRD) detectable pre- or post-HCT. Relapse-free survival (panel A) and overall survival (panel B) were significantly better among MRD-negative patients compared with MRD-positive patients, regardless of treatment arm. Among MRD-positive patients (panel C) but not among MRD-negative patients (panel D), gilteritinib versus placebo was associated with improved relapse-free survival. According to the authors, these data are among the first to support the use of MRD-based post-HCT therapy.