Health-Related Quality of Life with Gilteritinib versus Placebo Post-Transplant for FLT3-ITD+ Acute Myeloid Leukemia.

BMT CTN 1506 was a phase III randomized trial comparing gilteritinib versus placebo after allogeneic HCT for FLT3-ITD-positive AML. The primary analysis comparing relapse-free survival (RFS) was not statistically significant, however, patients with detectable FLT3-ITD MRD peri-HCT had significantly longer RFS with gilteritinib. The aim of this analysis is to describe the effect of post-HCT gilteritinib versus placebo on health-related quality of life (HRQOL). HRQOL was measured using the Functional Assessment of Cancer Therapy (FACT)-BMT, FACT-Leukemia (-Leu), and EQ-5D-5L at post-HCT randomization, day 29, month 3, 6, 12, 18, 24, and/or end of therapy. HRQOL and clinically meaningful differences were summarized using descriptive statistics and compared using mixed model repeated measures to evaluate longitudinal change from baseline and stratified Cox model to evaluate time to improvement. Between 8/2017 and 7/2020, 356 patients were randomized. HRQOL completion rate was acceptable (>70%) across all time points and measures. There were no differences in FACT-BMT, FACT-Leu, or EQ-5D-5L scores at any time point between cohorts. There was an increase in scores over time, indicating improvement in HRQOL post-HCT. Clinically meaningful improvement and time to improvement in HRQOL was similar in both arms. Despite higher TEAEs with gilteritinib, response to the question of being "bothered by side effects of treatment" did not differ between groups. Subgroup analysis of MRD detectable and negative patients demonstrated no differences in HRQOL between arms. For FLT3-ITD+ AML patients undergoing HCT, gilteritinib maintenance was not associated with any difference in HRQOL or patient-reported impact of side effects. Trial Registration: NCT02997202.