QUIZARTINIB was associated with improved 1-year overall survival (OS) compared with midostaurin in adults with newly diagnosed FLT3 internal tandem duplication (ITD)-positive acute myeloid leukaemia (AML) receiving intensive chemotherapy (IC), according to a multicentre cohort study involving 215 patients. The findings suggested that quizartinib may offer improved survival outcomes alongside higher remission rates and fewer treatment-related complications.
The retrospective cohort study included adults with newly diagnosed FLT3-ITD-positive AML treated across 12 referral centres in the USA between January 2018 and May 2025. Of the 215 patients included, 127 received midostaurin alongside IC, while 88 received quizartinib.
At 1 year, OS was 93.0% among patients receiving quizartinib, compared with 78.0% in those receiving midostaurin. This difference remained significant following adjustment for baseline AML-specific characteristics and haematopoietic cell transplantation (HCT) (hazard ratio [HR]: 0.22; 95% confidence interval [CI]: 0.08–0.58; p=0.002).
Event-free survival (EFS) also favoured quizartinib, with 1-year rates of 77.0% versus 56.0% for midostaurin (HR: 0.43; 95% CI: 0.25–0.74; p=0.003).
Patients receiving quizartinib achieved higher rates of composite complete remission than those receiving midostaurin (85.2% versus 73.2%; p=0.03). However, FLT3 clearance following induction and HCT rates during first remission were comparable between treatment groups.
Differences were also observed in several safety and treatment-related outcomes. Midostaurin was associated with higher intensive care unit admission rates (15.0% versus 4.5%; p=0.02), discharge to rehabilitation facilities (7.1% versus 1.1%; p=0.04), and treatment interruptions (25.2% versus 11.4%; p=0.01).
The findings provide comparative evidence for two targeted treatment approaches in newly diagnosed FLT3-ITD-positive AML, a setting in which direct comparisons from randomised clinical trials have not been available.
However, differences in follow-up duration warrant consideration. Patients receiving quizartinib had a shorter median follow-up of 324 days, compared with 667 days for midostaurin, reflecting the more recent approval of quizartinib.
A sensitivity analysis restricted to patients treated from 2023 onwards demonstrated directionally consistent differences in OS and EFS, although these were not statistically significant.
Overall, the findings suggest that quizartinib alongside IC may be associated with improved 1-year survival, remission outcomes, and treatment tolerability compared with midostaurin. Further comparative investigation will be important to clarify these associations and establish their implications for treatment selection in patients with FLT3-ITD-positive AML.
Reference
Lee MH et al. Overall survival in adults with acute myeloid leukemia initiating quizartinib vs midostaurin with chemotherapy. JAMA Netw Open. 2026;9;(11):e2642122.
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