Table 1.

Summary of clinically applicable FLT3 inhibitors for FLT3-mutated AML.

Patient eligibility	Disease status	Drug	Target mutated lesion	Representative trial	Usage	Benefit	Approval	In Korea
Intensive induction eligible	Newly diagnosed	Midostaurin	FLT3-ITD/TKD	RATIFY (Phase3) [7]	Combination with 7+3 induction and consolidation chemotherapy	Median OS (74.7 vs. 25.6 mo), P=0.009	FDA, EMA	Available
	Maintenance	Midostaurin	FLT3-ITD/TKD	RATIFY (Phase3) [7]	Maintain until relapse for up to 12 monthsas the extension of RATIFY trial		EMA	Not available
	Post-HCT maintenance	Sorafenib	FLT3-ITD	SORMAIN (Phase 2) [9]	Maintain untilrelapse for up to 24 months	2-year RFS (85 vs. 53%), P=0.002	Off-label	Not available
	Relapsed or refractory	Gilteritinib	FLT3-ITD/TKD	ADMIRAL (Phase 3)	Monotherapy	Median OS (9.3 vs. 5.6 mo), P<0.001	FDA, EMA	Available
Intensive induction ineligible	Newly diagnosed	Sorafenib	FLT3-ITD	NCT02196857 (Phase 2) and NCT01254890 Phase 1/2) [35]	Combination with azacitidine	Median OS (8.3 mo)	Off-label	Not available
	Relapsed or refractory	Sorafenib	FLT3-ITD	NCT01254890 [36]	Combination with azacitidine	Response rate: 46%	Off-label	Not available

Abbreviations: AML, acute myeloid leukemia; EMA, European Medicines Agency; FDA, Food and Drug Administration; FLT3, FMS-like tyrosine kinase 3; HCT, hematopoietic cell transplantation; ITD, internal tandem duplication; mo, months; OS, overall survival; RFS, relapse-free survival; TKD, tyrosine kinase domain.