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Gilteritinib
Source: Childhood Acute Myeloid Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 15, 2025 · Captured 2026-09-09.
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As in de novo AML, most of the focus and published experience with FLT3 inhibitors is in adults with AML and this applies to the relapsed and refractory setting as well. Gilteritinib is a type 1 selective FLT3 inhibitor with activity against both FLT3 variants (ITD and D835/I836 tyrosine kinase domain [TKD]). In relapsed or refractory AML, gilteritinib is the first and only FLT3 inhibitor that has received FDA approval for single-agent use in adults. The approval was based on the ADMIRAL (NCT02421939) trial.[31]
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The phase III ADMIRAL trial included adults (aged 18 years and older) with relapsed or refractory AML and FLT3 variants. In this study, 247 patients were randomly assigned to receive either single-agent gilteritinib (120 mg/day given once daily) or one of four salvage chemotherapy regimens.[31]
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The phase III ADMIRAL trial included adults (aged 18 years and older) with relapsed or refractory AML and FLT3 variants. In this study, 247 patients were randomly assigned to receive either single-agent gilteritinib (120 mg/day given once daily) or one of four salvage chemotherapy regimens.[31]
Median OS was significantly better in patients who received gilteritinib (9.3 months vs. 5.6 months; hazard ratio [HR], 0.64; 95% confidence interval [CI], 0.49–0.83; P < .001), with 37.1% versus 16.7% of patients alive at 1 year.
The phase III ADMIRAL trial included adults (aged 18 years and older) with relapsed or refractory AML and FLT3 variants. In this study, 247 patients were randomly assigned to receive either single-agent gilteritinib (120 mg/day given once daily) or one of four salvage chemotherapy regimens.[31]
Importantly, because HSCT is felt to be essential for long-term survival in patients with AML and FLT3 variants, a higher percentage of gilteritinib recipients underwent an HSCT (25.5% vs. 15.3%). It had equal efficacy in both FLT3 ITD and FLT3 TKD AML cohorts.
The phase III ADMIRAL trial included adults (aged 18 years and older) with relapsed or refractory AML and FLT3 variants. In this study, 247 patients were randomly assigned to receive either single-agent gilteritinib (120 mg/day given once daily) or one of four salvage chemotherapy regimens.[31]
There were fewer adverse events in patients who received gilteritinib than in patients who received salvage chemotherapy regimens. However, some patients who received gilteritinib had elevated hepatic transaminase levels. The main toxic effect was myelosuppression.
Gilteritinib is now being studied in children with FLT3-positive de novo AML in the COG AAML1831 (NCT04293562) trial.
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