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Outcomes with single-agent gilteritinib for relapsed or refractory FLT3-mutant AML after contemporary induction
Jad Othman1,2,3, Angela Hwang4, Maximillian Brodermann4
1Department of Medical and Molecular Genetics, King's College London, London, United Kingdom.
Blood Advances
|September 12, 2024
Summary
Gilteritinib shows real-world effectiveness in FLT3-mutated AML, but outcomes remain suboptimal. Further research is needed for improved treatment strategies in relapsed or refractory acute myeloid leukemia.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Gilteritinib is a standard treatment for relapsed/refractory FLT3-mutated AML.
- Outcomes after contemporary first-line therapies and gilteritinib toxicity data are limited.
Purpose of the Study:
- To evaluate the real-world effectiveness, toxicity, and healthcare resource use of gilteritinib in FLT3-mutated AML patients.
Main Methods:
- A large, real-world cohort of 152 patients receiving single-agent gilteritinib across 38 UK hospitals was analyzed.
- Data on patient demographics, prior therapies, treatment cycles, hospitalizations, transfusions, remission rates, and survival were collected.
Main Results:
- Median age was 61; 36% had received ≥2 prior lines of therapy. 56% required hospitalization in the first cycle, and over half needed transfusions in the first 4 cycles.
- Complete remission (CR) or CR with incomplete recovery (CRi) was achieved in 30% of patients. Day-60 mortality was 10.6%, with a median overall survival of 9.5 months.
- Outcomes were poorer in patients with FLT3-tyrosine kinase domain mutations or adverse karyotype. Patients treated after venetoclax had a median survival of 4.5 months.
Conclusions:
- Real-world gilteritinib use in FLT3-mutated AML mirrors clinical trial findings but indicates suboptimal outcomes.
- More effective therapeutic strategies are necessary for patients relapsing after first-line treatments, especially those progressing on venetoclax.

