Acute myeloid leukemia (AML) is a type of cancer of the blood and bone marrow with excess immature leukocytes (WBC). In patients with refractory or relapsed AML with mutations in the FMS-like FTL3 gene, salvage chemotherapy hasn’t been effective. Gilteritinib is an oral, potent, and selective FLT3 inhibitor that is known to impede the activity of FTL3 in FTL3-mutated AML. This study aims to compare the effectiveness of chemotherapy and Gilteritinib in the treatment of relapsed or refractory FTL3-mutated AML.

This is a phase-3 clinical trial that included a total of 371 eligible patients. All the participants had relapsed or refractory FTL3-mutated AML. The patients were divided into two groups in a 2:1 ratio; the first group was assigned to gilteritinib (120 mg per day), and the other to salvage chemotherapy.

The median overall survival in the gilteritinib group (9.3 months) was significantly longer than the salvage chemotherapy group (5.6 months). The hazard ratio between the two groups was 0.68. The median event-free survival in the gilteritinib group was 2.8 months, compared to 0.7 months in the chemotherapy group.

The research concluded that gilteritinib significantly improved the median overall survival and event-free survival in patients with relapsed or refractory FLT3-mutated AML than salvage chemotherapy.

Ref: https://www.nejm.org/doi/full/10.1056/NEJMoa1902688