Patients with newly diagnosed FLT3-mutated acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy often experience resistance to the current standard-of-care treatment, azacitidine plus venetoclax. In a Phase II trial led by Nicholas Short, M.D., the addition of gilteritinib, an oral FLT3 inhibitor, to the standard regimen resulted in high complete remission rates, deep FLT3 molecular responses and encouraging survival in trial participants.
The trial enrolled 30 patients with newly diagnosed FLT3-mutated AML, who received 80 milligrams of gilteritinib once daily in combination with azacitidine and venetoclax. All patients responded to the regimen, and the relapse-free survival and overall survival rates at 18 months were 71% and 72%, respectively. These results compare favorably to historical expectations with the current standard of care, highlighting the combination’s therapeutic potential in this population.