Multicenter phase 1b study of venetoclax plus gilteritinib for FLT3-mutated relapsed or refractory acute myeloid leukemia

The FLT3 inhibitor gilteritinib is standard therapy for relapsed or refractory (R/R) AML with FLT3 mutation but seldom induces sustained efficacy. Gilteritinib combines synergistically with the BCL-2 inhibitor venetoclax in preclinical models of FLT3-mutated AML. A multicenter phase 1b study in the USA found that the combination of gilteritinib and venetoclax for R/R FLT3-mutated AML is a highly active and tolerable oral combination regimen that potentially improves response frequency and depths over existing standards in a high-risk, mutation-defined disease.