2025 Volume 14 Issue 4 Pages 214-223
Acute myeloid leukemia (AML) with FLT3-ITD mutation is characterized by a high tumor burden due to increased leukemia cell proliferation driven by the constant activation of FLT3 signaling. FLT3-ITD mutation-positive AML was previously categorized as high-risk AML due to its high relapse rate with conventional chemotherapy. However, with the advent of FLT3 inhibitors, the prognosis has improved significantly, and FLT3-ITD mutation-positive AML is now classified as intermediate-risk. In particular, the combination of FLT3 inhibitors and allogeneic hematopoietic cell transplantation has demonstrated a high cure rate, suggesting a synergistic effect between FLT3 inhibitors and graft-versus-leukemia (GVL) effects. This article will discuss basic findings on the enhancement of GVL effects by FLT3 inhibitors and will outline clinical data regarding their role as maintenance therapy following allogeneic hematopoietic cell transplantation in clinical practice.