Ther Adv Hematol. 2025 ;16 20406207251326802
Acute myeloid leukemia (AML), the most common type of leukemia in adults, is a highly heterogeneous and aggressive hematologic malignancy. Since the 20th century, the combination of cytosine arabinoside and anthracyclines has been the most common chemotherapy drug used to treat patients with AML. Although, new targeted medicines have emerged, such as midostaurin and gilteritinib targeting FMS-like tyrosine kinase 3 (FLT3), ivosidenib (isocitrate dehydrogenase 1 (IDH1) inhibitor) and enasidenib (IDH2 inhibitor) targeting IDH, and gemtuzumab ozogamicin targeting CD33, which have changed the treatment strategies of AML. But, until now, hematopoietic stem cell transplantation remains the best treatment option in most cases. However, treatment resistance and relapse are still the major consequences of disease progression in AML, highlighting the urgent need for novel therapeutic approaches. As an alternative, chimeric antigen receptor (CAR)-T cells are engineered T-cells developed as a breakthrough in cancer therapy in recent years, and explored and used in various tumor types. In particular, it has achieved remarkable efficacy in the field of relapsed and refractory B lymphocyte tumors. This review mainly summarizes and discusses the research progress and the clinical application of CAR-T cell immunotherapy in AML in recent years.
Keywords: acute myeloid leukemia; chimeric antigen receptor T cells; immunotherapy; target antigen