急性髓系白血病（AML）的常规治疗以高复发率、严重毒性和低整体生存率为特征。因此，开发新的治疗策略对于改善AML患者的生存和生活质量至关重要。CD19定向嵌合抗原受体（CAR）T细胞免疫疗法在治疗B细胞急性淋巴样白血病和一些成熟B细胞淋巴瘤方面取得了极大的成功。然而，由于缺乏类似于CD19的髓系细胞表面靶点，CAR T细胞疗法目前无法用于AML治疗，因为目前已知的白血病细胞表面靶点也存在于健康造血干细胞及其子代中。此外，免疫抑制性肿瘤微环境对CAR-T细胞的抗肿瘤活性产生了阻尼效应。在这里，我们回顾了限制CAR T细胞疗法用于AML治疗的治疗挑战，并讨论了克服这些挑战的有希望的新策略。版权所有 © 2023 作者。由 Wolters Kluwer Health, Inc. 代表欧洲血液学协会出版。

Conventional therapies for acute myeloid leukemia (AML) are characterized by high rates of relapse, severe toxicities, and poor overall survival rates. Thus, the development of new therapeutic strategies is crucial for improving the survival and quality of life of AML patients. CD19-directed chimeric antigen receptor (CAR) T-cell immunotherapy has been extremely successful in the treatment of B-cell acute lymphoid leukemia and several mature B-cell lymphomas. However, the use of CAR T-cell therapy for AML is currently prevented due to the lack of a myeloid equivalent to CD19, as currently known cell surface targets on leukemic blasts are also expressed on healthy hematopoietic stem and progenitor cells as well as their progeny. In addition, the immunosuppressive tumor microenvironment has a dampening effect on the antitumor activity of CAR-T cells. Here, we review the therapeutic challenges limiting the use of CAR T-cell therapy for AML and discuss promising novel strategies to overcome them.Copyright © 2023 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Hematology Association.