急性髓性白血病（AML）是一种髓系造血细胞的侵袭性恶性肿瘤，其特征是未成熟的粒母细胞异常克隆增殖和造血功能受损。白血病细胞人群高度异质性。白血病干细胞（LSC）是带有干细胞特征和自我更新能力的重要白血病细胞亚群，促进难治性或复发性AML的发展。现在认为，LSC是在骨髓（BM）区域受选择性压力影响下从造血干细胞（HSC）或表型定向的细胞群中发展而来。外泌体是含有生物活性物质的细胞外囊泡，在稳态和病理情况下参与细胞间通讯和物质交换。多项研究报道了外泌体在LSC、白血病爆发和骨髓基质细胞之间介导分子交流的作用，促进LSC的维持和AML的进展。本综述简要描述了LSC转化过程和外泌体的生物合成，重点介绍了白血病细胞和BM基质的外泌体在LSC维持和AML进展中的作用。此外，我们还讨论了外泌体在诊所中作为生物标志物、治疗靶点和针对性药物传递体的潜在应用。 版权所有 © 2023 Elsevier Inc。

Acute myeloid leukemia (AML) is an aggressive malignancy of myeloid hematopoietic cells, which is characterized by the aberrant clonal proliferation of immature myeloblasts and compromised hematopoiesis. The leukemic cell population is strongly heterogeneous. Leukemic stem cells (LSCs) are an important leukemic cell subset with stemness characteristics and self-renewal ability, which contribute to the development of refractory or relapsed AML. It is now acknowledged that LSCs develop from hematopoietic stem cells (HSCs) or phenotypically directed cell populations with transcriptional stemness characteristics under selective pressure from the bone marrow (BM) niche. Exosomes are extracellular vesicles containing bioactive substances involved in intercellular communication and material exchange under steady state and pathological conditions. Several studies have reported that exosomes mediate molecular crosstalk between LSCs, leukemic blasts, and stromal cells in the BM niche, promoting LSC maintenance and AML progression. This review briefly describes the process of LSC transformation and the biogenesis of exosomes, highlighting the role of leukemic-cell- and BM-niche-derived exosomes in the maintenance of LSCs and AML progression. In addition, we discuss the potential application of exosomes in the clinic as biomarkers, therapeutic targets, and carriers for targeted drug delivery.Copyright © 2023 Elsevier Inc. All rights reserved.