获得性再生障碍性贫血(AA) 是一种骨髓功能衰竭的自身免疫性疾病，由异常激活的T细胞介导，表现为造血干细胞及外周血细胞严重耗竭。由于造血干细胞移植的供体限制，免疫抑制治疗(IST) 成为目前有效的一线治疗。但是，相当比例的AA患者不适合IST治疗，而且可能出现复发和其他血液恶性肿瘤，比如IST后的急性髓性白血病。因此，阐明AA的致病机制，并确定可治疗的分子靶点，是改善这些结果的有吸引力的途径。在本文中，我们总结了AA的免疫相关病因、药理学靶点，以及目前主流免疫抑制剂的临床效应。这为多靶点免疫抑制药物的组合以及基于当前干预途径发现新的靶点药物提供了新思路。版权所有 © 2023 Elsevier B.V.

Acquired aplastic anemia (AA) is an autoimmune disease of bone marrow failure mediated by abnormally activated T cells, manifested by severe depletion of hematopoietic stem and progenitor cells (HSPCs) and peripheral blood cells. Due to the limitation of donors for hematopoietic stem cell transplantation, immunosuppressive therapy (IST) is currently an effective first-line treatment. However, a significant proportion of AA patients remain ineligible for IST, relapse, and develop other hematologic malignancies, such as acute myeloid leukemia after IST. Therefore, it is important to elucidate the pathogenic mechanisms of AA and to identify treatable molecular targets, which is an attractive way to improve these outcomes. In this review, we summarize the immune-related pathogenesis of AA, pharmacological targets, and clinical effects of the current mainstream immunosuppressive agents. It provides new insight into the combination of immunosuppressive drugs with multiple targets, as well as the discovery of new druggable targets based on current intervention pathways.Copyright © 2023 Elsevier B.V. All rights reserved.