CXCR4/CXCL12 轴调节细胞增殖、存活和分化，以及造血干细胞 (HSC) 从骨髓微环境到外周血的归巢和动员。此外，CXCR4 和 CXCL12 是血液恶性肿瘤与其微环境之间串扰的关键介质。 CXCR4 过表达会促进疾病进展、促进肿瘤细胞存活并促进化疗耐药，从而导致预后不良。根据这些发现，科学研究和临床试验强调了小分子拮抗剂（如普乐沙福、肽/肽模拟物）的治疗前景。例如 BKT140、单克隆抗体（例如 PF-06747143 和 ulocuplumab）以及 microRNA。它们在减轻肿瘤负荷、诱导细胞凋亡和提高恶性细胞对传统化疗的敏感性方面的功效是显而易见的。本概述深入探讨了 CXC4/CXCL12 轴在血液肿瘤中的致病作用，并研究了关键 CXCR4 拮抗剂的临床应用。这些信息共同强调了 CXCR4 拮抗剂作为血液恶性肿瘤治疗策略的潜力，展示了临床前和临床研究的进展。随着这些治疗策略在临床试验中取得进展，它们重塑血液恶性肿瘤预后的潜力将变得越来越明显。

CXCR4/CXCL12 axis regulates cell proliferation, survival, and differentiation, as well as the homing and mobilization of hematopoietic stem cells (HSCs) from bone marrow niches to the peripheral blood. Furthermore, CXCR4 and CXCL12 are key mediators of cross-talk between hematological malignancies and their microenvironments. CXCR4 overexpression drives disease progression, boosts tumor cell survival, and promotes chemoresistance, leading to poor prognosis.In light of these discoveries, scientific investigations, and clinical trials have underscored the therapeutic promise found in small-molecule antagonists like plerixafor, peptides/peptidomimetics, such as BKT140, monoclonal antibodies like PF-06747143 and ulocuplumab, as well as microRNAs. Their efficacy is evident in reducing tumor burden, inducing apoptosis and sensitizing malignant cells to conventional chemotherapies. This overview delves into the pathogenic role of the CXC4/CXCL12 axis in hematological neoplasms and examines the clinical application of key CXCR4 antagonists.The information collectively emphasizes the potential of CXCR4 antagonists as a therapeutic strategy for hematologic malignancies, showcasing advancements in preclinical and clinical studies. As these therapeutic strategies progress through clinical trials, their potential to reshape the prognosis of hematologic malignancies will become increasingly apparent.