Cao Junjie, Zhuang Xianxu, Luo Danjie, Pei Renzhi, Lu Ying, Chen Dong, Li Shuangyue, Du Xiaohong, Liu Xuhui
Department of Hematology, The Affiliated People's Hospital of Ningbo University, Ningbo, China.
Institute of Hematology, Ningbo University, Ningbo, China.
Acta Haematol. 2024 Oct 25:1-10. doi: 10.1159/000542034.
The combination of venetoclax (VEN) and azacytidine (AZA) has demonstrated potential in achieving rapid and effective remissions in elderly patients with acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation is a promising potential cure for high-risk AML, as VEN-based therapies have a worse prognosis in elderly patients. This study aimed to assess the efficacy of sequential haploidentical HSCT following two courses of VEN and AZA therapy in patients with AML aged 55 years and older.
We conducted a retrospective study on AML patients aged 55-70 years who received intensive chemotherapy or two courses of VEN/AZA therapy, followed by haploidentical allo-HSCT (haplo-HSCT) based on disease risk degree, measurable residual disease status, and patient's preference.
Between January 2019 and December 2023, 141 newly diagnosed AML patients received initial treatment with intensive chemotherapy or VEN/AZA therapy. Among them, 64 patients received haplo-HSCT, while 77 did not. The 1-year overall survival (OS) and relapse-free survival (RFS) of patients who received haplo-HSCT were significantly higher than those who did not receive haplo-HSCT (p < 0.05). Among patients who received transplantation, there was no significant difference in 1-year OS and RFS between the VEN/AZA and intensive chemotherapy groups: 76.3% versus 69.3% (p = 0.367) for OS, and 74.5% versus 69.7% (p = 0.473) for RFS. High-risk ELN stratification and the presence of ≥4 gene mutations were associated with lower OS and RFS in both univariate and multivariate analyses.
AML patients over 55 years of age who received haplo-HSCT after two courses of VEN/AZA therapy had outcomes similar to those who received haplo-HSCT after intensive chemotherapy, suggesting that two courses of VEN/AZA therapy as a bridge to haplo-HSCT are feasible for patients over 55 years old.
维奈克拉（VEN）与阿扎胞苷（AZA）联合使用已显示出在老年急性髓系白血病（AML）患者中实现快速有效缓解的潜力。异基因造血干细胞移植是高危AML有前景的潜在治愈方法，因为基于VEN的疗法在老年患者中预后较差。本研究旨在评估在年龄≥55岁的AML患者中，接受两个疗程的VEN和AZA治疗后序贯单倍体同基因造血干细胞移植（HSCT）的疗效。
我们对年龄在55 - 70岁的AML患者进行了一项回顾性研究，这些患者接受了强化化疗或两个疗程的VEN/AZA治疗，随后根据疾病风险程度、可测量残留疾病状态和患者偏好进行单倍体同基因异基因造血干细胞移植（haplo-HSCT）。
在2019年1月至2023年12月期间，141例新诊断的AML患者接受了强化化疗或VEN/AZA治疗作为初始治疗。其中，64例患者接受了haplo-HSCT，而77例未接受。接受haplo-HSCT的患者的1年总生存率（OS）和无复发生存率（RFS）显著高于未接受haplo-HSCT的患者（p < 0.05）。在接受移植的患者中，VEN/AZA组和强化化疗组的1年OS和RFS无显著差异：OS分别为76.3%和69.3%（p = 0.367），RFS分别为74.5%和69.7%（p = 0.473）。在单因素和多因素分析中，高危欧洲白血病网络（ELN）分层以及存在≥4个基因突变均与较低的OS和RFS相关。
年龄≥55岁的AML患者在接受两个疗程的VEN/AZA治疗后接受haplo-HSCT与在接受强化化疗后接受haplo-HSCT的患者预后相似，这表明两个疗程的VEN/AZA治疗作为haplo-HSCT的桥梁对55岁以上患者是可行的。
