患有骨髓发育相关基因突变的急性髓性白血病患者可以从移植后的低甲基化剂维持中受益
Jiayu Huang1, Yi Xia2, Mengqi Xiang3
1State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, National Research Center for Translational Medicine, Shanghai Rui Jin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Transplantation and cellular therapy
|December 21, 2025
概括
在全源干细胞移植后维持低甲基化剂 (HMA) 改善了具有不良风险的急性髓性白血病 (AML) 患者的生存率. 这种策略对患有骨髓质疏松症相关基因突变的人特别有益,但对无法检测到的MRD缓解期的患者可能不需要.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 移植免疫学 移植免疫学
背景情况:
- 复发是异性造血干细胞移植 (allo-HSCT) 后具有不良风险的急性髓性白血病 (AML) 死亡的主要原因.
- 低甲基化剂 (HMA) 维持治疗在预防移植后复发的有效性,特别是在特定的遗传亚组内,仍在争论中.
研究的目的:
- 评估HMA维护在接受allo-HSCT的具有不良风险的AML患者的有效性.
- 确定从HMA维持疗法中受益最多的特定患者子组.
主要方法:
- 在2017年1月至2023年4月期间接受allo-HSCT的469名连续不良风险AML患者的回顾性多中心分析.
- 在HMA维持治疗组 (n=136) 和非维持治疗组 (n=333) 之间的结果比较.
- 分析包括移植结果,复发的累积发病率,无事件生存 (EFS),无复发生存 (RFS) 和整体生存 (OS).
主要成果:
- 维持HMA显著降低了3年的复发累积发病率 (19.2%对34.0%,P=0.026).
- 维护HMA改善了3年的EFS (68.6%与43.4%,P<0.001),RFS (69.1%与48.6%,P=0.001) 和OS (72.1%与61.0%,P=0.034).
- 观察到最大的益处在AML患者与骨髓质疏松症相关的基因突变;在CR1中,患有无法检测的MRD的患者可能不需要HMA维护.
结论:
- 移植后的HMA维护是一种有效的策略,可以改善选择的不良风险AML患者的治疗结果.
- 鉴定特定的基因子组,特别是那些与骨髓质疏松症相关的突变,可以指导HMA维护决策.
- 进一步的研究可能会根据可测量的残留疾病状态和缓解质量来完善HMA维持策略.
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