在Allo-HSCT后使用表观遗传剂预防RUNX1::RUNXT1阳性高风险AML的预防性治疗
Wenwen Guo1,2, Haixiao Zhang1,2, Yawei Zheng1,2
1State Key Laboratory of Experimental Hematology, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, National Clinical Research Center for Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, 300020, China.
Annals of hematology
|June 22, 2024
概括
与预防性治疗相比,预防性表观遗传剂在移植后的高风险RUNX1::RUNXT1阳性急性髓性白血病患者中显著改善了无复发和整体存活率. 这种方法显示出更好的长期结果的希望.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 干细胞移植 干细胞移植
背景情况:
- 疾病复发是导致RUNX1::RUNXT1阳性急性髓性白血病 (AML) 治疗失败的主要原因,该病发生在全源造血干细胞移植 (allo-HSCT) 后.
- 目前的移植后维持策略,包括最小残留疾病 (MRD) 监测,还没有足够减少高复发率.
研究的目的:
- 评估预防性表观遗传剂在高风险AML患者的疗效和安全性,这些患者在alo-HSCT后具有RUNX1::RUNXT1融合基因.
- 为了比较预防疗法和在分子复发后启动的预防性治疗之间的结果.
主要方法:
- 一项前性研究涉及30名高风险RUNX1::RUNXT1阳性AML患者,他们在2019年1月至2023年7月期间接受预防性表观遗传疗法 (chidamide或AZA).
- 与34名高风险患者进行比较,这些患者接受了针对分子复发的预防性治疗.
- 评估两年无复发存活率 (RFS),整体存活率 (OS) 和复发率的累积发病率.
主要成果:
- 与预防治疗组相比,预防治疗组的两年RFS (82.82%对51.38%,P=0.014) 和OS (86.42%对56.16%,P=0.025) 显着更高.
- 预防组的两年累计复发发病率明显较低 (13.8%与36.40%,P=0.037).
- 预防性表观遗传药物治疗被很好地容忍.
结论:
- 预防性表观遗传药物治疗显示,在接受alo-HSCT的高风险RUNX1::RUNXT1阳性AML患者中,有可能改善长期预后.
- 移植后的早期预防性干预可能优于以MRD监测为指导的预防性治疗.
- 观察到的安全概况表明,这种方法被人很好地容忍.
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