高风险MDS和CMML的低甲基化剂加上venetoclax作为移植的桥梁疗法:GESMD研究
Ines Zugasti1,2,3, Monica Lopez-Guerra1,4,5, Sandra Castaño-Díez1,2,4
1Hospital Clínic Barcelona, Barcelona, Spain.
Experimental hematology & oncology
|April 26, 2025
概括
低甲基化剂和venetoclax (HMA/VEN) 组合表明,它有望为患有高风险骨髓分裂性综合征 (HR-MDS) 和慢性骨髓单细胞白血病 (CMML) 的患者架起桥梁,以实现全源干细胞移植 (allo-SCT). 这种方法实现了高响应率,并在大多数患者中促进了alo-SCT.
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:
- 高风险骨髓发育综合征 (HR-MDS) 和慢性骨髓单细胞白血病 (CMML) 具有显著的治疗挑战,治疗选择有限.
- 同源干细胞移植 (allo-SCT) 是这些疾病的唯一潜在的治愈治疗方法.
- 目前使用低甲基化剂 (HMA) 的标准移植前疗法存在局限性,大约40%的患者由于疾病进展无法继续使用alo-SCT.
研究的目的:
- 评估将低甲基化剂与venetoclax (HMA/VEN) 结合在一起的疗效,作为HR-MDS或CMML患者与allo-SCT之间的桥梁策略.
- 确定在alo-SCT之前接受HMA/VEN治疗的患者的移植资格和结果.
主要方法:
- 对30名HR-MDS或CMML患者进行了回顾性分析,这些患者有资格接受allo-SCT并接受HMA/VEN治疗.
- 患者队列包括18名以前没有接受过治疗的个人和12名耐药或复发 (R/R) 疾病患者.
主要成果:
- 总体响应率 (ORR) 为90%,根据IWG 2023标准,复合完整响应率为77%.
- 显著的83%的患者接受了allo-SCT,其中76%接受了HMA/VEN作为桥梁治疗.
- 合并SCT后的一年和两年的生存率为75%,两年累计复发发病率为30.5%.
结论:
- 对于HR-MDS和CMML患者来说,HMA/VEN组合显示出作为有效的桥梁战略与allo-SCT之间的潜力.
- 这种组合疗法可以改善这种具有挑战性的患者群体的移植资格和结果.
关键词:
分配-SCTT 的情况.桥梁疗法是一种治疗方法.在CMMLML中,CMML是CMML.细胞还原性疗法 细胞还原性治疗在HMA/VENEN中使用.在 MDS MDS 中.在MDS/MPN中使用.在MRD中,我们可以使用MRD.分子后续检查.更多相关视频
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