与HLA不匹配的无血缘捐赠干细胞移植相比,移植后的环胺改善了存活率
Jonathan Bordat1, Eleonore Kaphan1, Marie Robin1
1Department of Hematology and Bone Marrow Transplantation, Saint-Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris.
Haematologica
|August 28, 2025
概括
移植后循环胺 (PTCY) 改善了不匹配的无血缘捐赠者造血干细胞移植 (alloHSCT) 的结果. 与抗血球蛋白 (ATG) 相比,PTCY降低了非复发性死亡率 (NRM) 和与GVHD相关的死亡率,提高了整体存活率 (OS) 和无进展存活率 (PFS).
科学领域:
- 血液学
- 免疫学
- 癌症学
背景情况:
- 来自不匹配的非亲属捐赠者的全源造血干细胞移植 (alloHSCT) 存在重大风险,包括非复发性死亡率 (NRM) 和移植对宿主疾病 (GVHD).
- 抗血球蛋白 (ATG) 已成为GVHD预防的标准,但其在MMUD alloHSCT中的有效性和安全性仍然是研究领域.
- 移植后的环胺 (PTCY) 已成为各种alloHSCT中GVHD预防的有希望的替代品.
研究的目的:
- 为了比较移植后环胺 (PTCY) 与高剂量 (HD) 和低剂量 (LD) 抗血球蛋白 (ATG) 的疗效和安全性,用于移植对宿主疾病 (GVHD) 预防.
- 评估不同GVHD预防策略对MMUD alloHSCT患者的整体存活率 (OS),非复发性死亡率 (NRM) 和无进展存活率 (PFS) 的影响.
- 在治疗组中评估急性和慢性GVHD,感染和与GVHD相关的死亡率.
主要方法:
- 一个单一中心的回顾性研究,涉及155名不匹配的非相关捐赠者的血造干细胞移植 (MMUD alloHSCT).
- 患者接受了移植后的环胺 (PTCY,n=41) 或高剂量ATG (HD- ATG) 或低剂量ATG (LD- ATG) 的预防.
- 分析了包括整体存活率 (OS),非复发性死亡率 (NRM),无进展存活率 (PFS),急性和慢性移植对宿主疾病 (GVHD) 以及感染率在内的结果.
主要成果:
- 与高剂量ATG (HD- ATG) (56. 5%) 和低剂量ATG (LD- ATG) (64. 8%) 相比,移植后的环胺 (PTCY) 与显著改善的一年整体存活率 (OS) 相关.
- 与HD- ATG (24. 4%) 和LD- ATG (29. 8%) 相比,PTCY显著降低了一年无复发死亡率 (NRM).
- 虽然复发发病率相似,但PTCY显示了一年无进展生存率 (PFS) (78. 4%) 与HD- ATG (50. 0%) 和LD- ATG (54. 0%) 相比有所改善. GVHD发病率没有显著差异,但与GVHD相关的死亡率较低.
结论:
- 与高剂量和低剂量抗甲基球蛋白 (ATG) 相比,移植后环胺 (PTCY) 是一种优越的GVHD预防策略,用于不匹配的无血缘捐赠者血造干细胞移植 (MMUD alloHSCT).
- 通过大幅降低非复发性死亡率 (NRM) 和与GVHD相关的死亡率,PTCY显著改善了整体存活率 (OS) 和无进展性存活率 (PFS).
- 这些发现支持使用PTCY作为MMUDalloHSCT中首选的GVHD预防药物,可能是由于免疫复合和减少感染并发症的差异.
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