非骨髓化的异构血造干细胞移植用于骨髓纤维化. 来自丹麦东部的一项基于人口的研究
Morten Orebo Holmström1, Lia Minculescu2, Katrine Nørgaard3
1National Center for Cancer Immune Therapy, Department of Oncology, Herlev-Gentofte Hospital, Herlev, Denmark.
Transplantation and cellular therapy
|March 19, 2025
概括
在所有原性干细胞移植用于骨髓纤维化之前,非骨髓衰变性调节是可行的,显示出低的非复发死亡率,但高的复发率. 患者的性别,捐赠者类型和并发症会影响整体存活率.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 移植医学 移植医学
背景情况:
- 针对骨髓纤维化 (MF) 的全源造血干细胞移植 (allo-HCT) 采用了骨髓可塑性调节 (MAC) 和减少强度调节 (RIC) 方案.
- 患有MAC的患者中位数年龄增加和高非复发性死亡率 (NRM) 导致更多地使用非骨髓缩 (NMA) 疗法.
- 在丹麦,NMA调节是MF的allo-HCT标准治疗方案.
研究的目的:
- 描述一组均的MF患者在NMA条件下接受allo-HCT的结果.
- 为了确定与这种队列中移植结果相关的因素.
主要方法:
- 从2007年到2023年,对67名多发性肌痛患者进行了回顾性队列研究,这些患者接受了NMA调节 (fludarabine和低剂量全身辐射) 治疗.
- 分析整体存活 (OS),NRM,复发累积发病率 (CIR) 和无进展存活 (PFS).
- 多变量分析以确定与OS,PFS和NRM相关的因素.
主要成果:
- 随访时间中位数为3.4年,58%的患者还活着.
- 一年的OS为77%,1年的NRM为15%,1年的CIR为24%.
- 男性性别,无血缘关系的捐赠者移植和高HCT-CI (>2) 与更糟糕的生存状况和更高的NRM有关.
结论:
- 用fludarabine和低剂量TBI进行NMA调节是一种可行的方法,用于MF患者的allo-HCT.
- 虽然NMA调节显示早期NRM低,但相对较高的早期复发率表明,应该考虑对特定患者进行差异化调节.
- 捐赠者与患者之间的关系,患者的并发症和患者的性别是OS的独立预测因素.
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