中期剂量移植后循环胺用于骨髓修复性HLA-形相同的骨髓移植
Mustafa A Hyder1, Dimana Dimitrova1, Ruby Sabina1
1Center for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD.
Blood advances
|February 5, 2025
概括
中期剂量移植后环胺 (ID-PTCy) 显示,在单独的骨髓移植中,对移植对宿主疾病 (GVHD) 的预防有前途. 这种降低剂量策略似乎有效,并且比标准的高剂量疗法提供了临床益处.
科学领域:
- 造血干细胞移植 造血干细胞移植
- 免疫学 免疫学 免疫学
- 在瘤学瘤学.
背景情况:
- 高剂量的移植后循环胺 (HD-PTCy) 是在全源造血细胞移植 (HCT) 中用于移植对宿主疾病 (GVHD) 预防的标准.
- 鼠类研究表明,中剂量PTCy (ID-PTCy) 与HD-PTCy相比,可以提供更好的GVHD控制.
研究的目的:
- 评估降低剂量PTCy疗法 (25 mg/kg/天在+3/+4日或仅在+4日) 在肌肉衰变性HLA-形骨髓HCT中的安全性和有效性.
- 将ID-PTCy的结果与历史的HD-PTCy数据进行比较.
主要方法:
- 预期的1/2期临床试验,涉及35名患者接受了骨髓损伤性HLA-合同样性HCT.
- 患者接受了PTCy,西洛和mycophenolate mofetil,并减少了PTCy的剂量.
- 在第一阶段评估了剂量限制性毒性 (III-IV级急性GVHD),第二阶段关注PTCy 25 mg/kg/天在+3/+4.4天.
主要成果:
- 在降低PTCy剂量时,没有观察到剂量限制性毒性 (III-IV级急性GVHD).
- 第二阶段的剂量 (ID-PTCy; n=23) 没有导致II-IV级急性GVHD.
- 2年累计发生率:慢性GVHD13%,非复发死亡率17%,复发22%. 两年生存结果:总生存率为61%,无疾病生存率为61%,无GVHD/无复发生存率为52%.
结论:
- ID-PTCy (25 mg/kg/day在+3/+4天) 是有效和安全的GVHD预防在HLA-haploidentical HCT.
- 与HD-PTCy相比,ID-PTCy显示出临床益处,包括更快的移植,改善T细胞复制和降低毒性.
- 需要进一步的研究来证实这些发现,并优化在不同的HCT环境中PTCy剂量.
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