对于治疗不耐药的克罗恩病患者进行全基性骨髓移植
George B McDonald1,2, Ole J B Landsverk3, Dermot P B McGovern4
1Clinical Research Division, Fred Hutchinson Cancer Center, Seattle, WA, USA.
Heliyon
|January 29, 2024
概括
降低强度调节和异质骨髓移植对耐火性克罗恩病 (CD) 有希望. 一名患者在肠道粘膜中确立了捐赠者的免疫嵌合体后实现了持久的缓解.
科学领域:
- 免疫学 免疫学 免疫学
- 胃肠病学 胃肠病学
- 血液学 血液学 血液学
背景情况:
- 经过骨髓修复和异构骨髓移植后,在克罗恩病 (CD) 中观察到持久的缓解.
- 治疗不耐用的CD是一个重大挑战,需要采用替代治疗方法.
研究的目的:
- 评估降低强度调节和全原骨髓移植对治疗耐药克罗恩病的疗效.
- 为了尽量减少与移植相关的毒性和移植与宿主疾病 (GVHD) 的风险,使用低多基因风险评分的捐赠者和长时间的免疫抑制.
主要方法:
- 患者和HLA匹配的非相关捐赠者的基因定型,以计算多基因风险评分.
- 在非骨髓衰变条件下输注供体骨髓.
- 移植后对患者症状和内镜检测结果的监测.
主要成果:
- 两名患者接受了整体移植;一个经历了并发症和死亡,而另一个患者实现了症状解消.
- 第二名患者的CD复发了,但在免疫抑制停止后恢复了缓解,导致外周血液和肠道粘膜中占主导地位的捐赠者免疫嵌合体.
- 这位患者在8年内停用了所有药物,轻微的症状通过严格uroplasty来控制关狭窄.
结论:
- 移植后的免疫奇默主义动力学受到调节强度和免疫抑制的影响.
- 只有在在肠道粘膜中确立了供体免疫奇默症后,在一名患者中才能实现持久的CD缓解.
- 整形移植是对耐火性CD的潜在治疗方法,在未来的研究中需要考虑免疫抑制条件.
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