自体血干细胞移植治疗耐克罗恩氏病:随机临床试验
Christopher J Hawkey1, Matthieu Allez2, Miranda M Clark1
1Nottingham Digestive Diseases Centre, School of Clinical Sciences, Queens Medical Centre, Nottingham, United Kingdom.
JAMA
|December 17, 2015
概括
血造干细胞移植 (HSCT) 没有显著改善耐克罗恩病患者的持续缓解. 治疗显示出显著的毒性, 这些发现不支持其广泛使用.
科学领域:
- 胃肠病学
- 免疫学
- 移植医学
背景情况:
- 克罗恩病是一种慢性炎症性肠病.
- 耐药病例往往会影响生活质量和有限的治疗选择.
- 血造干细胞移植 (HSCT) 是一种潜在的治疗方法.
研究的目的:
- 评估自主性HSCT在耐克克罗恩病的成年患者中的疗效和安全性.
- 在随机临床试验中比较HSCT与传统治疗.
主要方法:
- 在11个欧洲移植单位进行了平行组随机临床试验.
- 耐克性克朗病患者接受免疫和HSCT或标准治疗 (HSCT推迟).
- 通过临床,内镜和放射学标准定义的首要终点是持续的疾病缓解.
主要成果:
- 在HSCT患者中,8. 7%的患者在1年内获得持续的疾病缓解,而对照患者则为4. 5% (P=0. 60).
- 更多的HSCT患者停止了免疫抑制疗法 (61%对23%,P=. 01).
- 在HSCT组中,严重不良事件的发生率较高,报告有1例死亡.
结论:
- 自律性HSCT没有导致持续性疾病缓解的统计学上显著改善.
- 这种方法与显著的毒性有关.
- 目前的证据不支持在这种患者群体中广泛使用HSCT.
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