患有扩散性系统性硬化症,有资格接受传统疗法治疗的自身干细胞移植的患者的结果
Kate Gregory1, Dylan Hansen2, Ross Penglase3
1Monash University and Monash Health, Melbourne, Victoria, Australia.
Arthritis & rheumatology (Hoboken, N.J.)
|April 1, 2024
概括
与ASCT接受者相比,不接受自身干细胞移植 (ASCT) 的澳大利亚患有扩散性皮肤系统性硬化症 (dcSSc) 的患者表现出类似的无事件生存期 (EFS). 这表明,改善护理标准可能会改善dcSSc患者的治疗结果.
科学领域:
- 类风湿病学 类风湿病学
- 免疫学 免疫学 免疫学
- 血液学 血液学 血液学
背景情况:
- 扩散性皮肤全身性硬化症 (dcSSc) 是一种严重的自身免疫性疾病.
- 自主干细胞移植 (ASCT) 是对耐火性dcSSc.的潜在治疗方法.
- 之前的试验,如ASTIS和SCOT,为ASCT和环胺治疗建立了基准.
研究的目的:
- 评估符合ASCT试验标准但未接受移植的澳大利亚dcSSc患者的无事件生存 (EFS).
- 为了将这些患者的结果与历史的ASCT和环胺试验数据进行比较.
主要方法:
- 从澳大利亚硬化硬质皮症队列研究 (ASCS) 中确定了dcSSc患者,他们符合ASTIS或SCOT试验的纳入标准.
- 评估的4年EFS,定义为没有心脏,或肺衰竭或死亡的生存期.
- 不包括已经接受过ASCT的ASCS患者.
主要成果:
- 在ASCS dcSSc患者中,11.4%符合ASTIS标准;6.1%符合SCOT标准.
- 满足ASTIS标准的4年EFS为83.3%,满足SCOT标准的EFS为81.2%.
- 符合包括但也符合排除标准的患者EFS显著降低 (46.7%和45.7%).
结论:
- 符合ASCT试验标准但未移植的ASCS患者的4年EFS与ASCT接受者相比较.
- 在历史试验中,结果优于环胺治疗.
- 改善的EFS可能反映了提高的护理标准,或受到诸如幸存者偏见等混因素的影响.
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