与组合疗法相比,通过自身造血干细胞移植治疗的进展性全身性硬化症的结果
Shiri Keret1, Israel Henig2, Tsila Zuckerman2
1Rheumatology Unit, Bnai Zion Medical Center, Faculty of Medicine, Technion, Haifa, Israel.
Rheumatology (Oxford, England)
|September 6, 2023
概括
对于早期扩散性渐进性系统性硬化症 (SSc) 符合自主造血干细胞移植 (AHSCT) 资格的患者,前期结合治疗与mycophenolate mofetil (MMF) 和rituximab提供了与AHSCT相比的临床改善和更好的安全性.
科学领域:
- 风湿病学和免疫学
- 血液学和干细胞移植
- 肺部病理学 肺部病理学
背景情况:
- 自主造血干细胞移植 (AHSCT) 改善了早期扩散性渐进性系统性硬化症 (SSc) 的生存率.
- 环胺 (CYC) 在SSc.中没有显示长期益处.
- 结合疗法与mycophenolate mofetil (MMF) 和rituximab是一个潜在的替代方案.
研究的目的:
- 为了追溯比较接受AHSCT的SSc患者的结果.
- 为了比较AHSCT与前期的MMF和rituximab联合治疗在SSc患者符合AHSCT.
主要方法:
- 21名接受MMF和rituximab的SSc患者与接受AHSCT的16名SSc患者的回顾性比较.
- 修改后的罗丹恩皮肤评分 (mRSS),强迫生命能力 (FVC) 和扩散能力 (DLCO) 的评估.
- 临床改善定义为>25%的mRSS降低或>10%的FVC增加;无事件生存 (EFS) 定义为没有持续的主要器官衰竭或死亡.
主要成果:
- 在12个月后,临床改善率相似 (86%的组合 vs. 81%的AHSCT).
- 组合疗法有利于24个月的无事件生存 (HR=0.09,P=0.04).
- 两组都显示了mRSS的可比降低和FVC的增加,长达24个月.
结论:
- 在符合条件的SSc患者中,MMF和rituximab联合治疗与AHSCT相比,显示出类似的皮肤和肺部改善.
- 货币货币基金和rituximab疗法在24个月后显示出更好的安全性.
- 前期组合疗法是选择SSc患者的AHSCT可行的替代方案.
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