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系统性硬化症治疗的新视野:2025年进步和新兴疗法

Cristiana Sieiro Santos¹, Francesco Del Galdo^2,3

¹The University of Manchester Centre for Musculoskeletal Research, Manchester, UK.

|July 1, 2025

概括

系统性硬化症 (SSc) 治疗正在发展,已建立的器官参与疗法和新兴的个性化方法,如CAR-T细胞. 这些进展旨在改变疾病的发展轨迹,并为SSc患者提供早期,有针对性的干预措施.

科学领域:

风湿病学和免疫学
内部医学内部医学
药理学药理学是一个学科.

背景情况:

系统性硬化症 (SSc) 是一种罕见的多系统性自身免疫性疾病,导致显著的发病率和死亡率.
由于迅速的进展,目前的治疗指南 (EULAR) 正在更新.
新的治疗策略和精准医学正在改变SSc护理.

研究的目的:

综合目前基于器官的系统性硬化症治疗策略.
审查新兴疗法和个性化医疗方法在SSc.
强调疾病修饰策略和SSc护理的未来方向.

主要方法:

审查关于SSc治疗的当前文献.
对皮肤,肺部和心血管表现的已知治疗方法的综合.
分析新兴的治疗目标和个性化医疗方法.

主要成果:

已建立的免疫抑制剂 (mycophenolate mofetil,cyclophosphamide,rituximab) 和HSCT是皮肤/肺部问题的关键.
托西利祖马布和宁泰达尼布对于SSc相关的间歇性肺病 (SSc-ILD) 是至关重要的.
组合疗法和较新的药物 (selexipag, riociguat) 改善了肺动脉高血压 (PAH) 的结果.
新兴疗法 (CAR-T细胞,双特异性抗体,途径抑制剂) 标志着转向个性化,生物标志物驱动的SSc治疗.

结论:

目前的器官特异性治疗有效地解决了主要的SSc表现.
新兴疗法对改变疾病轨迹并使早期有针对性的干预成为可能具有重大前景.
预计系统性硬化症的个性化,生物标志物驱动的护理的未来.

关键词:

临床试验临床试验造血干细胞移植造血干细胞移植肺部疾病,间歇性疾病肺动脉高血压肺动脉高血压系统性硬化症系统性硬化症

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