自体造血干细胞移植治疗系统性硬化症相关间质性肺病。

IF 5.2 2区 医学 Q1 RHEUMATOLOGY Current opinion in rheumatology Pub Date : 2024-11-01 Epub Date: 2024-09-27 DOI:10.1097/BOR.0000000000001050
Jana Zielonka, Jean Paul Higuero Sevilla
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引用次数: 0

摘要

综述目的:在过去的 25 年中,自体造血干细胞移植(HSCT)在弥漫性皮肤系统性硬化症(dcSSc)治疗中的作用已得到阐明。然而,这种疗法仍存在多个关键问题。尤其令人感兴趣的是造血干细胞移植在治疗系统性硬化症(SSc)相关间质性肺病方面的作用,因为这是导致系统性硬化症患者死亡的主要原因:大多数造血干细胞移植治疗系统性硬化症的临床试验和观察性研究都将肺部结果作为次要结果。尽管存在这些局限性,但越来越多的证据表明,造血干细胞移植可导致间质性肺病稳定,而且根据肺功能测试和影像学检查,肺功能可能得到改善。未来的研究需要完善或扩大患者选择范围,优化调理方案,并评估维持性免疫抑制的潜在作用。我们建议加强对间质性肺病的关注,因为这是导致 SSc 患者死亡的主要原因。
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Autologous hematopoietic stem cell transplant for systemic sclerosis associated interstitial lung disease.

Purpose of review: Over the last 25 years, the role of autologous hematopoietic stem cell transplant (HSCT) in the treatment of diffuse cutaneous systemic sclerosis (dcSSc) has been elucidated. However, multiple critical questions remain regarding this therapy. Of particular interest is the role of HSCT in the treatment of systemic sclerosis (SSc)-associated interstitial lung disease since this is the leading cause of death in SSc.

Recent findings: Most clinical trials and observational studies of HSCT for the treatment of dcSSc have reported pulmonary outcomes as secondary outcomes, Also, most studies have excluded patients with significant pulmonary function impairment. Despite these limitations, there is increasing evidence that suggests that HSCT leads to interstitial lung disease stabilization and possibly improvement of lung function based on pulmonary function tests and imaging.

Summary: HSCT has demonstrated improved long-term outcomes compared to conventional therapies for dcSSC. Future research is needed to refine or expand patient selection, optimize conditioning regimens, and evaluate the potential role of maintenance immunosuppression. We recommend an increased focus on interstitial lung disease since this is the primary cause of death in SSc.

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来源期刊
Current opinion in rheumatology
Current opinion in rheumatology 医学-风湿病学
CiteScore
9.70
自引率
2.00%
发文量
89
审稿时长
6-12 weeks
期刊介绍: A high impact review journal which boasts an international readership, Current Opinion in Rheumatology offers a broad-based perspective on the most recent and exciting developments within the field of rheumatology. Published bimonthly, each issue features insightful editorials and high quality invited reviews covering two or three key disciplines which include vasculitis syndromes, medical physiology and rheumatic diseases, crystal deposition diseases and rheumatoid arthritis. Each discipline introduces world renowned guest editors to ensure the journal is at the forefront of knowledge development and delivers balanced, expert assessments of advances from the previous year.
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