硬皮病的自体干细胞移植。

Autologous stem cell transplantation in scleroderma.

机构信息

Assistance Publique-Hôpitaux de Paris, Saint-Louis Hospital, Internal Medicine (UF04), MATHEC, Centre of reference for rare systemic autoimmune diseases (FAI2R); Université de Paris, EA 3518, Paris, France; McGill university, department of medicine, Montreal, QC, Canada.

出版信息

Presse Med. 2021 Apr;50(1):104065. doi: 10.1016/j.lpm.2021.104065. Epub 2021 Feb 3.

DOI:10.1016/j.lpm.2021.104065

PMID:33548374

Abstract

Patients with severe rapidly progressive systemic sclerosis (SSc) have a poor prognosis. Standard immunosuppressive therapies may have modest effects on stabilizing disease, but they fail to improve overall survival. Hematopoietic stem cell transplant (HSCT) is the first treatment to induce disease-modifying therapeutic benefits in rapidly progressive SSc patients. HSCT in rapidly progressive SSc can induce regression of fibrosis in skin and lung, and increase survival. Initially, HSCT was associated with high treatment-related mortality rates. Improvements in patient screening, a better understanding of the risks associated with different treatment regimens, and centre experience have improved the AHSCT safety profile for patients with scleroderma.

摘要

患有严重快速进展性系统性硬皮病（SSc）的患者预后不良。标准的免疫抑制疗法可能对稳定疾病有一定效果，但不能提高总体生存率。造血干细胞移植（HSCT）是第一种在快速进展性 SSc 患者中诱导疾病改善治疗效果的治疗方法。HSCT 可诱导皮肤和肺部纤维化的消退，并提高生存率。最初，HSCT 与高治疗相关死亡率相关。通过改进患者筛选、更好地了解不同治疗方案相关的风险以及中心经验，提高了 AHSCT 在硬皮病患者中的安全性。