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系统性硬化症中的干细胞疗法。

Stem cell therapy in systemic sclerosis.

作者信息

Sakkas Lazaros I, Simopoulou Theodora, Alexiou Ioannis, Liaskos Christos, Chikanza Ian C

机构信息

Faculty of Medicine, School of Health Sciences, University of Thessaly, 41110, Larissa, Greece.

Department of Rheumatology and clinical Immunology, Faculty of Medicine, School of Health Sciences, University of Thessaly, Larissa, 41 110, Greece.

出版信息

Clin Rheumatol. 2025 Jul 2. doi: 10.1007/s10067-025-07557-y.

Abstract

Systemic sclerosis is a complex autoimmune disease with widespread fibrosis in skin and internal organs, microvasculopathy, and autoantibodies. The disease causes ischemic changes and leads to impairment of internal organs with reduced quality of life and life expectancy. The pathogenesis is not clearly known but involves adaptive and innate immune cells which infiltrate skin lesions mostly early in the disease process. Current treatment is based on immunosuppressives, but there is a significant unmet therapeutic need, and a new therapeutic approach is required. Autologous haematopoietic stem cell transplantation appears to be an effective therapeutic option for SSc but requires standardization to reduce transplant-related mortality and post-transplant adverse effects. Mesenchymal stem cells (MSCs), exerting immunosuppressive, antifibrotic, and angiogenic actions, appear to be a promising therapeutic option but require further refinement. MSC-derived microvesicles retain MSC functions and circumvent some of the MSC challenges and thus may provide a more favorable therapeutic approach. Key Points • There are unmet therapeutic needs for systemic sclerosis. • Autologous haematopoietic stem cell transplantation is an effective therapeutic option but needs standarization. • Mesenchymal stem cells are a promising therapeutic option but requires refinement.

摘要

系统性硬化症是一种复杂的自身免疫性疾病,其特征为皮肤和内脏广泛纤维化、微血管病变以及自身抗体。该疾病会引发缺血性改变,并导致内脏功能受损,进而降低生活质量和预期寿命。其发病机制尚不清楚,但涉及适应性免疫细胞和先天性免疫细胞,这些细胞大多在疾病早期浸润皮肤病变部位。目前的治疗基于免疫抑制剂,但仍存在显著未满足的治疗需求,因此需要一种新的治疗方法。自体造血干细胞移植似乎是系统性硬化症的一种有效治疗选择,但需要标准化以降低移植相关死亡率和移植后不良反应。间充质干细胞具有免疫抑制、抗纤维化和血管生成作用,似乎是一种有前景的治疗选择,但需要进一步完善。间充质干细胞衍生的微泡保留了间充质干细胞的功能,规避了间充质干细胞面临的一些挑战,因此可能提供一种更有利的治疗方法。要点 • 系统性硬化症存在未满足的治疗需求。 • 自体造血干细胞移植是一种有效的治疗选择,但需要标准化。 • 间充质干细胞是一种有前景的治疗选择,但需要完善。

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