Hematopoietic and Bone Marrow Transplant Unit, San Raffaele Hospital, 20132 Milan, Italy.
Department of Rheumatology, ASST G. Pini-CTO, 20122 Milan, Italy.
Cells. 2022 Oct 24;11(21):3346. doi: 10.3390/cells11213346.
Systemic sclerosis (SSc) is a systemic disease characterized by autoimmune responses, vasculopathy and tissue fibrosis. The pathogenic mechanisms involve a wide range of cells and soluble factors. The complexity of interactions leads to heterogeneous clinical features in terms of the extent, severity, and rate of progression of skin fibrosis and internal organ involvement. Available disease-modifying drugs have only modest effects on halting disease progression and may be associated with significant side effects. Therefore, cellular therapies have been developed aiming at the restoration of immunologic self-tolerance in order to provide durable remissions or to foster tissue regeneration. Currently, SSc is recommended as the 'standard indication' for autologous hematopoietic stem cell transplantation by the European Society for Blood and Marrow Transplantation. This review provides an overview on cellular therapies in SSc, from pre-clinical models to clinical applications, opening towards more advanced cellular therapies, such as mesenchymal stem cells, regulatory T cells and potentially CAR-T-cell therapies.
系统性硬化症（SSc）是一种以自身免疫反应、血管病变和组织纤维化为特征的系统性疾病。发病机制涉及广泛的细胞和可溶性因子。这种复杂的相互作用导致皮肤纤维化和内脏器官受累的程度、严重程度和进展速度的临床表现存在异质性。现有的疾病修饰药物对阻止疾病进展仅有适度的效果，并且可能与显著的副作用相关。因此，已经开发了细胞疗法，旨在恢复免疫耐受，以提供持久的缓解或促进组织再生。目前，欧洲血液和骨髓移植学会将 SSc 推荐为自体造血干细胞移植的“标准适应证”。本文综述了 SSc 中的细胞疗法，从临床前模型到临床应用，展望了更先进的细胞疗法，如间充质干细胞、调节性 T 细胞和潜在的 CAR-T 细胞疗法。
