Hügle Thomas, Huigens Christiaan A, van Laar Jacob M
Musculoskeletal Research Group, Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.
Arzneimittelforschung. 2009;59(12):615-24. doi: 10.1055/s-0031-1296449.
Systemic sclerosis (SSc) is an autoimmune disease of unknown origin. The clinical hallmarks are progressive fibrosis of skin and internal organs and vasculopathic changes in the form of digital ulcers and pulmonary arterial hypertension. The chronicity and heterogeneity of the disease has hampered research in SSc in the past, but new research tools and animal models have contributed to a greater understanding of the pathogenesis of SSc and resulted in new findings in the fields of genomics, cytokine expression, autoantibodies and abnormalities of blood progenitor or effector cells. As a consequence, targeted therapeutic compounds such as imatinib are currently under clinical investigation. Whilst the search for an effective "targeted therapy" is still ongoing, autologous stem cell transplantation represents a "multitarget" approach aiming at "resetting" the immune system. This review gives an overview of the translation from pathogenic findings into therapeutical application.
系统性硬化症（SSc）是一种病因不明的自身免疫性疾病。其临床特征为皮肤和内脏进行性纤维化以及以指端溃疡和肺动脉高压形式出现的血管病变。过去，该疾病的慢性和异质性阻碍了系统性硬化症的研究，但新的研究工具和动物模型有助于更深入地了解系统性硬化症的发病机制，并在基因组学、细胞因子表达、自身抗体以及血液祖细胞或效应细胞异常等领域取得了新发现。因此，诸如伊马替尼等靶向治疗化合物目前正在进行临床研究。虽然寻找有效的“靶向治疗”仍在继续，但自体干细胞移植代表了一种旨在“重置”免疫系统的“多靶点”方法。本综述概述了从致病研究结果到治疗应用的转化。
