Abstract:
Over the past 25 years, hematopoietic stem cell transplantation (HSCT) has been evolving as specific treatment for patients with severe and refractory systemic autoimmune diseases, where mechanistic studies have provided evidence for a profound immune renewal facilitating the observed beneficial responses. In addition to autoimmune neurologic diseases, such as multiple sclerosis (MS) or neuromyelitis optica (NMO), rheumatic diseases with central or peripheral nervous system involvement and insufficient response to conventional immunosuppressive or biologic therapies represent a growing indication for autologous HSCT. They most commonly include connective tissue diseases, such as systemic lupus erythematosus (SLE), vasculitides, or rarer diseases from the autoinflammatory spectrum, such as Behçet\'s disease, where neurologic manifestations may represent the greatest disease burden. Neurologic manifestations may resemble those of MS, including myelitis optic neuropathy, stroke, or seizures. Outcomes of such manifestations are variable after autologous HSCT but most frequently improve or even resolve with the underlying disease, especially in SLE. This article will provide the current evidence and summarize the outcomes of HSCT for rheumatic autoimmune diseases with neurologic manifestations.
摘要:
在过去的25年里,造血干细胞移植(HSCT)已发展成为严重和难治性系统性自身免疫性疾病患者的特异性治疗方法,其中机理研究提供了深刻的免疫更新促进观察到的有益反应的证据。除了自身免疫性神经系统疾病,如多发性硬化症(MS)或视神经脊髓炎(NMO),有中枢神经系统或周围神经系统受累以及对常规免疫抑制或生物疗法反应不足的风湿性疾病代表了自体HSCT越来越多的适应症。它们最常见的包括结缔组织疾病,如系统性红斑狼疮(SLE),血管炎,或自身炎症谱中罕见的疾病,比如Behçet病,神经系统表现可能代表最大的疾病负担。神经系统表现可能类似于MS,包括脊髓炎视神经病变,中风,或癫痫发作。自体HSCT后,这些表现的结果是可变的,但最常见的是改善甚至解决潜在的疾病,尤其是在SLE。本文将提供目前的证据,并总结HSCT对有神经系统表现的风湿性自身免疫性疾病的治疗效果。