自身免疫性疾病的治疗现状与挑战

Current situation and challenges of the treatment of autoimmune diseases

自身免疫性疾病（AID）是指自身免疫耐受被打破,激活免疫系统攻击自身抗原,引起组织器官损害的一类疾病.AID可分为器官特异性和系统性两大类,其病因和发病机制尚不清楚.目前尚无根治药物,传统的糖皮质激素和免疫抑制剂适时应用能够控制病情,提高患者生存率.但长期使用会带来一系列不良反应,严重时可影响患者生活质量,甚或威胁生命.而且,还有部分患者对糖皮质激素和免疫抑制剂治疗不敏感.因此,迫切需要新的安全有效治疗药物和方法应用于临床.随着对AID认识的不断深入,发现了许多新的治疗靶点,研发了许多新的治疗药物和方法,包括基因治疗、细胞治疗、表观遗传干预等,针对细胞因子、B细胞和共刺激分子的生物制剂等治疗新策略成为传统治疗方法的重要补充,但由于种种因素的限制,这些治疗方法目前还不能取代糖皮质激素成为AID临床治疗的一线药物.本文就AID的治疗现状与存在的挑战做一综述.

Autoimmune diseases （AIDs） refer to immune disorders in which the immune tolerance of self antigens is broken to lead to multiple organ damages. AIDs are categorized into organ-specific and systemic AIDs. The exact mechanism underlying AIDs is still elusive, and challenges remain in the treatment decisions. Conventional therapeutic strategies rely on corticosteroids and immuno- suppressive reagents, which can control disease conditions and have increased the survival of patients with AIDs, but the drug-associat- ed adverse effects negatively impact the quality of patients＇life, sometimes can even be life threatening. Moreover, some patients are non-responsive to conventional therapy. Thus, novel therapeutic strategies are required. Based on the progress made in elucidating mo- lecular mechanisms underlying AIDs, novel therapeutic targets are identified. Gene therapy, cell therapy, epigenetic interference, and biological medication have become important supplement to conventional therapy and have improved disease activities. However, chal- lenges remain and limit these strategies to become the first line therapy. In this review, we summarized the current situation and chal- lenges of the treatment of autoimmune diseases.