Department of Pediatrics and Medicine, Division of Allergy, Immunology, and Rheumatology, University of California at San Diego, School of Medicine, 9500 Gilman Dr, La Jolla, CA 92093-0635, USA.
J Allergy Clin Immunol. 2009 Dec;124(6):1129-38; quiz 1139-40. doi: 10.1016/j.jaci.2009.11.001.
The therapy of autoinflammatory syndromes is an excellent example of the power of translational research. Recent advances in our understanding of the molecular and immunologic basis of this newly identified classification of disease have allowed for the application of novel, effective, targeted treatments with life-changing effects on patients. Although colchicine and TNF-alpha inhibitors are important therapies for specific autoinflammatory syndromes, the novel IL-1-targeted drugs are particularly effective for many of these diseases. Recently, the pharmaceutical industry has adopted a strategy of confirming the efficacy of new targeted drugs in often-ignored patients with orphan diseases, and US Food and Drug Administration policies have allowed for accelerated approval of these drugs, creating a win-win situation for patients and industry. This article reviews the general approach to the therapy of autoinflammatory diseases, focusing on current approved therapies and novel approaches that might be used in the future.
自身炎症性疾病的治疗是转化研究威力的绝佳例证。我们对这类新确定疾病分类的分子和免疫学基础的理解不断深入，这使得针对这些疾病的新型、有效、靶向治疗得以应用，为患者带来了改变人生的效果。尽管秋水仙碱和 TNF-α 抑制剂是某些特定自身炎症性疾病的重要治疗方法，但新型 IL-1 靶向药物对许多此类疾病特别有效。最近，制药行业采用了一种策略，即在经常被忽视的孤儿疾病患者中确认新型靶向药物的疗效，美国食品和药物管理局的政策也允许加速批准这些药物，为患者和行业创造了双赢局面。本文综述了自身炎症性疾病治疗的一般方法，重点介绍了目前已批准的治疗方法和未来可能使用的新方法。
