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生物治疗:治疗单基因自身炎症性疾病的新武器。

Biological treatments: new weapons in the management of monogenic autoinflammatory disorders.

机构信息

Research Center of Systemic Autoimmune and Autoinflammatory Diseases, Rheumatology Unit, Policlinico Le Scotte, University of Siena, Viale Bracci 1, 53100 Siena, Italy.

出版信息

Mediators Inflamm. 2013;2013:939847. doi: 10.1155/2013/939847. Epub 2013 Jul 21.

Abstract

Treatment of monogenic autoinflammatory disorders, an expanding group of hereditary diseases characterized by apparently unprovoked recurrent episodes of inflammation, without high-titre autoantibodies or antigen-specific T cells, has been revolutionized by the discovery that several of these conditions are caused by mutations in proteins involved in the mechanisms of innate immune response, including components of the inflammasome, cytokine receptors, receptor antagonists, and oversecretion of a network of proinflammatory molecules. Aim of this review is to synthesize the current experience and the most recent evidences about the therapeutic approach with biologic drugs in pediatric and adult patients with monogenic autoinflammatory disorders.

摘要

治疗单基因自身炎症性疾病,这是一组遗传性疾病,其特征是明显无诱因的炎症反复发作,没有高滴度自身抗体或抗原特异性 T 细胞。这些疾病中的几种是由参与先天免疫反应机制的蛋白质的突变引起的,包括炎症小体的组成部分、细胞因子受体、受体拮抗剂以及促炎分子网络的过度分泌,这一发现彻底改变了该病的治疗方法。本篇综述的目的是综合目前在儿科和成年单基因自身炎症性疾病患者中使用生物药物治疗的经验和最新证据。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0191/3736401/8f5ef966931b/MI2013-939847.001.jpg

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