托法替尼治疗幼年特发性关节炎研究进展

doi:10.19803/j.1672-8629.20220611

摘要/Abstract

摘要： 幼年特发性关节炎是一种病因不清、高度异质性的儿童时期常见关节炎,为自身免疫性疾病。托法替尼作为一种新型的口服小分子Janus激酶（主要是JAK1和JAK3）特异性抑制剂,改变了不同年龄阶段许多皮肤疾病的治疗。2020年9月,美国食品药品监督管理局（FDA）批准托法替尼用于2岁及以上的活动性多关节型幼年特发性关节炎,目前在儿童幼年特发性关节炎中的应用相对较少,因此在儿童中使用的安全性、有效性和耐受性证据有限。本文主要从托法替尼治疗幼年特发性关节炎的作用机制、药动学特征、临床应用及安全性4个方面总结其相关研究进展,为未来开展多中心临床研究提供参考。

关键词: 托法替尼, 幼年特发性关节炎, 作用机制, 药动学特征, 临床应用, 安全性

Abstract: Juvenile idiopathic arthritis, an autoimmune disease, is a common childhood arthritis with unclear etiology and high heterogeneity. Tofacitinib, as a novel oral specific inhibitor of small molecule Janus kinases (mainly JAK1 and JAK3), has revolutionized the treatment of several types of skin diseases in different age groups. Tofacitinib was approved in September 2020 for active polyarticular juvenile idiopathic arthritis 2 years of age and older by the United States Food and Drug Administration. There are relatively few applications in children with juvenile idiopathic arthritis, leaving evidence about safety, efficacy, and tolerability for use in children limited. This article summarizes the research progress in the treatment of juvenile idiopathic arthritis with tofacitinib in terms of the mechanism of action, pharmacokinetic characteristics, clinical application and safety so as to provide reference for future multicenter clinical studies.

Key words: tofacitinib, juvenile idiopathic arthritis, mechanism of action, pharmacokinetic profile, clinical applications, safety

中图分类号:

R971
R994

李亚梅, 水荣. 托法替尼治疗幼年特发性关节炎研究进展[J]. 中国药物警戒, 2023, 20(8): 956-960.

LI Yamei, SHUI Rong. Research progress in tofacitinib for juvenile idiopathic arthritis[J]. Chinese Journal of Pharmacovigilance, 2023, 20(8): 956-960.

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