Pediatric Rheumatologist, Western University, Children's Hospital, London Health Sciences Centre, 800 Commissioners Road East, London, Ontario N6A5W9 , Canada +519 685 8138 ; +519 685 8156 ;
Expert Opin Biol Ther. 2013 Nov;13(11):1623-30. doi: 10.1517/14712598.2013.840580. Epub 2013 Sep 26.
Juvenile idiopathic arthritis (JIA) is a relatively common multidimensional and heterogeneous chronic disease of childhood. Children with JIA are at risk for significant morbidity in terms of joint damage, impairments in physical function and health-related quality of life. Outcomes for children with JIA have significantly improved with the use of biologic therapies in the past 15 years, with the most clinical experience being with etanercept .
Basic pharmacokinetic and pharmacodynamic data for etanercept will be highlighted. This article will review the clinical trials and open-label registry data for the efficacy and safety of etanercept for use in JIA.
Etanercept is very effective for the treatment of JIA. Data from clinical trials and open-label studies support its clinical efficacy in 80% of patients which appears to be sustained over several years for the majority of treated patients. The safety profile is also acceptable with a serious adverse event rate of 0.03 - 0.12 per patient-year. Further research is needed to evaluate any possible link between biologic therapy, JIA and malignancy, to obtain more long-term safety data, and to document improvements in quality of care and cost-benefit for associated with biologic therapies which may additionally assist in access to these medications. Further, identification of potential clinical or laboratory markers allowing for prediction of response and timing of starting and cessation of this biologic therapy are urgently required.
幼年特发性关节炎(JIA)是一种相对常见的儿童多维度和异质性慢性疾病。患有 JIA 的儿童在关节损伤、身体功能和健康相关生活质量方面存在显著发病风险。在过去 15 年中,生物疗法的应用显著改善了儿童 JIA 的预后,其中最具临床经验的是依那西普。
将重点介绍依那西普的基本药代动力学和药效学数据。本文将回顾依那西普治疗 JIA 的疗效和安全性的临床试验和开放标签注册数据。
依那西普对 JIA 的治疗非常有效。临床试验和开放标签研究的数据支持其在 80%的患者中具有临床疗效,且在大多数接受治疗的患者中,这种疗效似乎可持续数年。安全性状况也可以接受,严重不良事件发生率为每患者年 0.03-0.12。需要进一步研究以评估生物疗法、JIA 和恶性肿瘤之间的任何可能联系,以获得更多的长期安全性数据,并记录与生物疗法相关的护理质量和成本效益的改善,这可能有助于获得这些药物。此外,迫切需要确定潜在的临床或实验室标志物,以预测对该生物疗法的反应,并确定开始和停止这种生物疗法的时间。