Adalimumab in Juvenile Idiopathic Arthritis-associated Uveitis Trial (ADJUST) - Diversity Supplement

项目摘要 多达三分之一的幼年特发性关节炎（JIA）患者发展为慢性、无症状、前节段性关节炎。 在关节炎发作后4 - 7年内发生葡萄膜炎。患有JIA相关性葡萄膜炎的患者通常具有不良的长期预后。 视力结果，包括近10%至15%的患者成为法定失明。阿达木单抗，一种TNF-α 抑制剂，已被证明是一种有效的治疗青少年特发性关节炎相关（JIA） 葡萄膜炎，但没有证据为基础的指导方针的最佳治疗时间。存在安全 阿达木单抗作为TNF-α抑制剂长期治疗的担忧与风险增加相关 机会性感染和免疫介导的抗药性的发展。阿达木单抗也是 对病人和医疗系统来说都很昂贵。英国国家卫生服务局实施了一项 因经济原因对治疗反应良好的儿童停用阿达木单抗的政策， 然而，并没有科学证据支持这种强制性的退出政策。 阿达木单抗治疗青少年特发性关节炎（JIA）相关葡萄膜炎停止试验（ADJUST）是一项 研究停用阿达木单抗患者葡萄膜炎复发率的随机对照试验 与继续治疗的患者相比，治疗的目的如下：（1）在对照组患者中， JIA相关葡萄膜炎，比较患者眼部炎症复发率和至复发时间 与继续治疗的患者相比，随机停用阿达木单抗，（2）确定 JIA相关葡萄膜炎疾病复发的预测因子，以及（3）确定停用阿达木单抗是否导致 12个月访视时炎症控制总体较差，即使患者在葡萄膜炎后重新开始阿达木单抗治疗 复发 本研究将为以下患者停用阿达木单抗的风险和获益指南提供证据基础： JIA相关性葡萄膜炎患者。这个建议的多样性补充将支持我们的候选人在学习 如何管理国际眼科临床试验并获得第一手数据经验 通过使用ADJUST和其他已建立的数据集进行管理和分析。

PROJECT SUMMARY Up to one-third of all patients with juvenile idiopathic arthritis (JIA) develop chronic, asymptomatic, anterior uveitis within 4 to 7 years of their arthritis onset. Patients with JIA-associated uveitis often have poor long-term visual outcomes, including nearly 10 to 15% of patients becoming legally blind. Adalimumab, a TNF-alpha inhibitor, has been demonstrated to be an effective treatment for juvenile idiopathic arthritis-associate (JIA) uveitis, but there is no evidence-based guidelines for the optimal duration of treatment. There are safety concerns with long-term adalimumab treatment as TNF-alpha inhibitors are associated with an increased risk of opportunistic infections and development of immune-mediated resistance to drug. Adalimumab is also expensive for patients and the healthcare systems. The National Health Service in the UK has implemented a policy to withdraw adalimumab from children who respond well to the treatment because of economic reasons, yet there is no scientific evidence to back up this policy of mandatory withdrawal. The ADalimumab in Juvenile Idiopathic Arthritis (JIA)-associated Uveitis Stopping Trial (ADJUST) is a randomized controlled trial to examine uveitis recurrence rates between patients who discontinue adalimumab treatment compared to patients who continue treatment with the following aims: (1) In patients with controlled JIA-associated uveitis, to compare rate of recurrence and time to recurrence of ocular inflammation in patients randomized to discontinue adalimumab compared to those who continue treatment, (2) To determine predictors of JIA-associated uveitis disease recurrence, and (3) To determine if stopping adalimumab leads to overall less control of inflammation at the 12-month visit, even if patients restart adalimumab after a uveitis recurrence. This study will provide an evidence base for guidelines on the risks and benefits of withdrawing adalimumab in patients with JIA-associated uveitis. This proposed diversity supplement will support our candidate in learning how to manage international clinical trials in ophthalmology and gain first-hand experience in data management and analysis by working on ADJUST and other established datasets.