青少年异常性关节炎中的托法西替尼:双盲,安慰剂对照,停药阶段随机试验
Nicolino Ruperto1, Hermine I Brunner2, Olga Synoverska3
1IRCCS Istituto Giannina Gaslini, UOSID Centro Trial, PRINTO, Genova, Italy.
Lancet (London, England)
|November 12, 2021
概括
托法西替尼有效地减少了多关节性形性关节炎 (JIA) 的儿童和青少年的JIA爆发. 这种口服的Janus激酶抑制剂提供了一个新的治疗选择,可能比注射更好.
科学领域:
- 儿童风湿病学
- 免疫学
- 药理学
背景情况:
- 青少年异常性关节炎 (JIA) 涵盖了几种影响儿童的慢性自身免疫性疾病.
- 多关节性JIA涉及多个关节,需要有效的长期管理.
- 目前的JIA治疗包括生物药物和常规合成DMARDs,需要口服替代品.
研究的目的:
- 与安慰剂相比,对多关节性JIA患者评估托法西替尼的疗效和安全性.
- 评估托法西替尼对儿科患者中JIA复发率的影响.
- 探索托法西替尼作为一种口服治疗选择.
主要方法:
- 一个双盲,随机的,停药期3试验,涉及2至18岁多关节性JIA患者.
- 患者最初接受了18周的开放性托法西替尼,随后随机选择继续服用托法西替尼或转换为安慰剂26周.
- 主要终点是JIA在第44周的复发率,在整个研究过程中评估了安全性.
主要成果:
- 与安慰剂相比,托法西替尼在44周内显著降低了JIA发作率 (29%与53%相比;HR为0. 46).
- 77% 的托法西替尼患者和 74% 的安慰剂患者报告了不良反应;严重的不良反应很少 (1% 与 2% 相比).
- 接触托法西替尼的患者中有48%发生感染;没有报告死亡.
结论:
- 在多关节性JIA患者中,托法西替尼在减少JIA爆发方面表现出显著的有效性.
- 托法西替尼口服为儿科患者提供了一个方便的替代方案.
- 托法西替尼是治疗儿童和青少年多关节性JIA的一种有前途的新疗法.
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