Division of Rheumatology, University of Pavia, IRCCS S. Matteo Foundation, Pavia, Italy.
Medical Department, Pfizer Italy, Rome, Italy.
Clin Exp Rheumatol. 2019 May-Jun;37(3):485-495. Epub 2018 Aug 29.
Oral targeted synthetic disease-modifying anti-rheumatic drugs (DMARDs), including the Janus kinase inhibitors tofacitinib and baricitinib, are the latest addition to the therapeutic options for rheumatoid arthritis (RA). Tofacitinib 5 mg, twice daily, is approved for treatment, with or without methotrexate, of moderate to severe active RA in adults not adequately responding to, or not tolerating one or more DMARDs. In this narrative review we aimed to provide an overview of the real-world evidence for tofacitinib in RA.
The literature was reviewed up to March 2018 for studies regarding the efficacy and safety of tofacitinib for the treatment of RA. The focus was mainly on real-world studies with implications for every day clinical practice.
The efficacy and safety of tofacitinib have been comprehensively assessed in a wide programme of randomised controlled trials. Extensive observational research on tofacitinib in RA is also ongoing worldwide and a substantial body of post-marketing real-world data from clinical practice is becoming available. There was a degree of consistency across the real-world studies reviewed. Tofacitinib tends to be used as monotherapy more frequently than bDMARDS and appears to be effective without background methotrexate. The data show a manageable safety profile, with no new safety signals and a discontinuation rate from safety issues <10%. Patients initiating tofacitinib usually have longer disease duration and have been exposed to longer bDMARDs than patients initiating a bDMARD.
Real-world data are a key component of the evidence supporting the effectiveness of this novel drug and are of interest to all stakeholders. Treatment persistence and adherence to tofacitinib are good overall and similar to those seen for bDMARDs.
口服靶向合成改善病情的抗风湿药物（DMARDs），包括 Janus 激酶抑制剂托法替布和巴瑞替尼，是类风湿关节炎（RA）治疗选择的最新补充。托法替布 5mg，每日两次，获批用于治疗成人中对一种或多种 DMARDs 反应不足或不耐受的中度至重度活动性 RA，无论是否联合甲氨蝶呤。在本叙述性综述中，我们旨在概述托法替布治疗 RA 的真实世界证据。
对截至 2018 年 3 月的有关托法替布治疗 RA 的疗效和安全性的研究进行了文献回顾。重点主要是对具有日常临床实践意义的真实世界研究。
托法替布的疗效和安全性已在广泛的随机对照试验计划中得到全面评估。全球范围内也正在对托法替布治疗 RA 的观察性研究进行广泛研究，并且越来越多的上市后真实世界数据来自临床实践。在回顾的真实世界研究中存在一定程度的一致性。与 bDMARDs 相比，托法替布更常作为单药使用，并且似乎在没有背景甲氨蝶呤的情况下也有效。数据显示其具有可管理的安全性，没有新的安全性信号，因安全性问题停药率<10%。开始使用托法替布的患者通常疾病持续时间较长，并且接受 bDMARDs 的时间也比开始使用 bDMARDs 的患者长。
真实世界数据是支持这种新型药物有效性的证据的关键组成部分，也是所有利益相关者都感兴趣的。托法替布的治疗持续时间和对其的依从性总体良好，与 bDMARDs 相似。
