Center of Clinical Neuroscience, University Hospital Carl Gustav Carus, Dresden University of Technology, Dresden D-01307, Germany.
Novartis Pharma AG, CH-4002 Basel, Switzerland.
Autoimmun Rev. 2017 Apr;16(4):355-376. doi: 10.1016/j.autrev.2017.02.007. Epub 2017 Feb 15.
The aim of our study was to systematically review the growing body of published literature reporting on one specific multiple sclerosis (MS) treatment, fingolimod, in the real world to assess its effectiveness in patients with MS, evaluate methodologies used to investigate MS in clinical practice, and describe the evidence gaps for MS as exemplified by fingolimod.
We conducted a PRISMA-compliant systematic review of the literature (cut-off date: 4 March 2016). Published papers reporting real-world data for fingolimod with regard to clinical outcomes, persistence, adherence, healthcare costs, healthcare resource use, treatment patterns, and patient-reported outcomes that met all the eligibility criteria were included for data extraction and quality assessment.
Based on 34 included studies, this analysis found that fingolimod treatment improved outcomes compared to the period before treatment initiation and was more effective than interferons or glatiramer acetate. However, among studies comparing fingolimod with natalizumab, overall trends were inconsistent: some reported natalizumab to be more effective than fingolimod and others reported similar effectiveness for natalizumab and fingolimod. These studies illustrate the challenges of investigating MS in the real world, including the subjectivity in evaluating some clinical outcomes and the heterogeneity of methodologies used and patient populations investigated, which limit comparisons across studies. Gaps in available real-world evidence for MS are also highlighted, including those relating to patient-reported outcomes, combined clinical outcomes (to measure overall treatment effectiveness), and healthcare costs/resource use.
The included studies provide good evidence of the real-world effectiveness of fingolimod and highlight the diversity of methodologies used to assess treatment benefit in clinical practice. Future studies could address the evidence gaps found in the literature and the challenges associated with researching MS when designing real-world studies, assessing data, and comparing evidence across studies.
我们的研究目的是系统地回顾越来越多的关于多发性硬化症（MS）单一治疗药物——芬戈莫德的已发表文献，以评估其在 MS 患者中的有效性，评估在临床实践中研究 MS 所采用的方法，并举例说明芬戈莫德如何体现 MS 的证据差距。
我们按照 PRISMA 原则进行了文献系统回顾（截止日期：2016 年 3 月 4 日）。纳入的文献需为报告了关于芬戈莫德的真实世界数据的研究，包括临床结局、持久性、依从性、医疗成本、医疗资源使用、治疗模式和患者报告结局等，所有纳入研究均须符合纳入和排除标准，并进行数据提取和质量评估。
基于 34 项纳入研究，该分析发现与治疗前相比，芬戈莫德治疗可改善结局，且比干扰素或那他珠单抗更有效。然而，在比较芬戈莫德与那他珠单抗的研究中，整体趋势并不一致：一些研究报告那他珠单抗比芬戈莫德更有效，而另一些研究则报告那他珠单抗和芬戈莫德的疗效相似。这些研究说明了在真实世界中研究 MS 面临的挑战，包括一些临床结局评估的主观性以及所采用的方法学和研究人群的异质性，这限制了研究间的比较。也突出了 MS 真实世界证据的差距，包括与患者报告结局、综合临床结局（衡量整体治疗效果）和医疗成本/资源使用相关的差距。
纳入的研究为芬戈莫德的真实世界疗效提供了良好的证据，并强调了在临床实践中评估治疗益处所采用的方法多样性。未来的研究可以在设计真实世界研究、评估数据和比较研究证据时，解决文献中发现的证据差距以及与研究 MS 相关的挑战。
