Head-to-head drug comparisons in multiple sclerosis

Head-to-head drug comparisons in multiple sclerosis
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多发性硬化症的头对头药物比较

DOI:
10.1212/wnl.0000000000008319
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发表时间:
2019
期刊:
影响因子:
9.9
通讯作者:
X. Montalban
X. Montalban
中科院分区:
医学1区
文献类型:
--
作者:
C. Tur;T. Kalincik;Jiwon Oh;M. Sormani;M. Tintoré;H. Butzkueven;X. Montalban

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疾病修饰药物正在改变多发性硬化症(MS)的自然病史。然而,目前可用的临床试验数据不足以开发准确的个性化治疗算法,以便根据疾病特征、治疗历史和合并疾病为每个MS患者分配可能的最佳治疗。如此精确的算法需要大量长时间的面对面试验,考虑到经济成本、所需时间和自然减员的困难,这几乎是不可能的。因此,正在努力通过观察性设计、使用大型多中心前瞻性队列或“大MS数据”和网络荟萃分析来比较相对治疗效果。虽然这样的研究可以提供有用的信息,但它们容易产生偏差,其结果应该在其他研究人群中得到证实,包括较小的单中心队列,其中一些偏差可以被最小化。在这篇观点文章中,我们分析了所有这些策略替代面对面试验在多发性硬化症中的潜在好处和偏见。最后,我们建议在缺乏随机设计的情况下,结合所有这些类型的研究来获得可靠的面对面药物比较。
Disease-modifying drugs are changing the natural history of multiple sclerosis (MS). However, currently available clinical trial data are insufficient to develop accurate personalized treatment algorithms to assign the best possible treatment to each person with MS according to disease features, treatment history, and comorbidities. Such accurate algorithms would require the presence of numerous head-to-head trials of long duration, which is virtually impossible, given the economic costs, required time, and difficulties with attrition. Thus, efforts are being made to compare relative treatment efficacy through observational designs, using large multicenter prospective cohorts or “big MS data,” and network meta-analyses. Although such studies can yield useful information, they are liable to biases and their results should be confirmed in other study populations, including smaller, single-center cohorts, where some of these biases can be minimized. In this View article, we analyze the potential benefits and biases of all these strategies alternative to head-to-head trials in MS. Finally, we propose the combination of all these types of studies to obtain reliable head-to-head drug comparisons in the absence of randomized designs.
DOI: 10.1093/aje/kwv254
发表时间: 2016-04-15
影响因子: 5
作者:
Hernan, Miguel A.;Robins, James M.
通讯作者: Robins, James M.
DOI: 10.1111/1475-6773.12182
发表时间: 2014-10-01
影响因子: 3.4
作者:
Garrido, Melissa M.;Kelley, Amy S.;Aldridge, Melissa D.
通讯作者: Aldridge, Melissa D.