目前肌炎的临床试验和磨难。

IF 20.3 1区 医学 Q1 RHEUMATOLOGY
Didem Saygin, Victoria Werth, Julie J Paik, Jin Kyun Park, Merrilee Needham, Ingrid E Lundberg, Lisa Christopher-Stine
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引用次数: 0

摘要

随着人们对疾病发病机理的进一步了解以及对结果测量方法的掌握,特发性炎症性肌病(肌炎)的治疗性临床试验数量在过去三年中显著增加,每个研究机构的试验数量多达五项。这些试验具有相似的设计和纳入/排除标准,导致肌炎临床试验竞争激烈。虽然这是肌炎领域令人振奋的时刻,但我们对肌炎试验的设计和实施仍有许多担忧。这些问题包括:竞争激烈、安慰剂组时间过长、少数群体参与人数不足、使用患者报告的结果测量方法,而这些方法在肌炎中的有效性数据有限/没有数据、疾病分类标准陈旧,以及 ACR/EULAR 肌炎反应标准在皮肤为主的患者中的表现不明确,尽管这些患者被纳入了试验。在本文中,我们将进一步讨论这些问题,并提出潜在的解决方案,例如在试验设计中纳入患者观点和采用创新框架。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Current myositis clinical trials and tribulations.

With improved understanding of disease pathogenesis and availability of outcome measures, there has been a remarkable increase in the number of therapeutic clinical trials in idiopathic inflammatory myopathies (myositis) over the last three years reaching as many as five trials per site. These trials share similar design and inclusion/exclusion criteria resulting in a competitive clinical trial landscape in myositis. While these are exciting times for the myositis field, we have a number of concerns about the design and conduct of the myositis trials. These include competitive landscape, lengthy placebo arms, underrepresentation of minority groups among participants, use of patient reported outcome measures with limited/no data on validity in myositis, antiquated disease classification criteria, and unclear performance of the ACR/EULAR Myositis Response Criteria in skin-predominant patients despite inclusion of these patients in trials. In this viewpoint, we further discuss these concerns and offer potential solutions such as including patient perspectives in the trial design and adoption of innovative frameworks.

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来源期刊
Annals of the Rheumatic Diseases
Annals of the Rheumatic Diseases 医学-风湿病学
CiteScore
35.00
自引率
9.90%
发文量
3728
审稿时长
1.4 months
期刊介绍: Annals of the Rheumatic Diseases (ARD) is an international peer-reviewed journal covering all aspects of rheumatology, which includes the full spectrum of musculoskeletal conditions, arthritic disease, and connective tissue disorders. ARD publishes basic, clinical, and translational scientific research, including the most important recommendations for the management of various conditions.
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