肌炎
医学
包涵体肌炎
临床试验
临床研究设计
物理疗法
安慰剂
纳入和排除标准
重症监护医学
物理医学与康复
替代医学
病理
作者
Didem Saygın,Victoria P. Werth,Julie J Paik,Jin Kyun Park,Merrilee Needham,Ingrid E. Lundberg,Lisa Christopher‐Stine
标识
DOI:10.1136/ard-2023-224652
摘要
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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