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骨关节炎预防或风险降低的临床试验中的方法学问题。

Methodologic issues in clinical trials for prevention or risk reduction in osteoarthritis.

机构信息

Thurston Arthritis Research Center, University of North Carolina, Chapel Hill, NC 27599-7280, USA.

出版信息

Osteoarthritis Cartilage. 2011 May;19(5):500-8. doi: 10.1016/j.joca.2010.10.031. Epub 2011 Mar 23.

DOI:10.1016/j.joca.2010.10.031
PMID:21396470
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3260468/
Abstract

The design and execution of prevention trials for OA have methodological issues that are distinct from trials designed to impact prevalent disease. Disease definitions and their precise and sensitive measurement, identification of high-risk populations, the nature of the intervention (pharmaceutical, nutraceutical, behavioral) and its potential pleiotropic impacts on other organ systems are critical to consider. Because prevention trials may be prolonged, close attention to concomitant life changes and co-morbidities, adherence and participant retention in the trial is of primary importance, as is recognition of the potential for "preventive misconception" and "behavioral disinhibition" to affect the ability of the trial to show an effect of the intervention under study. None of these potential pitfalls precludes a successful and scientifically rigorous process and outcome. As technology improves the means to measure and predict the OA process and its clinical consequences, it will be increasingly possible to screen individuals for high-risk phenotypes, combining clinical factors with information from imaging, genetic, metabolic and other biomarkers and to impact this high-risk condition to avoid or delay OA both structurally and symptomatically.

摘要

OA 的预防试验设计和实施存在一些方法学问题,这些问题与旨在影响现有疾病的试验不同。疾病定义及其精确和敏感的测量、高危人群的识别、干预措施(药物、营养保健品、行为)的性质及其对其他器官系统的潜在多效性影响都需要认真考虑。由于预防试验可能会持续很长时间,因此密切关注伴随的生活变化和合并症、参与者在试验中的依从性和保留率非常重要,同时还需要认识到“预防误解”和“行为抑制”可能会影响试验显示研究干预措施效果的能力。这些潜在的陷阱都不会排除成功和严格科学的过程和结果。随着技术的进步,我们可以越来越多地使用各种手段来测量和预测 OA 进程及其临床后果,从而有可能对高危表型进行个体筛查,将临床因素与影像学、遗传学、代谢学和其他生物标志物信息相结合,并对这种高危情况进行干预,以避免或延迟 OA 的结构性和症状性发生。

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