乳糜泻试验的结局指标：坦佩雷建议。
Outcome measures in coeliac disease trials: the Tampere recommendations.
机构信息
Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Stockholm, Sweden.
Department of Pediatrics, Örebro University Hospital, Örebro, Sweden.
出版信息
Gut. 2018 Aug;67(8):1410-1424. doi: 10.1136/gutjnl-2017-314853. Epub 2018 Feb 13.
Abstract
OBJECTIVE
A gluten-free diet is the only treatment option of coeliac disease, but recently an increasing number of trials have begun to explore alternative treatment strategies. We aimed to review the literature on coeliac disease therapeutic trials and issue recommendations for outcome measures.
DESIGN
Based on a literature review of 10 062 references, we (17 researchers and 2 patient representatives from 10 countries) reviewed the use and suitability of both clinical and non-clinical outcome measures. We then made expert-based recommendations for use of these outcomes in coeliac disease trials and identified areas where research is needed.
RESULTS
We comment on the use of histology, serology, clinical outcome assessment (including patient-reported outcomes), quality of life and immunological tools including gluten immunogenic peptides for trials in coeliac disease.
CONCLUSION
Careful evaluation and reporting of outcome measures will increase transparency and comparability of coeliac disease therapeutic trials, and will benefit patients, healthcare and the pharmaceutical industry.
摘要
目的
无麸质饮食是乳糜泻的唯一治疗选择，但最近越来越多的试验开始探索替代治疗策略。我们旨在综述乳糜泻治疗试验的文献，并对结局指标提出建议。
设计
我们（来自 10 个国家的 17 名研究人员和 2 名患者代表）基于对 10062 篇参考文献的文献回顾，回顾了临床和非临床结局指标的使用和适用性。然后，我们针对这些结局指标在乳糜泻试验中的应用提出了专家建议，并确定了需要研究的领域。
结果
我们就组织学、血清学、临床结局评估（包括患者报告结局）、生活质量以及免疫工具（包括麸质免疫原性肽）在乳糜泻试验中的应用进行了评论。
结论
仔细评估和报告结局指标将提高乳糜泻治疗试验的透明度和可比性，并使患者、医疗保健和制药行业受益。
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