Why Metrics Matter in Thyroid Eye Disease

IF 10.5 1区 医学 Q1 OPHTHALMOLOGY
Michael Kazim, Lelio Baldeshi, A. Augusto Cruz, Peter J. Dolman, Chrysoula Dosiou, Anja Eckstein, Don Kikkawa, Andrea L. Kossler, Louise A. Mawn, Naykky Singh Ospina, Petros Perros, Daniel B. Rootman, Mario Salvi, Marius N. Stan, Timothy J. Sullivan, Sara T. Wester
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引用次数: 0

Abstract

Importance Advances in the understanding of the pathophysiology of thyroid eye disease (TED) provide an opportunity to use novel, targeted therapeutic agents. However, our capacity to assess the efficacy and indications for the new agents is potentially limited by the clinical metrics used in recent research trials, which fail to adequately capture the clinical complexity and diversity of TED. Objectives To review the features of TED that make it atypical among autoimmune diseases, review the limitations of the currently available metrics, and introduce the Innovate TED Initiative to develop new outcome metrics. Conclusions and Relevance To best evaluate and use potential new therapeutic agents for TED, physicians with expertise in the field perceive the need for a novel set of metrics. The outlined Innovate TED Initiative can, through the design and execution of prospective validation studies, develop the requisite metrics for the mutual benefit of physicians, regulatory bodies, the pharmaceutical industry, and patients with TED.
为什么指标在甲状腺眼病中很重要
重要性甲状腺眼病(TED)病理生理学的进展为使用新的靶向治疗药物提供了机会。然而,我们评估新药疗效和适应症的能力可能受到近期研究试验中使用的临床指标的限制,这些指标未能充分捕捉到TED的临床复杂性和多样性。目的回顾TED在自身免疫性疾病中不典型的特点,回顾目前可用指标的局限性,并介绍创新TED倡议以开发新的结果指标。结论和相关性为了最好地评估和使用潜在的新治疗药物,在该领域具有专业知识的医生认为需要一套新的指标。通过前瞻性验证研究的设计和执行,概述的创新TED倡议可以为医生、监管机构、制药行业和TED患者的共同利益制定必要的指标。
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来源期刊
JAMA ophthalmology
JAMA ophthalmology OPHTHALMOLOGY-
CiteScore
13.20
自引率
3.70%
发文量
340
期刊介绍: JAMA Ophthalmology, with a rich history of continuous publication since 1869, stands as a distinguished international, peer-reviewed journal dedicated to ophthalmology and visual science. In 2019, the journal proudly commemorated 150 years of uninterrupted service to the field. As a member of the esteemed JAMA Network, a consortium renowned for its peer-reviewed general medical and specialty publications, JAMA Ophthalmology upholds the highest standards of excellence in disseminating cutting-edge research and insights. Join us in celebrating our legacy and advancing the frontiers of ophthalmology and visual science.
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