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Thyroid-associated ophthalmopathy: Emergence of teprotumumab as a promising medical therapy.
Best Practice & Research Clinical Endocrinology & Metabolism ( IF 6.1 ) Pub Date : 2020-01-31 , DOI: 10.1016/j.beem.2020.101383
Terry J Smith 1
Affiliation  

Thyroid-associated ophthalmopathy (TAO) remains a vexing autoimmune component of Graves’ disease that can diminish the quality of life as a consequence of its impact on visual function, physical appearance and emotional well-being. Because of its relative rarity and variable presentation, the development of highly effective and well-tolerated medical therapies for TAO has been slow relative to other autoimmune diseases. Contributing to the barriers of greater insight into TAO has been the historical absence of high-fidelity preclinical animal models. Despite these challenges, several agents, most developed for treatment of other diseases, have found their way into consideration for use in active TAO through repurposing. Among these, teprotumumab is a fully human inhibitory monoclonal antibody against the insulin-like growth factor I receptor. It has shown remarkable effectiveness in moderate to severe, active TAO in two completed multicenter, double masked, and placebo controlled clinical trials. The drug exhibits a favorable safety profile. Teprotumumab has recently been approved by the U.S. F.D.A, and may rapidly become the first line therapy for this disfiguring and potentially blinding condition.



中文翻译:


甲状腺相关眼病:teprotumumab 作为一种有前景的药物疗法的出现。



甲状腺相关眼病 (TAO) 仍然是格雷夫斯病中令人烦恼的自身免疫性疾病,由于其对视觉功能、身体外观和情绪健康的影响,可能会降低生活质量。由于 TAO 相对罕见且表现多样,与其他自身免疫性疾病相比,针对 TAO 的高效且耐受性良好的药物疗法的开发进展缓慢。历史上缺乏高保真度的临床前动物模型,阻碍了对 TAO 的深入了解。尽管存在这些挑战,一些药物(大多数是为治疗其他疾病而开发的)已经通过重新利用找到了用于活性 TAO 的方法。其中,teprotumumab是一种针对胰岛素样生长因子I受体的全人源抑制性单克隆抗体。在两项已完成的多中心、双盲和安慰剂对照临床试验中,它在中度至重度活动性 TAO 中显示出显着的疗效。该药物表现出良好的安全性。 Teprotumumab 最近已获得美国 FDA 批准,并可能迅速成为治疗这种毁容和潜在致盲疾病的一线疗法。

更新日期:2020-01-31
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