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特罗特鲁单抗在甲状腺眼病中的应用:灵丹妙药还是巨大分歧?

Teprotumumab in thyroid eye disease: wonder drug or great divider?

机构信息

Department of Endocrinology, Royal Victoria Infirmary, Newcastle upon Tyne, UK.

Department of Endocrinology Odense University Hospital, Odense, Denmark.

出版信息

Eur Thyroid J. 2023 Jun 12;12(4). doi: 10.1530/ETJ-23-0043. Print 2023 Aug 1.

Abstract

Progress in the management of thyroid eye disease (TED) has been slow for many decades. The recent introduction of teprotumumab (TEP) in the therapeutic arena for TED has had a major impact in view of its efficacy, particularly with respect to its ability to reduce proptosis. However, the high cost of TEP, limited availability to patients outside the USA, and the lack of data on cost-effectiveness are significant barriers to improving the care of patients with TED globally. Recent guidance from authoritative professional organisations deliver different perspectives on the role of TEP in the routine management of patients with TED, underscoring the complexities of interpreting the evidence. The advance that TEP undoubtedly represents in managing TED effectively has highlighted inequities faced by patients and uncertainties about appropriate metrics of efficacy. Professional organisations have an important role addressing these problems. Future studies need to focus on optimising the measurement of outcomes and on assessing cost-effectiveness.

摘要

甲状腺眼病(TED)的管理进展缓慢已有数十年。鉴于其疗效,尤其是其减少眼球突出的能力,teprotumumab(TEP)在 TED 的治疗领域的引入产生了重大影响。然而,TEP 的高成本、美国以外患者的有限可用性以及关于成本效益的数据缺乏,是改善全球 TED 患者护理的重大障碍。权威专业组织的最新指南对 TEP 在 TED 患者常规管理中的作用提出了不同的观点,突出了解释证据的复杂性。TEP 无疑在 TED 的有效管理方面代表了一个进步,这凸显了患者面临的不平等和对疗效适当衡量标准的不确定性。专业组织在解决这些问题方面发挥着重要作用。未来的研究需要集中优化结果的测量,并评估成本效益。

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