[综述]
甲状腺相关性眼病(TAO)是一种病因及发病机制复杂，可引起多种眼部表现甚至威胁视力的自身免疫性疾病，临床上常将糖皮质激素作为一线治疗药物，但有部分患者对其产生了耐药。目前认为胰岛素样生长因子-1受体(IGF-1R)在TAO的发生和发展中起关键作用，替妥木单抗是IGF-1R的单克隆抗体，可特异性结合IGF-1R并阻断其与IGF-1的α亚基结合从而发挥生物学效应，对于活动期中重度TAO，已有临床试验证实其在减少眼球突出(减少≥2 mm)、降低炎症活动度(临床活动性评分减少≥2分)方面有良好的疗效。替妥木单抗起效快，安全性高，大多数不良反应为轻度或中度。高血糖是目前可确定与替妥木单抗相关的不良反应，并可通过药物来控制。鉴于药物对胎儿的致畸性且可使炎症性肠病病情严重恶化，妊娠和炎症性肠病为替妥木单抗治疗的禁忌证。美国FDA已于2020年1月正式批准替妥木单抗用于活动期中重度TAO的治疗。本文就替妥木单抗治疗TAO的作用机制、有效性及安全性等临床研究进展进行综述。
Thyroid-associated ophthalmopathy (TAO) is an autoimmune disease of complex etiology and pathogenesis, which can cause a variety of ocular manifestations and even threaten vision.Glucocorticoids are often used as the first-line treatment in the clinic, but some patients become resistant to them.The insulin-like growth factor-1 receptor (IGF-1R) plays a key role in the development of TAO.Teprotumumab is an anti-IGF-1R monoclonal antibody that can specifically bind to the IGF-1R and block its binding to the α-subunit of IGF-1 to exert biological effects.Clinical trials have shown that teprotumumab has good efficacy in reducing proptosis (decrease≥2 mm) and inflammatory activity (CAS decrease≥2 points) of active moderate-to-severe TAO.Most side effects are mild or moderate.Hyperglycemia is currently an identifiable adverse event associated with teprotumumab that can be controlled with medication.Pregnancy and inflammatory bowel disease are contraindications for teprotumumab due to its teratogenicity to the fetus and its ability to severely exacerbate inflammatory bowel disease.The US FDA has officially approved teprotumumab for the treatment of active moderate-to-severe TAO in January 2020.This article reviews the progress of clinical trials on the mechanism, efficacy and safety of teprotumumab in the treatment of TAO.
梁从碧,陈长征. 替妥木单抗治疗甲状腺相关性眼病的研究进展[J]. 中华实验眼科杂志,2023,41(11)：1140-1144.
DOI:10.3760/cma.j.cn115989-20220526-00249版权归中华医学会所有。
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