Lin Chia-Hung, Wei Yi-Hsuan, Lu Jin-Ying, Li Hung-Yuan, Lee Chung-Wei, Yang Chung-Yi, Chang Chin-Hao, Wu Wan-Chen, Wang Chih-Yuan, Shih Shyang-Rong
Division of Endocrinology and Metabolism, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.
Department of Ophthalmology, National Taiwan University Hospital, Taipei, Taiwan.
Trials. 2025 Aug 4;26(1):272. doi: 10.1186/s13063-025-09002-6.
Graves' orbitopathy (GO) is a prevalent manifestation of Graves' disease (GD), characterized by proptosis, eyelid retraction, soft tissue swelling, diplopia, and potential visual acuity impairment. Furthermore, even mild GO can significantly impact mental health and overall quality of life for patients with GD. Despite its severity, available medical treatments for mild GO are limited. Recent basic studies on orbital fibroblasts suggest hydroxychloroquine (HCQ) as a promising therapeutic agent for GO patients. This randomized controlled trial (RCT) was designed to assess the efficacy of HCQ in treating mild GO. METHODS: This multi-center open-label RCT will be conducted in Taiwan with a total of 108 participants randomized into HCQ and control groups at 2:1 allocation ratio. The primary endpoint of this study is a composite outcome of ophthalmic parameters including eyelid aperture, soft tissue involvement, and exophthalmos. Secondary endpoints comprise changes in quality of life (GO-QoL), orbital volumetry via computed tomography (CT), diplopia scores, clinical activity scores (CAS), visual acuity, and thyroid autoantibodies.
This RCT will elucidate the clinical benefits of oral HCQ in patients with mild GO, assessing ophthalmic outcomes, quality of life, disease activity, and thyroid autoantibodies. In addition, data obtained from orbital CT measurements will provide valuable insights into subtle changes in orbital fat and extra-ocular muscle volumes, potentially offering an objective tool for monitoring GO progression. TRIAL REGISTRATION {2A, 2B}: ClinicalTrials.gov NCT05126147. Registered on November 2021. https://clinicaltrials.gov/study/NCT05126147 . All items from the World Health Organization (WHO) Trial Registration Data Set are addressed within the relevant sections of this protocol.
格雷夫斯眼眶病（GO）是格雷夫斯病（GD）的一种常见表现，其特征为眼球突出、眼睑退缩、软组织肿胀、复视以及潜在的视力损害。此外，即使是轻度的GO也会对GD患者的心理健康和整体生活质量产生重大影响。尽管病情严重，但针对轻度GO的现有药物治疗方法有限。最近关于眼眶成纤维细胞的基础研究表明，羟氯喹（HCQ）是一种有前景的GO治疗药物。本随机对照试验（RCT）旨在评估HCQ治疗轻度GO的疗效。
本多中心开放标签RCT将在台湾进行，共有108名参与者，按照2:1的分配比例随机分为HCQ组和对照组。本研究的主要终点是包括睑裂、软组织受累和眼球突出在内的眼科参数的综合结果。次要终点包括生活质量（GO-QoL）的变化、通过计算机断层扫描（CT）测量的眼眶容积、复视评分、临床活动评分（CAS）、视力和甲状腺自身抗体。
本RCT将阐明口服HCQ对轻度GO患者的临床益处，评估眼科结局、生活质量、疾病活动度和甲状腺自身抗体。此外，从眼眶CT测量获得的数据将为眼眶脂肪和眼外肌体积的细微变化提供有价值的见解，可能为监测GO进展提供一种客观工具。
试验注册{2A, 2B}：ClinicalTrials.gov NCT05126147。于2021年11月注册。https://clinicaltrials.gov/study/NCT05126147 。本方案的相关部分涵盖了世界卫生组织（WHO）试验注册数据集的所有项目。
