Kahaly George J, Xi Ann, Barretto Naina, Patel Haridarshan, Qashqai Anahita, Shokoohi Mostafa, Spin Paul, Holt Robert J
Department of Medicine I, Johannes Gutenberg University Medical Center, Mainz 55101, Germany.
Amgen Inc., Thousand Oaks, CA 91320-1789, USA.
J Endocr Soc. 2025 Apr 8;9(6):bvaf063. doi: 10.1210/jendso/bvaf063. eCollection 2025 Jun.
Teprotumumab and/or intravenous methylprednisolone (IVMP) are first-line treatments for thyroid eye disease (TED)/Graves' orbitopathy. Direct comparisons of these treatments on Graves' Orbitopathy Quality of Life Questionnaire (GO-QoL) are lacking.
Systematic review, meta-analysis, and matching-adjusted indirect treatment comparison (MAIC) of GO-QoL improvements.
A literature search was performed using PubMed and Embase between inception to April 1, 2023, including studies of patients with moderate to severe active TED treated with teprotumumab or IVMP.Changes in GO-QoL scores and appearance and visual function subscales were extracted. Two teprotumumab (n = 84) and placebo (n = 87) randomized clinical trials and 5 studies with IVMP (4.5 mg/12 weeks, n = 304) were identified. MAIC compared teprotumumab/placebo by adjusting for baseline GO-QoL, diplopia, proptosis, smoking, age, and sex. Significant improvements in overall GO-QoL were observed for teprotumumab vs IVMP [mean difference (MD): 13.26; 95% confidence interval (CI): 7.44, 19.09) and placebo (MD: 12.57; 95% CI: 5.94, 19.21), but not IVMP vs placebo (MD: -2.06; 95% CI: -8.24, 4.12). Improvements were noted in appearance subscale GO-QoL for teprotumumab vs IVMP (MD: 7.50; 95% CI: 0.35, 14.64) and placebo (MD: 10.80; 95% CI: 3.62, 17.98) but not for IVMP vs placebo (MD: 0.91; 95% CI: -6.36, 8.18). Visual subscale GO-QoL displayed greater improvements for teprotumumab vs IVMP (MD: 17.66; 95% CI: 7.86, 27.47) and placebo (MD: 14.54; 95% CI: 6.10, 22.99), with no significant change for IVMP vs placebo (MD: -3.65; 95% CI: -12.88, 5.57). Orbital pain, teprotumumab treatment, diplopia, proptosis, and male sex were significant independent predictors of overall GO-QoL ( < .05).
This unique analysis demonstrated a more clinically meaningful improvement in the health-related quality of life, as measured by the GO-QoL, for TED patients with teprotumumab vs both the most recommended IVMP dosage and placebo.
替普罗单抗和/或静脉注射甲泼尼龙（IVMP）是甲状腺眼病（TED）/格雷夫斯眼眶病的一线治疗方法。目前缺乏对这些治疗方法在格雷夫斯眼眶病生活质量问卷（GO-QoL）方面的直接比较。
对GO-QoL改善情况进行系统评价、荟萃分析和匹配调整间接治疗比较（MAIC）。
使用PubMed和Embase从数据库建立至2023年4月1日进行文献检索，纳入接受替普罗单抗或IVMP治疗的中度至重度活动性TED患者的研究。提取GO-QoL评分以及外观和视觉功能子量表的变化。确定了两项替普罗单抗（n = 84）和安慰剂（n = 87）的随机临床试验以及5项IVMP（4.5mg/12周，n = 304）的研究。MAIC通过调整基线GO-QoL、复视、眼球突出、吸烟、年龄和性别来比较替普罗单抗/安慰剂。观察到替普罗单抗与IVMP相比总体GO-QoL有显著改善[平均差异（MD）：13.26；95%置信区间（CI）：7.44，19.09]以及与安慰剂相比（MD：12.57；95%CI：5.94，19.21），但IVMP与安慰剂相比无显著差异（MD：-2.06；95%CI：-8.24，4.12）。在外观子量表GO-QoL方面，替普罗单抗与IVMP相比（MD：7.50；95%CI：0.35，14.64）以及与安慰剂相比（MD：10.80；95%CI：3.62，17.98）有改善，但IVMP与安慰剂相比无改善（MD：0.91；95%CI：-6.36，8.18）。视觉子量表GO-QoL方面，替普罗单抗与IVMP相比（MD：17.66；95%CI：7.86，27.47）以及与安慰剂相比（MD：14.54；95%CI：6.10，22.99）有更大改善，IVMP与安慰剂相比无显著变化（MD：-3.65；95%CI：-12.88，5.57）。眼眶疼痛、替普罗单抗治疗、复视、眼球突出和男性是总体GO-QoL的显著独立预测因素（P <.05）。
这项独特的分析表明，对于TED患者，以GO-QoL衡量，与最推荐的IVMP剂量和安慰剂相比，替普罗单抗在健康相关生活质量方面有更具临床意义的改善。
