Department of Ophthalmology and Visual Sciences and Department of Internal Medicine, Kellogg Eye Center-Michigan Medicine and University of Michigan, Ann Arbor, Michigan.
Senta Clinic, San Diego, California.
Endocr Pract. 2024 May;30(5):470-475. doi: 10.1016/j.eprac.2024.02.002. Epub 2024 Feb 8.
In thyroid eye disease (TED), inflammation and expansion of orbital muscle and periorbital fat result in diplopia and proptosis, severely impacting patient quality of life (QOL). The reported health state utility (HSU) scores, which are QOL measures, allow quantification of TED impact and improvement with therapies; however, no current QOL instrument has been validated with HSU scores for TED. Here, we used the disease-specific Graves Ophthalmopathy Quality of Life (GO-QOL) questionnaire and HSU scores to validate QOL impact.
The GO-QOL scores from patients in 2 randomized, masked, placebo-controlled teprotumumab trials (N=171) were compared with 6 HSU values based on severity of proptosis/diplopia in those studies. Patient GO-QOL and HSU scores were compared at baseline and after 6-month treatment via regression analyses. GO-QOL and HSU scores were correlated for validation and quantification of QOL impact by severity state and to estimate quality-adjusted life year improvement.
GO-QOL scores were correlated with TED severity, indicating that worse severity was associated with lower (worse) GO-QOL scores. Less severe health states were represented by higher (better) GO-QOL scores. Importantly, GO-QOL scores were positively correlated with utility scores of the 6 health states, allowing for conversion of the GO-QOL scores to utility scores. A positive (improved) 0.013 utility change was found for each 1-point (positive) improvement in GO-QOL score produced by teprotumumab versus placebo.
Patients with moderate-to-severe active TED health states demonstrate increasing TED severity associated with declining utility values and worsening GO-QOL scores. These results indicate that the GO-QOL scores can be used to bridge to the HSU scores for benefit quantification.
在甲状腺眼病（TED）中，眼眶肌肉和眶周脂肪的炎症和扩张导致复视和眼球突出，严重影响患者的生活质量（QOL）。报告的健康状态效用（HSU）评分是 QOL 衡量标准，可以量化 TED 的影响以及治疗效果的改善；然而，目前尚无针对 TED 的 QOL 工具与 HSU 评分相关联。在这里，我们使用了专门针对 Graves 眼病生活质量（GO-QOL）问卷和 HSU 评分来验证 QOL 的影响。
比较了 2 项随机、双盲、安慰剂对照的 teprotumumab 试验（N=171）中患者的 GO-QOL 评分与这些研究中基于眼球突出/复视严重程度的 6 个 HSU 值。通过回归分析比较了患者的基线和 6 个月治疗后的 GO-QOL 和 HSU 评分。为了验证和量化 QOL 影响的严重程度状态，并估计质量调整生命年的改善，对 GO-QOL 和 HSU 评分进行了相关性分析。
GO-QOL 评分与 TED 严重程度相关，表明严重程度越差与 GO-QOL 评分越低（越差）相关。较轻的健康状态由较高（较好）的 GO-QOL 评分表示。重要的是，GO-QOL 评分与 6 种健康状态的效用评分呈正相关，这使得可以将 GO-QOL 评分转换为效用评分。与安慰剂相比，teprotumumab 每使 GO-QOL 评分提高 1 分（阳性），效用变化就会产生正（改善）0.013 个单位。
患有中度至重度活动性 TED 的患者，随着 TED 严重程度的增加，与效用值降低和 GO-QOL 评分恶化相关联。这些结果表明，GO-QOL 评分可用于与 HSU 评分相衔接，以量化获益。
