As new migraine therapies emerge, it is crucial for measures to capture the complexities of health-related quality of life (HRQoL) improvement beyond improvements in monthly migraine day (MMD) reduction. Investigations into the correlations between MMD reduction, symptom management, and HRQoL are lacking, particularly those that focus on improvements in canonical symptoms and improvement in patient-identified most-bothersome symptoms (PI-MBS), in patients treated with eptinezumab. This exploratory analysis identified efficacy measures mediating the effect of eptinezumab on HRQoL improvements in patients with migraine. Data from the DELIVER study of patients with 2–4 prior preventive migraine treatment failures (NCT04418765) were inputted to two structural equation models describing sources of HRQoL improvement via Migraine-Specific Quality-of-Life Questionnaire (MSQ) scores. A single latent variable was defined to represent HRQoL and describe the sources of HRQoL in DELIVER. One model included all migraine symptoms while the second model included the PI-MBS as the only migraine symptom. Mediating variables capturing different aspects of efficacy included MMDs, other canonical symptoms, and PI-MBS. In the first model, reductions in MMDs and other canonical symptoms accounted for 35% (standardized effect size [SES] − 0.11) and 25% (SES − 0.08) of HRQoL improvement, respectively, with 41% (SES − 0.13) of improvement comprising “direct treatment effect,” i.e., unexplained by mediators. In the second model, substantial HRQoL improvement with eptinezumab (86%; SES − 0.26) is due to MMD reduction (17%; SES − 0.05) and change in PI-MBS (69%; SES − 0.21). Improvements in HRQoL experienced by patients treated with eptinezumab can be substantially explained by its effect on migraine frequency and PI-MBS. Therefore, in addition to MMD reduction, healthcare providers should discuss PI-MBS improvements, since this may impact HRQoL. Health technology policymakers should consider implications of these findings in economic evaluation, as they point to alternative measurement of quality-adjusted life years to capture fully treatment benefits in cost-utility analyses. ClinicalTrials.gov (Identifier: NCT04418765 ; EudraCT (Identifier: 2019–004497-25; URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=2019-004497-25 ).

中文翻译：

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结构方程模型用于确定接受依替珠单抗治疗的偏头痛患者健康相关生活质量改善的驱动因素

随着新的偏头痛疗法的出现，除了每月偏头痛日数 (MMD) 减少的改善之外，衡量与健康相关的生活质量 (HRQoL) 改善的复杂性的措施也至关重要。目前缺乏对 MMD 减少、症状管理和 HRQoL 之间相关性的研究，特别是那些关注接受依替珠单抗治疗的患者典型症状改善和患者确定的最烦人症状 (PI-MBS) 改善的研究。这项探索性分析确定了调节 eptinezumab 对偏头痛患者 HRQoL 改善影响的疗效指标。来自 DELIVER 研究的数据被输入到两个结构方程模型中，该研究对既往有 2-4 次预防性偏头痛治疗失败的患者 (NCT04418765) 进行输入，通过偏头痛特异性生活质量问卷 (MSQ) 评分描述 HRQoL 改善的来源。定义单个潜在变量来表示 HRQoL 并描述 DELIVER 中 HRQoL 的来源。一个模型包含所有偏头痛症状，而第二个模型包含 PI-MBS 作为唯一的偏头痛症状。捕捉功效不同方面的中介变量包括 MMD、其他典型症状和 PI-MBS。在第一个模型中，MMD 和其他典型症状的减少分别占 HRQoL 改善的 35%（标准化效应量 [SES]−0.11）和 25%（SES−0.08），改善了 41%（SES−0.13）包括“直接治疗效果”，即调解者无法解释的效果。在第二个模型中，eptinezumab 的 HRQoL 显着改善（86%；SES - 0.26）是由于 MMD 降低（17%；SES - 0.05）和 PI-MBS 的变化（69%；SES - 0.21）。接受 eptinezumab 治疗的患者 HRQoL 的改善基本上可以通过其对偏头痛频率和 PI-MBS 的影响来解释。因此，除了减少 MMD 之外，医疗保健提供者还应该讨论 PI-MBS 的改进，因为这可能会影响 HRQoL。卫生技术政策制定者应考虑这些发现在经济评估中的影响，因为它们指出了质量调整生命年的替代衡量方法，以在成本效用分析中充分捕捉治疗效益。 ClinicalTrials.gov（标识符：NCT04418765；EudraCT（标识符：2019-004497-25；URL：https://www.clinicaltrialsregister.eu/ctr-search/search?query=2019-004497-25）。