Department for Neurobiology, Care Sciences and Society, Division of Neurogeriatrics, Karolinska Institutet, Solna, Sweden.
H. Lundbeck A/S, Copenhagen, Denmark.
J Headache Pain. 2024 Mar 28;25(1):45. doi: 10.1186/s10194-024-01752-z.
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 ).
随着新的偏头痛治疗方法的出现，衡量健康相关生活质量 (HRQoL) 改善的措施至关重要，而不仅仅是每月偏头痛天数 (MMD) 减少的改善。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eptinezumab 治疗的患者典型症状改善和患者确定的最困扰症状 (PI-MBS) 改善的研究。这项探索性分析确定了在偏头痛患者中，衡量eptinezumab 对 HRQoL 改善效果的疗效指标。
来自 2-4 次预防偏头痛治疗失败的患者的 DELIVER 研究的数据（NCT04418765）被输入到两个结构方程模型中，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。定义了一个单一的潜在变量来代表 HRQoL，并描述 DELIVER 中的 HRQoL 来源。一个模型包含所有偏头痛症状，而第二个模型仅包含 PI-MBS。捕获不同疗效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%（标准化效应大小[SES] -0.11）和 25%（SES -0.08），41%（SES -0.13）的改善由“直接治疗效果”组成，即不受中介物解释。在第二个模型中，eptinezumab 可显著改善 HRQoL（86%；SES -0.26），这是由于 MMD 减少（17%；SES -0.05）和 PI-MBS 变化（69%；SES -0.21）所致。
接受eptinezumab 治疗的患者的 HRQoL 改善可以通过其对偏头痛频率和 PI-MBS 的影响得到很好的解释。因此，除了 MMD 减少之外，医疗保健提供者还应该讨论 PI-MBS 的改善，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对经济评估的影响，因为它们指出了替代质量调整生命年的测量方法，以在成本效益分析中充分捕捉治疗益处。
ClinicalTrials.gov（标识符：NCT04418765；EudraCT（标识符：2019-004497-25；网址：https://www.clinicaltrialsregister.eu/ctr-search/search?query=2019-004497-25）。
