Patient-Centred Research, Evidera, Bethesda, MD, USA.
Global Patient Outcomes and Real World Evidence (GPORWE), Eli Lilly and Company, Indianapolis, IN, USA.
Headache. 2021 Mar;61(3):511-526. doi: 10.1111/head.14031. Epub 2021 Jan 22.
To evaluate the measurement properties of all three domains of the Migraine-Specific Quality of Life questionnaire version 2.1 (MSQ v2.1) electronic patient-reported outcome (ePRO) to assess the functional impact of migraine in patients with episodic or chronic migraine (CM); and identify meaningful within-patient change thresholds for the Role Function-Restrictive (RFR), Role Function-Preventive (RFP), and Emotional Function (EF) domains.
Data were drawn from three double-blind, placebo-controlled, and randomized Phase 3 clinical studies (episodic migraine [EM]: EVOLVE-1 and EVOLVE-2; CM: REGAIN). The psychometric properties of the MSQ v2.1 ePRO domains were demonstrated by evaluating reliability (internal consistency and test-retest), construct validity (convergent and known groups), and responsiveness. Meaningful within-patient change thresholds for domains were estimated using anchor-based approaches, supplemented by empirical cumulative distribution function curves and probability density function plots to enable interpretation of meaningful change over 3 months. The Patient Global Impression of Severity (PGI-S) and Patient Global Impression of Improvement served as anchors.
A total of 2,850 patients with either EM (EVOLVE-1: 851; EVOLVE-2: 909) or CM (REGAIN: 1,090) were included. The Cronbach's alpha estimates of internal consistency exceeded the recommended threshold of ≥0.70 for all domains from the three studies, indicating adequate internal consistency. Test-retest reliability intraclass correlation coefficients were ≥0.80 for all domains across all three studies, demonstrating almost perfect agreement. Convergent validity was supported by moderate-to-strong correlation (r ≥ 0.30) between all domains of MSQ v2.1 ePRO and studied anchors (Migraine Disability Assessment Score and PGI-S scores) across all three studies. Known group validity was established between all domains and subgroups of patients stratified by baseline PGI-S scores and baseline number of monthly migraine headache days for all three studies. The 3-month meaningful within-patient change thresholds were the same for EM and CM for RFP: 20.00 and EF: 26.67; and for RFR: 25.71.
These findings demonstrate that all three domains of the MSQ v2.1 ePRO have sufficient reliability, validity, responsiveness, and appropriate interpretation standards. Our results suggest that MSQ v2.1 ePRO is a well-defined and reliable patient-reported outcome instrument that is suitable for use in clinical studies for evaluating the impact of migraine on patient functioning in episodic and CM.
评估偏头痛特异性生活质量问卷 2.1 版（MSQ v2.1）电子患者报告结局（ePRO）所有三个领域的测量特性，以评估发作性或慢性偏头痛（CM）患者偏头痛对功能的影响；并为角色功能受限（RFR）、角色功能预防（RFP）和情绪功能（EF）领域确定有意义的患者内变化阈值。
数据来自三项双盲、安慰剂对照和随机 3 期临床研究（发作性偏头痛 [EM]：EVOLVE-1 和 EVOLVE-2；CM：REGAIN）。通过评估可靠性（内部一致性和测试-重测）、结构有效性（收敛和已知组）和反应性，证明了 MSQ v2.1 ePRO 领域的心理测量特性。使用基于锚定的方法估计各领域的患者内有意义的变化阈值，并补充经验累积分布函数曲线和概率密度函数图，以解释 3 个月内的有意义变化。患者总体严重程度印象（PGI-S）和患者总体改善印象作为锚定。
共纳入 2850 例 EM 患者（EVOLVE-1：851 例；EVOLVE-2：909 例）或 CM 患者（REGAIN：1090 例）。所有三个研究中，所有领域的内部一致性 Cronbach's alpha 估计值均超过了≥0.70 的推荐阈值，表明内部一致性良好。所有三个研究中，所有领域的测试-重测可靠性组内相关系数均≥0.80，表明几乎完全一致。在所有三个研究中，MSQ v2.1 ePRO 的所有领域与研究锚定（偏头痛残疾评估量表和 PGI-S 评分）之间的相关性均为中度至高度（r≥0.30），支持了收敛有效性。在所有三个研究中，在基于基线 PGI-S 评分和基线每月偏头痛头痛天数对患者进行分层的情况下，均在所有领域与患者亚组之间建立了已知的组间有效性。对于 RFP：20.00 和 EF：26.67，以及 RFR：25.71，EM 和 CM 的有意义的患者内 3 个月变化阈值相同。
这些发现表明，MSQ v2.1 ePRO 的所有三个领域均具有足够的可靠性、有效性、反应性和适当的解释标准。我们的研究结果表明，MSQ v2.1 ePRO 是一种定义明确且可靠的患者报告结局工具，适用于评估偏头痛对发作性和 CM 患者功能的影响的临床试验。
