Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy.

IF 16.4 1区化学 Q1 CHEMISTRY, MULTIDISCIPLINARY

Accounts of Chemical Research Pub Date : 2022-07-18 eCollection Date: 2022-01-01 DOI:10.2147/PROM.S357555

Jennifer Quinn, Kathleen A Delaney, Wing Yen Wong, Wolfgang Miesbach, Monika Bullinger

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引用次数: 2

Abstract

Purpose: The hemophilia-specific health-related quality of life (HRQOL) questionnaire (Haemo-QOL-A) is validated for detecting QOL changes following standard therapy for hemophilia A, but has not been rigorously evaluated after gene therapy. This post hoc analysis evaluated the psychometric properties of Haemo-QOL-A in adult people with severe hemophilia A (PWSHA) receiving valoctocogene roxaparvovec (AAV5-hFVIII-SQ) in 2 clinical trials (phase 1/2, NCT02576795; phase 3, NCT03370913).

Patients and methods: Adult PWSHA (factor VIII levels ≤1 IU/dL) received 1 AAV5-hFVIII-SQ infusion (6×10¹³ vg/kg). Participants were assessed using the Haemo-QOL-A and the EuroQOL (EQ)-5D-5L and visual analog scale (VAS) questionnaires pre- and post-infusion. Psychometric analyses included convergent and discriminant validity, internal consistency, and reliability. Clinically important difference (CID) was estimated using 3-point change in EQ-5D-5L VAS as anchor.

Results: Haemo-QOL-A data were analyzed from 7 (phase 1/2, 3-year follow-up) and 16 participants (phase 3, 26-week analysis). Change in Haemo-QOL-A Total Scores correlated with EQ-5D-5L VAS score change at 26 weeks (Pearson's correlation 0.77). At 26 weeks, increased Haemo-QOL-A Physical Functioning was associated with decreased EQ-5D-5L Pain and Discomfort and decreased Anxiety and Depression (Spearman's Rank correlations -0.73 and -0.62, respectively, P <0.01). Internal consistency analysis showed good reliability for all domains (Cronbach's alpha >0.7) except Treatment Concern (Cronbach's alpha = 0.31). Anchor-based CID estimates were met for Haemo-QOL-A Total Score (≥5.5) and domain scores (≥6) for Consequences of Bleeding, Physical Functioning, Role Functioning, and Worry.

Conclusion: Our preliminary results suggest that the Haemo-QOL-A is a valid, reliable instrument for HRQOL assessment in PWSHA undergoing gene therapy. Future research should be undertaken to confirm these findings in a larger number of participants.

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基因治疗的血友病A患者血液qol -A的心理测量学验证。

目的:血友病特异性健康相关生活质量(HRQOL)问卷(Haemo-QOL-A)用于检测血友病A标准治疗后生活质量的变化，但尚未对基因治疗后的生活质量进行严格评估。这项事后分析评估了在2项临床试验(1/2期，NCT02576795;第3期，NCT03370913)。患者和方法:成人PWSHA (factor VIII水平≤1 IU/dL)接受1次AAV5-hFVIII-SQ输注(6×1013 vg/kg)。参与者在输注前后分别使用Haemo-QOL-A和EuroQOL (EQ)- 5d - 5l以及视觉模拟量表(VAS)问卷进行评估。心理测量分析包括收敛效度、判别效度、内部一致性和信度。以EQ-5D-5L VAS的3点变化为锚点估计临床重要差异(CID)。结果:分析了7名参与者(1/2期，3年随访)和16名参与者(3期，26周分析)的Haemo-QOL-A数据。26周时Haemo-QOL-A总分变化与EQ-5D-5L VAS评分变化相关(Pearson相关系数0.77)。在26周时，除了治疗关注(Cronbach's alpha = 0.31)外，血液质量-质量- a生理功能的增加与EQ-5D-5L疼痛和不适的减少以及焦虑和抑郁的减少相关(Spearman's Rank相关性分别为-0.73和-0.62,P = 0.7)。基于锚定的CID估计符合Haemo-QOL-A总分(≥5.5)和出血后果、身体功能、角色功能和担忧的域评分(≥6)。结论:我们的初步结果表明，Haemo-QOL-A是一种有效、可靠的评估基因治疗的PWSHA患者HRQOL的工具。未来的研究应在更多的参与者中证实这些发现。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Accounts of Chemical Research 化学-化学综合

CiteScore

31.40

自引率

1.10%

发文量

312

审稿时长

2 months

期刊介绍： Accounts of Chemical Research presents short, concise and critical articles offering easy-to-read overviews of basic research and applications in all areas of chemistry and biochemistry. These short reviews focus on research from the author’s own laboratory and are designed to teach the reader about a research project. In addition, Accounts of Chemical Research publishes commentaries that give an informed opinion on a current research problem. Special Issues online are devoted to a single topic of unusual activity and significance. Accounts of Chemical Research replaces the traditional article abstract with an article "Conspectus." These entries synopsize the research affording the reader a closer look at the content and significance of an article. Through this provision of a more detailed description of the article contents, the Conspectus enhances the article's discoverability by search engines and the exposure for the research.