Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials

IF 4 3区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Advances in Therapy Pub Date : 2025-01-23 DOI:10.1007/s12325-024-03095-2

Khashayar Azimpour, Patricia Dorling, Irene Koulinska, Swati Kunduri, Zhiyi Lan, Julia Poritz, Gabriel Tremblay, Angie Raad-Faherty

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Abstract

Introduction

Fabry disease (FD) is a rare lysosomal storage disorder that is associated with pain and progressive damage to the renal, cardiac, and cerebrovascular systems. Enzyme replacement therapy (ERT) is one of the treatment options for FD and the most recently approved ERT agent, pegunigalsidase alfa, has shown clinical efficacy in three phase 3 clinical trials of adults with FD: BALANCE, BRIDGE, and BRIGHT. Recent published guidelines support the mapping of health utility state data to the EuroQol-5 Dimension-3 Level (EQ-5D-3L) index to align with the preferred methodology used by the National Institute for Health and Care Excellence (NICE). Therefore, the primary objective of this study was to estimate EQ-5D-3L values in clinical trials of pegunigalsidase alfa for FD for future cost–utility analyses.

Methods

A mixed effects model was developed to predict values derived from EQ-5D-3L for the following health states used in cost–utility analyses: no Fabry clinical event (FCE)/no pain-related adverse event (AE), pain-related AE, cardiac FCE, cerebrovascular FCE, and renal FCE.

Results

The baseline EQ-5D-3L utility value had a statistically significant (p < 0.0001) impact on utility values, whereas study, age, sex, disease type, treatment arm, kidney function, and serious AE were not statistically significant. Health state utility values with 95% confidence intervals (CI) for the final model were as follows: no FCE/no pain-related AE, 0.8005 (0.7675, 0.8334); pain-related AE, 0.7737 (0.7262, 0.8211); cardiac FCE, 0.7189 (0.6274, 0.8103); cerebrovascular FCE, 0.7923 (0.6633, 0.9212); and renal FCE, 0.6881 (0.3887, 0.9874).

Conclusions

The utility values generated by the present study are generally in line with EQ-5D values in the FD literature and can be used to inform both economic evaluations and our understanding of the impact that FD has on quality of life.

Trial Registration

NCT03018730, NCT02795676, NCT03180840.

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法布里病的健康状态效用价值：来自Pegunigalsidase α临床试验的见解。

简介：法布里病（FD）是一种罕见的溶酶体积存疾病，与疼痛和肾脏、心脏和脑血管系统的进行性损害有关。酶替代疗法（ERT）是FD的治疗选择之一，最近批准的ERT药物pegunigalsidase alfa在三个成人FD的3期临床试验中显示出临床疗效：BALANCE， BRIDGE和BRIGHT。最近发布的指南支持将卫生公用事业状态数据映射到EuroQol-5维度-3级别（EQ-5D-3L）指数，以与国家卫生和护理卓越研究所（NICE）使用的首选方法保持一致。因此，本研究的主要目的是估计pegunigalsidase alfa用于FD的临床试验中的EQ-5D-3L值，以用于未来的成本-效用分析。方法：建立了一个混合效应模型来预测EQ-5D-3L得出的用于成本-效用分析的以下健康状态的值：无Fabry临床事件(FCE)/无疼痛相关不良事件（AE）、疼痛相关AE、心脏FCE、脑血管FCE和肾脏FCE。结果：基线EQ-5D- 3l效用值具有统计学意义(p)。结论：本研究产生的效用值与FD文献中的EQ-5D值基本一致，可用于告知经济评估和我们对FD对生活质量影响的理解。试验注册号：NCT03018730、NCT02795676、NCT03180840。

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

Advances in Therapy 医学-药学

CiteScore

7.20

自引率

2.60%

发文量

353

审稿时长

6-12 weeks

期刊介绍： Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.