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峰值摄氧量在梗阻性肥厚型心肌病中的预后价值:文献综述以支持经济模型的开发。

The prognostic value of peak oxygen uptake in obstructive hypertrophic cardiomyopathy: a literature review to inform economic model development.

机构信息

Cytokinetics, Inc, South San Francisco, CA, USA.

Maple Health Group, New York, NY, USA.

出版信息

J Med Econ. 2024 Jan-Dec;27(1):817-825. doi: 10.1080/13696998.2024.2367920. Epub 2024 Jul 1.

Abstract

AIMS

Patients with obstructive hypertrophic cardiomyopathy (oHCM) experience significant clinical burden which is associated with a high economic burden. Peak oxygen uptake (pVO2), measured by cardiopulmonary exercise testing, is used to quantify functional capacity, and has been studied as a primary endpoint in recent clinical trials. This study aimed to gather evidence to consolidate the prognostic value of pVO2 in oHCM and to assess whether it is feasible to predict health outcomes in an economic model based on changes in pVO2.

METHODS

A targeted literature review was conducted in MEDLINE ( PubMed) and Embase databases to identify evidence on the prognostic value of pVO2 as a surrogate health outcome to support future oHCM economic model development. Following screening, study characteristics, population characteristics, and pVO2 prognostic association data were extracted.

RESULTS

A total of 4,687 studies were identified. In total, 3,531 and 538 studies underwent title/abstract and full-text screening, respectively, of which 151 were included and nine of these were in hypertrophic cardiomyopathy (HCM); only three studies focused on oHCM. The nine HCM studies consisted of one systematic literature review and eight primary studies reporting on 27 potentially predictive relationships from a pVO2-based metric with clinical outcomes including all-cause mortality, cardiovascular mortality, sudden cardiac death, transplant, paroxysmal, and permanent atrial fibrillation. pVO2 was described as a predictor of single and composite endpoints, in three and six studies, respectively, with one study reporting on both.

LIMITATIONS

This study primarily uses systemic literature review methods but does not qualify as one due to not entailing parallel reviewers during title-abstract and full-text stages of review.

CONCLUSION

The findings of this study suggest pVO2 is predictive of multiple health outcomes, providing a rationale to use pVO2 in the development of an economic model.

摘要

目的

梗阻性肥厚型心肌病(oHCM)患者的临床负担较重,且经济负担高。心肺运动试验(CPET)测量的峰值摄氧量(pVO2)用于量化功能能力,并且已经在最近的临床试验中作为主要终点进行了研究。本研究旨在收集证据,以整合 pVO2 在 oHCM 中的预后价值,并评估其是否可基于 pVO2 的变化在经济模型中预测健康结果。

方法

在 MEDLINE(PubMed)和 Embase 数据库中进行了有针对性的文献综述,以确定 pVO2 作为替代健康结果的预后价值的证据,以支持未来 oHCM 经济模型的开发。经过筛选,提取了研究特征、人群特征和 pVO2 预后关联数据。

结果

共确定了 4687 项研究。共有 3531 项和 538 项研究分别进行了标题/摘要和全文筛选,其中 151 项被纳入,其中 9 项是肥厚型心肌病(HCM);只有 3 项研究专注于梗阻性肥厚型心肌病。这 9 项 HCM 研究包括 1 项系统综述和 8 项原发性研究,报告了基于 pVO2 的指标与临床结局(包括全因死亡率、心血管死亡率、心脏性猝死、移植、阵发性和永久性心房颤动)之间的 27 个潜在预测关系。pVO2 被描述为单个和复合终点的预测因子,分别在 3 项和 6 项研究中,其中 1 项研究报告了两者。

局限性

本研究主要使用系统综述方法,但由于在标题-摘要和全文审查阶段没有并行审查人员,因此不能算作其中之一。

结论

本研究的结果表明,pVO2 可预测多种健康结果,为在经济模型开发中使用 pVO2 提供了依据。

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