Sections of Critical Care and Cardiovascular Medicine, Wake Forest School of Medicine, Winston-Salem, North Carolina, USA.
Respiratory Institute, Cleveland Clinic, Cleveland, Ohio, USA.
JACC Heart Fail. 2024 Feb;12(2):366-376. doi: 10.1016/j.jchf.2023.09.008. Epub 2023 Oct 25.
Despite the greater sensitivity and specificity of disease-specific patient-reported outcome measures (PROM) to detect clinical change, only recently have such instruments been developed for pulmonary hypertension (PH), specifically pulmonary arterial hypertension (PAH) and chronic thromboembolic disease (CTEPH). Although these valuable tools are now being incorporated into clinical studies of PH, they have not yet reached widespread integration into routine clinical care.
In this systematic review, the authors assess the psychometric properties of PROM developed for PH, compare PROM with other clinical outcomes in PH, and address the utility of PROM in clinical care.
The authors performed a systematic search of papers published between January 1, 2006, and October 1, 2022, using the MEDLINE database to identify PROM developed and validated for PH. The identified PROM were found to have been developed only in groups with PAH and CTEPH. The authors evaluated the identified instruments according to established psychometric criteria. An additional search was performed to identify randomized controlled trials (RCTs) utilizing these PROM for comparison with clinical outcomes.
From 527 papers retrieved, a total of 35 PROM were identified. Of these, 5 disease-specific instruments were included in the final analysis. While both CAMPHOR (Cambridge Pulmonary Hypertension Outcome Review) and emPHasis-10 performed well in patients with PAH and CTEPH with regard to their psychometric properties, emPHasis-10 demonstrated superior feasibility for use in clinical practice due to its concise format. The Pulmonary Arterial Hypertension-Symptoms and Impacts Questionnaire performed well in the authors' analysis, though additional data is needed regarding interpretability and feasibility.
EmPHasis-10 demonstrated strong psychometric properties and the greatest feasibility for clinical use. Further study assessing the integration of PROM into routine clinical care for PH is needed.
尽管针对特定疾病的患者报告结局测量（PROM）在检测临床变化方面具有更高的敏感性和特异性，但直到最近才为肺动脉高压（PH），特别是肺动脉高压（PAH）和慢性血栓栓塞性疾病（CTEPH）开发了这些仪器。虽然这些有价值的工具现在已被纳入 PH 的临床研究中，但它们尚未广泛应用于常规临床护理。
在这项系统评价中，作者评估了为 PH 开发的 PROM 的心理测量特性，比较了 PROM 在 PH 中的其他临床结局，并探讨了 PROM 在临床护理中的应用。
作者使用 MEDLINE 数据库进行了一项系统搜索，检索了 2006 年 1 月 1 日至 2022 年 10 月 1 日期间发表的论文，以确定为 PH 开发和验证的 PROM。确定的 PROM 仅在 PAH 和 CTEPH 组中开发。作者根据既定的心理测量标准评估了确定的工具。还进行了一项额外的搜索，以确定使用这些 PROM 的随机对照试验（RCT），以便与临床结局进行比较。
从检索到的 527 篇论文中，共确定了 35 种 PROM。其中，有 5 种疾病特异性仪器被纳入最终分析。虽然 CAMPHOR（剑桥肺动脉高压结局回顾）和 emPHasis-10 在 PAH 和 CTEPH 患者中在心理测量特性方面表现良好，但由于其简洁的格式，emPHasis-10 在临床实践中的应用更具可行性。作者的分析显示，肺动脉高压-症状和影响问卷表现良好，但需要更多关于可解释性和可行性的数据。
emPHasis-10 表现出较强的心理测量特性和最大的临床应用可行性。需要进一步研究评估 PROM 在 PH 常规临床护理中的整合。
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