A COSMIN systematic review of instruments for evaluating health-related quality of life in people with Hereditary Angioedema.

IF 3.2 2区医学 Q1 HEALTH CARE SCIENCES & SERVICES

Health and Quality of Life Outcomes Pub Date : 2025-02-13 DOI:10.1186/s12955-025-02342-6

Irene Baroni, Giulia Paglione, Giada De Angeli, Miriam Angolani, Edward Callus, Arianna Magon, Gianluca Conte, Stefano Terzoni, Maura Lusignani, Rosario Caruso, Andrea Zanichelli

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引用次数: 0

Abstract

Background: Hereditary angioedema (HAE) adversely affects health-related quality of life (HRQoL). HAE often compromises the HRQoL due to the impact on functional capacity caused by edema, pain, other symptoms, and psychosocial factors. Patient-Reported Outcome Measures (PROMs) focus on HRQoL and are crucial tools for evaluating the burden of the disease and choosing the most appropriate interventions for this population. However, no comprehensive evaluations of the characteristics of the available measurements to assess HRQoL have been conducted for this population.

Aim: To identify, analyze, and summarize the PROMs assessing HRQoL in individuals with HAE-C1-INH, addressing the gap in standardized assessment tools.

Methods: A systematic review was conducted up to December 2023 in PubMed, Scopus, Web of Science, Embase, and CINAHL databases, following PRISMA guidelines without language or time restrictions. Psychometric properties of the identified PROMs were appraised using COSMIN standards, and evidence was synthesized using a modified GRADE approach.

Results: From seven studies, five HRQoL PROMs were identified: two generic (SF-36 and SF-36v2) and three disease-specific (HAE-QoL, HAEA-QoL, and AE-QoL). These PROMs generally lacked comprehensive content, structural and cross-cultural validation, with none meeting the criteria for measurement invariance. This limitation affects their applicability across different demographics and cultures. However, the HAE-QoL and AE-QoL instruments were recognized for having moderate quality evidence, suggesting their potential reliability and validity.

Conclusions: This systematic review provides a moderate recommendation for the use of HAE-QoL and AE-QoL in assessing HRQoL in adults with HAE. Despite identified gaps, the moderate evidence quality for these tools supports their use, pending further validation, involving younger age groups and disease-specific contents in the assessments. Developing culturally and demographically adaptable PROMs is, therefore, a priority to improve the accuracy of PROMs in this field.

Review registration number: PROSPERO registration number is CRD42023440137.

查看原文本刊更多论文

COSMIN对遗传性血管性水肿患者健康相关生活质量评估工具的系统综述

背景：遗传性血管性水肿（HAE）对健康相关生活质量（HRQoL）有不利影响。由于水肿、疼痛、其他症状和社会心理因素对功能能力的影响，HAE通常会损害HRQoL。患者报告结果测量（PROMs）侧重于HRQoL，是评估疾病负担和为这一人群选择最适当干预措施的关键工具。然而，尚未对该人群进行评估HRQoL的可用测量指标的特征进行全面评估。目的：识别、分析和总结评估HAE-C1-INH患者HRQoL的PROMs，解决标准化评估工具的差距。方法：根据PRISMA指南，在没有语言和时间限制的情况下，对截至2023年12月的PubMed、Scopus、Web of Science、Embase和CINAHL数据库进行系统评价。使用COSMIN标准对鉴定出的prom的心理测量特性进行评价，并使用改进的GRADE方法合成证据。结果：从7项研究中，鉴定出5种HRQoL PROMs: 2种通用（SF-36和SF-36v2）和3种疾病特异性（HAE-QoL、HAEA-QoL和AE-QoL）。这些prom普遍缺乏全面的内容、结构和跨文化验证，没有一个满足测量不变性的标准。这一限制影响了它们在不同人口统计和文化中的适用性。然而，HAE-QoL和AE-QoL仪器被认为具有中等质量的证据，表明它们具有潜在的信度和效度。结论：本系统综述推荐使用HAE- qol和AE-QoL评估成人HAE患者的HRQoL。尽管发现了差距，但这些工具的证据质量适中，支持它们的使用，有待进一步验证，在评估中涉及更年轻的年龄组和特定疾病的内容。因此，开发具有文化和人口适应性的PROMs是提高该领域PROMs准确性的优先事项。审核注册号：普洛斯佩罗注册号为CRD42023440137。

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来源期刊

Health and Quality of Life Outcomes 医学-卫生保健

CiteScore

7.30

自引率

2.80%

发文量

154

审稿时长

3-8 weeks

期刊介绍： Health and Quality of Life Outcomes is an open access, peer-reviewed, journal offering high quality articles, rapid publication and wide diffusion in the public domain. Health and Quality of Life Outcomes considers original manuscripts on the Health-Related Quality of Life (HRQOL) assessment for evaluation of medical and psychosocial interventions. It also considers approaches and studies on psychometric properties of HRQOL and patient reported outcome measures, including cultural validation of instruments if they provide information about the impact of interventions. The journal publishes study protocols and reviews summarising the present state of knowledge concerning a particular aspect of HRQOL and patient reported outcome measures. Reviews should generally follow systematic review methodology. Comments on articles and letters to the editor are welcome.