期刊
CHEST
卷 146, 期 3, 页码 686-708出版社
ELSEVIER
DOI: 10.1378/chest.13-2634
关键词
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资金
- Actelion Pharmaceuticals Ltd
- Bayer AG
- GlaxoSmithKline
- Reseau en Sante Respiratoire of the FRSQ
BACKGROUND: Health-related quality of life (HRQoL) is severely impaired in pulmonary arterial hypertension (PAH). We aimed to assess the effect of PAH-specific therapies on HRQoL. METHODS: A literature search was performed in MEDLINE and Embase databases (January 1990 to September 2013) to retrieve prospective placebo-controlled randomized trials of at least 6 weeks duration reporting the effect of PAH-specific therapies on HRQoL in adult patients with PAH. The articles were independently reviewed, and the validity of the trials was assessed using the Cochrane's Risk of Bias Tool. RESULTS: The literature search identified 1,172 titles. Seventeen articles reporting on 14 trials were retrieved, all of which were associated with a low risk of bias. The median study duration of the different trials was 12 weeks. Most patients had idiopathic PAH or PAH associated with connective tissue disease. A variety of HRQoL questionnaires were used in these trials, and most were generic. HRQoL results were most commonly minimally detailed, and some pivotal trials did not even assess HRQoL. Nevertheless, these trials consistently demonstrated statistically significant improvements in HRQoL with PAH-specific therapies, especially for the physical domains. In most cases, however, these improvements were smaller than the minimal important difference in HRQoL previously reported in PAH. CONCLUSION: This review shows that PAH-specific therapies improve HRQoL in PAH. However, it remains difficult to draw any firm conclusion about the clinical significance of these improvements. Further work is mandatory to validate PAH-specific questionnaires that are responsive to clinical changes as well as to establish their interpretability.
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