Division of Cardiovascular Diseases, Mayo Clinic, Rochester, Minnesota.
Division of Cardiovascular Diseases, Allegheny General Hospital, Pittsburgh, Pennsylvania.
J Am Coll Cardiol. 2013 Dec 24;62(25 Suppl):D51-9. doi: 10.1016/j.jacc.2013.10.023.
Registries of patients with pulmonary arterial hypertension (PAH) have been instrumental in characterizing the presentation and natural history of the disease and provide a basis for prognostication. Since the initial accumulation of data conducted in the 1980s, subsequent registry databases have yielded information about the demographic factors, treatment, and survival of patients and have permitted comparisons between populations in different eras and environments. Inclusion of patients with all subtypes of PAH has also allowed comparisons of these subpopulations. We describe herein the basic methodology by which PAH registries have been conducted, review key insights provided by registries, summarize issues related to interpretation and comparison of the results, and discuss the utility of data to predict survival outcomes. Potential sources of bias, particularly related to the inclusion of incident and/or prevalent patients and missing data, are addressed. A fundamental observation of current registries is that survival in the modern treatment era has improved compared with that observed previously and that outcomes among PAH subpopulations vary substantially. Continuing systematic clinical surveillance of PAH will be important as treatment evolves and as understanding of mechanisms advance. Considerations for future directions of registry studies include enrollment of a broader population of patients with pulmonary hypertension of all clinical types and severity and continued globalization and collaboration of registry databases.
肺高血压(PAH)患者的登记研究在描述疾病的表现和自然史以及提供预后基础方面发挥了重要作用。自 20 世纪 80 年代首次积累数据以来,随后的登记数据库提供了有关人口统计学因素、治疗和患者生存的数据,并允许在不同时代和环境的人群之间进行比较。包括所有 PAH 亚型的患者也允许对这些亚人群进行比较。本文描述了 PAH 登记研究的基本方法,回顾了登记研究提供的关键见解,总结了与解释和比较结果相关的问题,并讨论了数据预测生存结果的实用性。还讨论了潜在的偏倚来源,特别是与包括新发病例和/或现患病例以及数据缺失相关的偏倚。当前登记研究的一个基本观察结果是,与以前观察到的情况相比,在现代治疗时代,PAH 的生存率有所提高,并且 PAH 亚人群的结局差异很大。随着治疗的发展和对机制的理解的提高,对 PAH 的持续系统临床监测将非常重要。未来登记研究的方向包括招募更广泛的各类临床严重程度的肺动脉高压患者,并继续进行登记数据库的全球化和协作。