Survival, morbidity, and quality of life in pulmonary arterial hypertension patients: a systematic review of outcomes reported by population-based observational studies.

IF 5.8 2区 医学 Q1 Medicine Respiratory Research Pub Date : 2024-10-16 DOI:10.1186/s12931-024-02994-w
Stefan Reinders, Eva-Maria Didden, Rose Ong
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引用次数: 0

Abstract

Background: Comprehensive summaries on real-world outcomes in pulmonary arterial hypertension (PAH)-a rare, incurable condition, are lacking. We conducted a systematic literature review to describe current survival, morbidity, and quality of life (QoL) outcomes in adult and pediatric PAH patients. We searched Medline and Embase electronic databases, clinicaltrials.gov, and encepp.eu entries, and grey literature to identify outcome estimates for right-heart catheterization-confirmed PAH patients from population-based observational studies (search date: 25 Nov 2021). Data were synthesized using a narrative approach and post-hoc subgroup meta-analyses were conducted to explore adult survival by region, disease severity, representativeness, and study period. The search yielded 7473 records. Following screening and full text review, 22 unique studies with 31 individual reports of outcomes were included. Studies were mostly national registries (n = 21), European (n = 13) and covering adults (n = 17); only six had systematic countrywide coverage of centers. Survival was the most frequently reported outcome (n = 22). Global adult 1-, 3-, and 5-year survival ranged from 85 to 99% (n = 15), 65 to 95% (n = 14), and 50 to 86% (n = 9), respectively. Subgroup meta-analysis showed that 1-, 3-, and 5-year survival in Europe was 90% (95% CI 86-94%; n = 8), 78% (95% CI 68-86%; n = 8), and 61% (95% CI 49-72%; n = 6), respectively; 1-year survival in North America was 88% (95% CI 83-93%; n = 3) and 3-year survival in Asia was 85% (95% CI 82-88%; n = 3). No difference in survival between regions was observed. Subgroup analysis suggested higher survival in patients with better baseline functional class; however, interpretation should be cautioned due to large subgroup heterogeneity and potential missingness of data.

Short conclusion: This review describes current disease outcomes based on well-defined and representative PAH populations. There is an overall lack of follow-up data for morbidity and QoL outcomes; survival estimates for pediatric patients are scarce and may not be generalizable to the current treatment era, although publications from large pediatric registries became available after our search date. This study demonstrated a remaining unmet need world-wide to improve long-term prognosis in PAH in the current era.

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肺动脉高压患者的存活率、发病率和生活质量:基于人群的观察性研究报告结果的系统回顾。
背景:肺动脉高压(PAH)是一种罕见的不治之症,目前尚缺乏对其实际治疗效果的全面总结。我们进行了一项系统性文献综述,以描述成人和儿童 PAH 患者目前的生存率、发病率和生活质量(QoL)结果。我们检索了 Medline 和 Embase 电子数据库、clinicaltrials.gov 和 encepp.eu 条目以及灰色文献,从基于人群的观察性研究(检索日期:2021 年 11 月 25 日)中确定了右心导管检查确诊的 PAH 患者的预后。我们采用叙述式方法对数据进行了综合,并进行了事后分组荟萃分析,以探讨不同地区、疾病严重程度、代表性和研究时期的成人存活率。搜索共获得 7473 条记录。经过筛选和全文审阅,共纳入了 22 项独特的研究和 31 份单独的结果报告。这些研究多为国家登记(21 项)、欧洲登记(13 项)和成人登记(17 项);只有六项研究对全国范围内的中心进行了系统性覆盖。存活率是最常报告的结果(22 例)。全球成人 1 年、3 年和 5 年生存率分别为 85% 至 99% (15 人)、65% 至 95% (14 人)和 50% 至 86% (9 人)。亚组荟萃分析显示,欧洲的 1 年、3 年和 5 年生存率分别为 90% (95% CI 86-94%; n = 8)、78% (95% CI 68-86%; n = 8) 和 61% (95% CI 49-72%; n = 6);北美的 1 年生存率为 88% (95% CI 83-93%; n = 3),亚洲的 3 年生存率为 85% (95% CI 82-88%; n = 3)。不同地区的存活率没有差异。亚组分析表明,基线功能分级较好的患者生存率较高;然而,由于亚组异质性较大且可能存在数据遗漏,因此在解释时应谨慎:简短结论:这篇综述描述了目前基于定义明确且具有代表性的 PAH 群体的疾病结果。简短结论:本综述描述了目前基于定义明确且具有代表性的 PAH 群体的疾病结果,但总体上缺乏发病率和 QoL 结果的随访数据;尽管在我们的搜索日期之后,大型儿科登记处的出版物可供使用,但儿科患者的生存率估计值很少,可能无法推广到当前的治疗时代。这项研究表明,在当今时代,全世界对改善 PAH 长期预后的需求仍未得到满足。
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来源期刊
Respiratory Research
Respiratory Research RESPIRATORY SYSTEM-
CiteScore
9.70
自引率
1.70%
发文量
314
审稿时长
4-8 weeks
期刊介绍: Respiratory Research publishes high-quality clinical and basic research, review and commentary articles on all aspects of respiratory medicine and related diseases. As the leading fully open access journal in the field, Respiratory Research provides an essential resource for pulmonologists, allergists, immunologists and other physicians, researchers, healthcare workers and medical students with worldwide dissemination of articles resulting in high visibility and generating international discussion. Topics of specific interest include asthma, chronic obstructive pulmonary disease, cystic fibrosis, genetics, infectious diseases, interstitial lung diseases, lung development, lung tumors, occupational and environmental factors, pulmonary circulation, pulmonary pharmacology and therapeutics, respiratory immunology, respiratory physiology, and sleep-related respiratory problems.
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