Independent Consultant, Lima, Peru.
Actelion Pharmaceuticals Ltd, A Johnson & Johnson Company, Global Epidemiology, Allschwil, Switzerland.
Respir Res. 2024 Oct 16;25(1):373. doi: 10.1186/s12931-024-02994-w.
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.
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.
目前缺乏关于肺动脉高压（PAH）这一罕见且无法治愈疾病的真实世界结局的综合总结。我们进行了一项系统文献回顾，以描述成人和儿科 PAH 患者的当前生存、发病率和生活质量（QoL）结局。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，以确定来自基于人群的观察性研究的右心导管检查确诊的 PAH 患者的结局估计值（搜索日期：2021 年 11 月 25 日）。使用叙述性方法综合数据，并进行事后亚组荟萃分析，以探索按地区、疾病严重程度、代表性和研究期划分的成人生存率。该搜索产生了 7473 条记录。经过筛选和全文审查，纳入了 22 项具有 31 项单独结局报告的独特研究。这些研究主要是国家登记处（n=21）、欧洲（n=13），并涵盖了成年人（n=17）；只有 6 项有系统性的全国范围的中心覆盖。生存率是最常报告的结局（n=22）。全球成人 1 年、3 年和 5 年生存率分别为 85%至 99%（n=15）、65%至 95%（n=14）和 50%至 86%（n=9）。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）。未观察到各地区之间生存率的差异。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数据可能缺失，因此解释应谨慎。
本综述描述了基于明确且具代表性的 PAH 人群的当前疾病结局。发病率和 QoL 结局的随访数据总体缺乏；儿科患者的生存估计值很少，并且可能无法推广到当前的治疗时代，尽管在我们的搜索日期之后，来自大型儿科登记处的出版物可用。这项研究表明，全球范围内仍需要改善当前时代 PAH 的长期预后。
