Pulmonary arterial hypertension registries: past, present and into the future

摘要

Disease registries are important sources of real-world evidence that inform clinical practice and health policy, particularly when randomised controlled trials do not exist [1]. Much of what we know today about pulmonary arterial hypertension (PAH) has come from observational studies from national and/or international disease registries. In this issue of the European Respiratory Review, SWINNEN et al. [2] provide a concise and comprehensive overview of how various registries have contributed to the current and evolving state of knowledge on PAH. They illustrate some of the major successes of modern PAH registries, which have broadened our understanding of changing patient demographics, epidemiology, natural history, risk factors and prognosis in PAH. Despite their inherent limitations, registry data continue to influence clinical practice and treatment guidelines. For example, the most recent PAH treatment algorithm in the 2018 World Symposium on Pulmonary Hypertension [3] proposes a risk-based approach to therapy, based largely on independent validation of risk assessment strategies from European and US PAH registries [4–8]. Another example of the influence of registry data on clinical care is the common use of initial triple combination therapy with an intravenous prostacyclin, an endothelin receptor antagonist and a phosphodiesterase type 5 inhibitor for patients with high risk PAH, which comes from registry studies showing improved outcomes in treated patients [9], despite an absence of randomised controlled study data to support this practice.