(161) DARWIN EU® - Real-World Evaluation of Pulmonary Arterial Hypertension Treatment and Outcomes in Europe

Background: Pulmonary Arterial Hypertension (PAH), a rare form of pulmonary hypertension, presents a significant clinical challenge due to its complex etiologies, suboptimal treatment response, and limited survival rates. Evaluating real-world treatment patterns and outcomes is essential for contextualizing future interventions. In response to the European Medicines Agency's request, we conducted a study to analyse PAH treatment patterns and outcomes in routine healthcare settings across the DARWIN EU® network.

Objectives: To investigate treatment patterns and outcomes for patients with pulmonary arterial hypertension in routine healthcare settings across the DARWIN EU® network.

Methods: This retrospective cohort study analyzed routinely collected health data from four databases between 2012-2022. The databases included primary care records from the UK (Clinical Practice Research Datalink GOLD [CPRD GOLD]), primary and outpatient specialist care records in Germany (IQVIA Disease Analyzer [DA]), hospital-based data in France (Clinical Data Warehouse of Bordeaux University Hospital [CWD Bordeaux]), and an Estonian biobank (Estonian Biobank [EBB]). The study outcomes were treatment sequences and duration, hospitalization rates, and mortality rates.

Results: Among 9,474 patients with incident PAH (median age, 62 – 77 years; female sex, 51 – 65%), prevalent comorbidities included cardiovascular diseases, COPD, diabetes, chronic kidney disease, and connective tissue disorders. Dyspnea and chest pain were predominant symptoms at diagnosis. Post-index treatment primarily consisted of phosphodiesterase- 5 inhibitors monotherapy in primary care and combination therapy with bosentan-tadalafil or tadalafil-ambrisentan in secondary care, with median treatment durations ranging from 1 to 189 days. Within one year of diagnosis, all treated patients were hospitalized, with mortality rates of 7.2–8.1%. A 2 to 4-fold increase in mortality rates was observed between the one- and five-year post-index.

Conclusions: PAH is prevalent among older adults with pre-existing cardiovascular conditions and is often managed with monotherapy in primary care and combination therapy in secondary care. The observed high hospitalization rates and progressively increasing mortality rates in PAH underscore the need for early diagnosis and effective management strategies to improve patient survival.