Key result
Initial dual therapy comprises only ~29% of treatment regimens for newly diagnosed PAH patients.
Why the study?
The 2022 ESC/ERS guidelines recommend initial ERA plus PDE5i combination therapy for low- to intermediate-risk PAH without cardiopulmonary comorbidities, but US treatment patterns in newly diagnosed patients remained uncharacterized.
What are the real-world treatment patterns and patient characteristics associated with the use of initial dual therapy versus monotherapy in newly diagnosed PAH patients in the US?
Cohort (n=2,868)
What are the real-world treatment patterns and patient characteristics associated with the use of initial dual therapy versus monotherapy in newly diagnosed PAH patients in the US?
Real-world US data reveals that 71.3% of newly diagnosed PAH patients initiate monotherapy rather than guideline-recommended dual therapy, likely driven by a high prevalence of cardiopulmonary comorbidities.
Background The 2022 European Society of Cardiology/European Respiratory Society (ESC/ERS) pulmonary hypertension guidelines recommend initial combination of endothelin receptor antagonist (ERA) and phosphodiesterase type-5 inhibitor (PDE5i) in patients with pulmonary arterial hypertension (PAH) at low to intermediate risk without cardiopulmonary comorbidities. Objective To examine US treatment patterns for newly diagnosed patients, including frequency of cardiopulmonary comorbidities. Methods Treatment-naïve adults (≥18 years) initiating treatment, identified using claims data (IQVIA PharMetrics® Plus; April 2013–June 2023), were assigned dual therapy if initiating ERA/PDE5i within a treatment-determination period (3 months), or monotherapy if initiating ERA or PDE5i. Descriptive statistics captured 25th/75th percentiles, means (SD), and medians. Results Of 2868 patients, 824 (28.7%) initiated dual therapy and 2044 (71.3%) monotherapy. In dual therapy, 461 (56.0%) initiated ERA first, 250 (30.3%) PDE5i first, and 113 (13.7%) both the same day. In monotherapy, 153 (7.5%) received ERA and 1891 (92.5%) PDE5i. For escalation to dual therapy, 330 (16.1%) monotherapy users initiated ERA (10.7%) or PDE5i (5.5%) during follow-up. Most had cardiopulmonary comorbidities (monotherapy: 86.8%; dual: 79.6%). Of the 824 on dual therapy, 20.4% started triple therapy during follow-up. Compared with monotherapy, dual therapy users were younger (54.9 vs 59.6 years) and mostly female (72.9% vs 60.9%). Discussion This study found that in the United States, among newly diagnosed PAH patients, 71.3% initiated monotherapy and 28.7% dual therapy, with 16.1% of monotherapy patients eventually escalating to dual therapy. High rates of initial monotherapy may reflect the high proportion of patients with comorbidities and their possible intolerance of initial dual therapy. As these data mostly precede the 2022 guidelines, future research should include treatment post-guidelines, rationales behind decision making, differences between initial monotherapy and dual therapy users, and monotherapy overreliance and effects on morbidity and mortality. Conclusions This analysis of real-world US treatment patterns for newly initiating PAH patients found low rates of upfront dual-therapy use with high rates of cardiopulmonary comorbidities.
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Paoli et al. (2025) conducted a cohort in Pulmonary arterial hypertension (n=2,868). Initial dual therapy (ERA and PDE5i) vs. Initial monotherapy (ERA or PDE5i) was evaluated on Frequency of initial treatment pattern (dual therapy vs monotherapy). Among newly diagnosed patients with pulmonary arterial hypertension in the US, 71.3% initiated monotherapy and 28.7% initiated dual therapy, with high rates of cardiopulmonary comorbidities observed in both groups.
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