Treatment Patterns in Patients With Incident Pulmonary Hypertension: Real-World Data From the Pulmonary Hypertension Association Registry

Sarah L Khan; Carly J Paoli; Noah Kime; Kayleen Williams; Gabriela Gomez Rendon; Adriano R Tonelli; Hector Cajigas; Stephen C Mathai; Sandeep Sahay

doi:10.1016/j.chest.2025.11.035

Treatment Patterns in Patients With Incident Pulmonary Hypertension: Real-World Data From the Pulmonary Hypertension Association Registry

Chest. 2025 Dec 8:S0012-3692(25)05817-9. doi: 10.1016/j.chest.2025.11.035. Online ahead of print.

Authors

Sarah L Khan¹, Carly J Paoli², Noah Kime³, Kayleen Williams³, Gabriela Gomez Rendon², Adriano R Tonelli⁴, Hector Cajigas⁵, Stephen C Mathai¹, Sandeep Sahay⁶

Affiliations

¹ Division of Pulmonary and Critical Care Medicine, Johns Hopkins University School of Medicine, Baltimore, MD.
² Johnson & Johnson, Titusville, NJ.
³ University of Washington Department of Biostatistics, Seattle, WA.
⁴ Department of Pulmonary and Critical Care Medicine, Integrated Hospital Care Institute, Cleveland Clinic, Cleveland, OH.
⁵ Mayo Clinic Rochester Division of Pulmonary and Critical Care Medicine, Rochester, MN.
⁶ Division of Pulmonary, Critical Care & Sleep Medicine, Houston Methodist Hospital, Houston, TX. Electronic address: ssahay@houstonmethodist.org.

PMID: 41371490
DOI: 10.1016/j.chest.2025.11.035

Abstract

Background: Current evidence supports risk-based treatment for pulmonary arterial hypertension (PAH) with an endothelin receptor antagonist and phosphodiesterase type 5 inhibitor as initial therapy for patients with low- and intermediate-risk PAH, and triple therapy with the addition of a parenteral prostacyclin for patients with high-risk PAH.

Research question: What are the current treatment patterns for patients with newly diagnosed PAH in the Pulmonary Hypertension Association Registry (PHAR)?

Study design and methods: This analysis was performed in a prospectively collected cohort of patients with incident PAH diagnosed by right heart catheterization and enrolled in the PHAR between September 30, 2015, and February 14, 2024. The primary objective was to assess the guideline-recommended risk-based treatment approaches and their impact in the real world. All-cause mortality was compared across risk groups and between patients receiving monotherapy, dual therapy, and triple therapy for PAH across risk strata.

Results: There were 1,019 patients with incident PAH in the PHAR who received either initial monotherapy (n = 344, 33.8%), dual therapy (n = 557, 54.7%), or triple therapy (n = 118, 11.6%). Of 323 patients with complete data for risk classification who received initial monotherapy, 25.1% were low risk, 31.3% were intermediate risk, and 43.7% were high risk by Registry to Evaluate Early and Long-term PAH Disease Management Lite 2 risk score. For the 540 patients treated with initial dual therapy, 35.7% were low risk, 26.5% were intermediate risk, and 37.8% were high risk. Finally, among the 113 patients who received initial triple therapy, 31.9% were low risk, 28.3% were intermediate risk, and 39.8% were high risk. All-cause mortality was lowest for patients with low-risk PAH at baseline and highest for those with high-risk PAH at baseline across the entire cohort and for patients treated with initial monotherapy, dual therapy, or triple therapy.

Interpretation: This analysis highlights the underutilization of PAH therapies for patients with newly diagnosed PAH in this real-world patient population.

Keywords: REVEAL risk score; mortality; pulmonary hypertension; risk stratification; survival; treatment.