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Multicenter Study
. 2025 Jan;42(1):193-215.
doi: 10.1007/s12325-024-03026-1. Epub 2024 Nov 4.

Clinical Characteristics and Treatment of Patients Diagnosed with Pulmonary Arterial Hypertension: A Real-World Study in the USA, Europe and Japan

C D Vizza  1 , R Klok  2 , J Harley  3 , M Small  3 , M Scott  3 , D Lautsch  2 , R J White  4
Affiliations
Multicenter Study

Clinical Characteristics and Treatment of Patients Diagnosed with Pulmonary Arterial Hypertension: A Real-World Study in the USA, Europe and Japan

C D Vizza et al. Adv Ther. 2025 Jan.

Abstract

Introduction: This study aimed to describe the clinical characteristics of patients with pulmonary arterial hypertension, treatment received, and factors predicting initial or earlier combination therapy.

Methods: The Adelphi Real World Pulmonary Arterial Hypertension (PAH) Disease Specific Programme™ is a cross-sectional survey with retrospective data collection conducted in the USA, Europe (France, Germany, Italy, Spain, and the UK), and Japan from March to August 2022. Physicians reported patient characteristics, treatment history, and reasons for treatment selection. Descriptive statistics were grouped by country and World Health Organization functional classification. A multivariable Cox regression analysis investigated factors predicting initial or earlier combination therapy use.

Results: Data for 1173 patients was provided by 293 physicians. Patients' mean (standard deviation) age was 58.7 (13.8) years and 54.6% were female. Overall, 91.2% of patients were receiving, or had previously received, PAH-specific treatment. About three-quarters of the cohort were still taking the initial treatment strategy: for this group, 54% were prescribed monotherapy and 32% combination therapy; 15% of patients received supportive therapy alone. The proportion of patients receiving PAH-specific treatment was lowest in the USA (82.0%) and highest in France (94.6%). The proportion of patients receiving PAH on combination therapy was lowest in the USA (23.8%) and highest in Germany (36.5%). Treatment was prescribed for PAH in 87.6%, 89.8%, 89.3%, and 75.0% of patients who were functional class I, II, III, and IV, respectively, and combination therapy usage was more likely for those with more advanced functional class. Higher risk status, care by a pulmonologist, Japanese residence, more complete assessments, and hospitalization in the past 12 months were statistically associated with decreased time to combination therapy for PAH. Older age was statistically associated with increased time to combination therapy.

Conclusion: In this real-world, geographically diverse sample, monotherapy treatment was common, even among patients with advanced disease.

Keywords: Combination therapy; Monotherapy; Pulmonary arterial hypertension; Real-world; Treatment line.

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Conflict of interest statement

Declarations. Conflict of Interest: This study was supported by MSD through a data subscription licence. Rogier Klok and Dominik Lautsch are employees of MSD and are Merck & Co., Inc., Rahway, NJ, USA shareholders. R. James White and Carmine Dario Vizza have received consultancy payments from Merck & Co., Inc. Julia Harley, Mark Small and Megan Scott are full-time employees of Adelphi Real World, an agency which received a subscription fee for data access and analysis. Ethical Approval: The survey was submitted to the Pearl Institutional Review Board, a central institutional review board (IRB), where ethical exemption determination was granted on 02/08/23 (Pearl IRB work order #22-ADRW-131). Data collection was undertaken in line with European Pharmaceutical Marketing Research Association [22] guidelines and as such it does not require ethics committee approval. Each survey was performed in full accordance with relevant legislation at the time of data collection, including the US Health Insurance Portability and Accountability Act 1996, [23] the Declaration of Helsinki 1964 [24], and Health Information Technology for Economic and Clinical Health Act legislation [25]. A complete description of the methods of the survey has been previously published and validated [17–20]. Using a check box, patients provided informed consent for use of their anonymized and aggregated data for research and publication in scientific journals. Data were collected in such a way that patients and physicians could not be identified directly; all data were aggregated and de-identified before receipt.

Figures

Fig. 1
Fig. 1
Cox regression analyses of factors versus time from diagnosis to initiation of therapy. The forest plot shows the HR, 95% CI, and p value for each covariate. A higher HR indicates a shorter time from diagnosis to receiving combination therapy. 6MWT 6-min walking test, BNP brain natriuretic peptide, CI confidence interval, HR hazard ratio, PAH pulmonary arterial hypertension, proBNP BNP prohormone
See this image and copyright information in PMC

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