Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study)

Abstract Background In pulmonary arterial hypertension (PAH), comparative assessment of treatment effect on survival in randomized controlled settings of contemporary patients has not been feasible. Objective The aim of this study was to use EXPOSURE, the ongoing, real-world, post-authorization safe...

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Main Authors: Stefan Söderberg, Pilar Escribano-Subias, Ciara Archey, Audrey Muller, Martina Fontana, Tobias J. Lange, Sean Gaine
Format: Article
Language:English
Published: Adis, Springer Healthcare 2025-04-01
Series:Drugs - Real World Outcomes
Online Access:https://doi.org/10.1007/s40801-025-00488-9
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author Stefan Söderberg
Pilar Escribano-Subias
Ciara Archey
Audrey Muller
Martina Fontana
Tobias J. Lange
Sean Gaine
author_facet Stefan Söderberg
Pilar Escribano-Subias
Ciara Archey
Audrey Muller
Martina Fontana
Tobias J. Lange
Sean Gaine
author_sort Stefan Söderberg
collection DOAJ
description Abstract Background In pulmonary arterial hypertension (PAH), comparative assessment of treatment effect on survival in randomized controlled settings of contemporary patients has not been feasible. Objective The aim of this study was to use EXPOSURE, the ongoing, real-world, post-authorization safety study, and commitment to the European Medicines Agency to perform pre-specified comparative survival analyses between patients that newly initiated selexipag versus other PAH-specific therapies by applying statistical methods to account for population differences. Methods EXPOSURE (EUPAS19085) is an observational study comprising patients with PAH who initiated selexipag or other PAH-specific therapy. To balance characteristics of patients in the other PAH therapy cohort with the selexipag cohort at PAH therapy initiation (baseline), propensity score weighting was applied. Mortality rate ratios (MRRs) were calculated. Results 2014 patients were available for analysis. Prior to applying propensity score weighting, patients in the selexipag cohort were more likely to have longer time from diagnosis, less functional impairment, and treatment with combination background therapy versus the other PAH therapy cohort. Following weighting, baseline variables for both cohorts were well balanced. Weighted treatment exposure was 827.9 and 840.5 person-years for the selexipag and modelled other PAH therapy cohort, respectively. Weighted proportion of deaths was lower in the selexipag versus modelled other PAH therapy cohort; MRR showed a higher survival rate for selexipag-treated patients (MRR [95% confidence interval]: 0.55 [0.31–0.99]). Conclusions Survival analyses in EXPOSURE suggest a reduced risk of mortality among the cohort of patients newly initiated on selexipag compared with the modelled cohort newly initiated with other PAH-specific therapies. Further research is needed to confirm this observation. Trial Registry Trial registration: EUPAS19085. Graphical Abstract
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spelling doaj-art-8bf527afeb504de997c764ceb119663c2025-08-20T03:47:16ZengAdis, Springer HealthcareDrugs - Real World Outcomes2199-11542198-97882025-04-0112218919910.1007/s40801-025-00488-9Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study)Stefan Söderberg0Pilar Escribano-Subias1Ciara Archey2Audrey Muller3Martina Fontana4Tobias J. Lange5Sean Gaine6Department of Public Health and Clinical Medicine, Medicine, Umeå UniversityPulmonary Hypertension Unit, Cardiology Department, CIBERCV, Hospital 12 de OctubreJohnson & JohnsonJohnson & JohnsonJohnson & JohnsonDepartment of Pulmonology, Kreisklinik Bad ReichenhallNational Pulmonary Hypertension Unit, Mater Misericordiae University HospitalAbstract Background In pulmonary arterial hypertension (PAH), comparative assessment of treatment effect on survival in randomized controlled settings of contemporary patients has not been feasible. Objective The aim of this study was to use EXPOSURE, the ongoing, real-world, post-authorization safety study, and commitment to the European Medicines Agency to perform pre-specified comparative survival analyses between patients that newly initiated selexipag versus other PAH-specific therapies by applying statistical methods to account for population differences. Methods EXPOSURE (EUPAS19085) is an observational study comprising patients with PAH who initiated selexipag or other PAH-specific therapy. To balance characteristics of patients in the other PAH therapy cohort with the selexipag cohort at PAH therapy initiation (baseline), propensity score weighting was applied. Mortality rate ratios (MRRs) were calculated. Results 2014 patients were available for analysis. Prior to applying propensity score weighting, patients in the selexipag cohort were more likely to have longer time from diagnosis, less functional impairment, and treatment with combination background therapy versus the other PAH therapy cohort. Following weighting, baseline variables for both cohorts were well balanced. Weighted treatment exposure was 827.9 and 840.5 person-years for the selexipag and modelled other PAH therapy cohort, respectively. Weighted proportion of deaths was lower in the selexipag versus modelled other PAH therapy cohort; MRR showed a higher survival rate for selexipag-treated patients (MRR [95% confidence interval]: 0.55 [0.31–0.99]). Conclusions Survival analyses in EXPOSURE suggest a reduced risk of mortality among the cohort of patients newly initiated on selexipag compared with the modelled cohort newly initiated with other PAH-specific therapies. Further research is needed to confirm this observation. Trial Registry Trial registration: EUPAS19085. Graphical Abstracthttps://doi.org/10.1007/s40801-025-00488-9
spellingShingle Stefan Söderberg
Pilar Escribano-Subias
Ciara Archey
Audrey Muller
Martina Fontana
Tobias J. Lange
Sean Gaine
Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study)
Drugs - Real World Outcomes
title Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study)
title_full Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study)
title_fullStr Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study)
title_full_unstemmed Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study)
title_short Comparative Analysis of Survival in Pulmonary Arterial Hypertension for Patients Treated with Selexipag in Clinical Practice (EXPOSURE Study)
title_sort comparative analysis of survival in pulmonary arterial hypertension for patients treated with selexipag in clinical practice exposure study
url https://doi.org/10.1007/s40801-025-00488-9
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