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Open Access 07-03-2024 | Arterial Hypertension | Original Research

Macitentan in Pulmonary Arterial Hypertension Associated with Connective Tissue Disease (CTD-PAH): Real-World Evidence from the Combined OPUS/OrPHeUS Dataset

Authors: Richard Channick, Kelly M. Chin, Vallerie V. McLaughlin, Matthew R. Lammi, Roham T. Zamanian, Stefano Turricchia, Rose Ong, Lada Mitchell, Nick H. Kim

Published in: Cardiology and Therapy | Issue 2/2024

Abstract

Introduction

Data on real-world clinical practice and outcomes of patients with pulmonary arterial hypertension associated with connective tissue disease (CTD-PAH) are scarce. The OPUS/OrPHeUS studies enrolled patients newly initiating macitentan, including those with CTD-PAH. This analysis describes patient characteristics, treatment patterns, outcomes, and safety profiles of patients with CTD-PAH newly initiating macitentan in the US using the OPUS/OrPHeUS combined dataset.

Methods

OPUS was a prospective, US, multicenter, long-term, observational drug registry (April 2014–June 2020). OrPHeUS was a retrospective, US, multicenter medical chart review (October 2013–March 2017). The characteristics, treatment patterns, safety, and outcomes during macitentan treatment of patients with CTD-PAH and its subgroups systemic sclerosis (SSc-PAH), systemic lupus erythematosus (SLE-PAH), and mixed CTD (MCTD-PAH) were descriptively compared to patients with idiopathic/heritable PAH (I/HPAH).

Results

The combined OPUS/OrPHeUS population included 2498 patients with I/HPAH and 1192 patients with CTD-PAH (708 SSc-PAH; 159 SLE-PAH; 124 MCTD-PAH, and 201 other CTD-PAH etiologies). At macitentan initiation for patients with I/HPAH and CTD-PAH, respectively: 61.2 and 69.3% were in World Health Organization functional class (WHO FC) III/IV; median 6-min walk distance was 289 and 279 m; and 58.1 and 65.2% received macitentan as combination therapy. During follow-up, for patients with I/HPAH and CTD-PAH, respectively: median duration of macitentan exposure observed was 14.0 and 15.8 months; 79.0 and 83.0% experienced an adverse event; Kaplan–Meier estimates (95% confidence limits [CL]) of patients free from all-cause hospitalization at 1 year were 60.3% (58.1, 62.4) and 59.3% (56.1, 62.3); and Kaplan–Meier estimates (95% CL) of survival at 1 year were 90.5% (89.1, 91.7) and 90.6% (88.6, 92.3).

Conclusions

Macitentan was used in clinical practice in patients with CTD-PAH and its subgroups, including as combination therapy. The safety and tolerability profile of macitentan in patients with CTD-PAH was comparable to that of patients with I/HPAH.

Trial Registration

OPsumit® Users Registry (OPUS): NCT02126943; Opsumit® Historical Users cohort (OrPHeUS): NCT03197688; www.clinicaltrials.gov

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Graphical Abstract

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Title: Macitentan in Pulmonary Arterial Hypertension Associated with Connective Tissue Disease (CTD-PAH): Real-World Evidence from the Combined OPUS/OrPHeUS Dataset
Authors: Richard Channick
Kelly M. Chin
Vallerie V. McLaughlin
Matthew R. Lammi
Roham T. Zamanian
Stefano Turricchia
Rose Ong
Lada Mitchell
Nick H. Kim
Publication date: 07-03-2024
Publisher: Springer Healthcare
Keywords: Arterial Hypertension
Arterial Hypertension
Systemic Sclerosis
Mixed Connective Tissue Disease
Systemic Lupus Erythematosus
Published in: Cardiology and Therapy / Issue 2/2024
Print ISSN: 2193-8261
Electronic ISSN: 2193-6544
DOI: https://doi.org/10.1007/s40119-024-00361-w

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