Macitentan in Pulmonary Arterial Hypertension Associated with Connective Tissue Disease (CTD-PAH) : Real-World Evidence from the Combined OPUS/OrPHeUS Dataset
© 2024. The Author(s)..
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 Graphical abstract available for this article.
Medienart: |
E-Artikel |
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Erscheinungsjahr: |
2024 |
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Erschienen: |
2024 |
Enthalten in: |
Zur Gesamtaufnahme - year:2024 |
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Enthalten in: |
Cardiology and therapy - (2024) vom: 07. März |
Sprache: |
Englisch |
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Beteiligte Personen: |
Channick, Richard [VerfasserIn] |
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Links: |
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Themen: |
Connective tissue disease |
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Anmerkungen: |
Date Revised 07.03.2024 published: Print-Electronic ClinicalTrials.gov: NCT02126943, NCT03197688 Citation Status Publisher |
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doi: |
10.1007/s40119-024-00361-w |
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funding: |
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Förderinstitution / Projekttitel: |
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PPN (Katalog-ID): |
NLM36941182X |
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245 | 1 | 0 | |a Macitentan in Pulmonary Arterial Hypertension Associated with Connective Tissue Disease (CTD-PAH) |b Real-World Evidence from the Combined OPUS/OrPHeUS Dataset |
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500 | |a published: Print-Electronic | ||
500 | |a ClinicalTrials.gov: NCT02126943, NCT03197688 | ||
500 | |a Citation Status Publisher | ||
520 | |a © 2024. The Author(s). | ||
520 | |a 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 | ||
520 | |a 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) | ||
520 | |a 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) | ||
520 | |a 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 | ||
520 | |a TRIAL REGISTRATION: OPsumit® Users Registry (OPUS): NCT02126943; Opsumit® Historical Users cohort (OrPHeUS): NCT03197688; www | ||
520 | |a CLINICALTRIALS: gov Graphical abstract available for this article | ||
650 | 4 | |a Journal Article | |
650 | 4 | |a Connective tissue disease | |
650 | 4 | |a Macitentan | |
650 | 4 | |a Mixed connective tissue disease | |
650 | 4 | |a Pulmonary arterial hypertension | |
650 | 4 | |a Real-world evidence | |
650 | 4 | |a Scleroderma | |
650 | 4 | |a Systemic lupus erythematosus | |
650 | 4 | |a Systemic sclerosis | |
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700 | 1 | |a Zamanian, Roham T |e verfasserin |4 aut | |
700 | 1 | |a Turricchia, Stefano |e verfasserin |4 aut | |
700 | 1 | |a Ong, Rose |e verfasserin |4 aut | |
700 | 1 | |a Mitchell, Lada |e verfasserin |4 aut | |
700 | 1 | |a Kim, Nick H |e verfasserin |4 aut | |
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