ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 1381

Real-World Mono-, Double and Triple Combination Treatment Patterns with Macitentan in Patients with Pulmonary Arterial Hypertension Associated with Connective Tissue Disease (PAH-CTD): Evidence from the Combined OPUS/OrPHeUS Dataset

Matthew Lammi1, Kelly Chin2, Nick H. Kim3, Vallerie McLaughlin4, Roham Zamanian5, Megan Flynn6, Sandrine Leroy7, Rose Ong7, Graham Wetherill7 and Richard Channick8, 1Lousiana State University Health Sciences Center, New Orleans, LA, 2UT Southwestern Medical Center, Dallas, TX, 3University of California San Diego, La Jolla, CA, 4University of Michigan, Ann Arbor, MI, 5Stanford University School of Medicine, Stanford, CA, 6Actelion Pharmaceuticals US, Inc., South San Francisco, CA, 7Actelion Pharmaceuticals Ltd, Allschwil, Switzerland, 8University of California Los Angeles, Los Angeles

Meeting: ACR Convergence 2020

Keywords: , ,

Session Information

Date: Sunday, November 8, 2020

Title: Systemic Sclerosis & Related Disorders – Clinical Poster III

Session Type: Poster Session C

Session Time: 9:00AM-11:00AM

Background/Purpose: Patients with PAH-CTD have a worse prognosis than patients with most other PAH etiologies. The OPsumit® USers (OPUS) Registry and OPsumit® Historical USers (OrPHeUS) study provide real-world evidence on the management of PAH-CTD patients newly treated with macitentan.

Methods: OPUS is a prospective, US, multicenter, drug registry ongoing since April 2014 (NCT02126943). OrPHeUS was a retrospective, US, multicenter chart review (NCT03197688); data collected October 2013–March 2017. Patient characteristics, safety and clinical outcomes are described by treatment pattern (mono-/double/triple therapy) at macitentan initiation (baseline) for PAH-CTD (WHO Clinical Classifications of PH Group 1) patients in the combined OPUS/OrPHeUS population. PAH etiology was investigator-assessed and not adjudicated.

Results: As of August 2019, the follow-up OPUS/OrPHeUS PAH population (N=4387) included 1130 PAH-CTD patients: 433 (38.3%) received macitentan monotherapy, 536 (47.4%) received double and 161 (14.2%) received triple combination therapy at baseline. Median (Q1, Q3) age was 63 (53, 70) years in mono-, 61 (50, 70) in double and 61 (51, 69) in triple therapy patients; most patients were female. In the monotherapy group, more patients were newly-diagnosed; median (Q1, Q3) time from diagnosis was 2.2 (0.6, 18.0) months in mono-, 8.7 (1.8, 37.1) in double and 34.8 (12.5, 80.4) in triple therapy patients. Of patients with WHO functional class (FC) recorded at baseline (49.2–57.3% of patients), 63.4% of mono-, 72.6% of double, and 68.2% of triple therapy patients were FC III/IV. Median (Q1, Q3) baseline 6‑minute walk distance (reported in 32.1-40.7% of patients) was 274 (161, 366), 270 (194, 347), and 305 (226, 362) m in mono-, double and triple therapy patients. Treatment patterns at month 6 are shown in the Figure. Patients with ≥1 hepatic adverse event included 28 (6.5%), 36 (6.7%) and 21 (13.0%) on mono-, double and triple therapy. Macitentan was discontinued in 146/433 (33.7%) mono-, 188/536 (35.1%) double and 80/161 (49.7%) triple therapy patients: 59 (13.6%), 80 (14.9%) and 39 (24.2%) due to an adverse event. Kaplan-Meier (KM) estimates (95% CI) for mono-, double and triple therapy patients at macitentan initiation showed that 69% (64, 74), 62% (57, 66) and 61% (52, 69) of patients were free from hospitalization at 12 months; 12-month KM survival estimates (95% CI) were 94% (90, 96), 90% (87, 93) and 88% (81, 92).

Conclusion: Despite the recommendation for combination therapy, a large proportion of patients received monotherapy at macitentan initiation and 6 months after initiation. For the vast majority of patients, combination treatment strategy did not change between baseline and 6 months.

Figure: Treatment patterns at month 6

Disclosure: M. Lammi, None; K. Chin, Actelion Pharmaceuticals Ltd, 2, 5, 8, United Therapeutics, 2, 5, 8, Ironwood Pharmaceuticals, 2, Bayer Healthcare (through UCSD), 5, Gossamer Bio, 8, American Heart Association, 6; N. Kim, Actelion Pharmaceuticals Ltd, 2, 5, 8, Bellerophen, 2, Eiger, 2, SoniVie, 2, Gilead, 2, Lung Biotechnology, 2, Gossamer, 2, Bayer Healthcare, 5, 8, Arena Pharmaceuticals, 5, 8, Merck, 5, United Therapeutics, 5; V. McLaughlin, Actelion Pharmaceuticals Ltd, 2, 5, 8, Acceleron, 2, 5, Bayer Healthcare, 2, 5, United Therapeutics, 2, 5, Reata Pharmaceutics, 2, SoniVie, 2, Arena Pharmaceuticals, 5, Caremark, 5, CiVi Biopharma, 5; R. Zamanian, Actelion Pharmaceuticals Ltd, 2, United Therapeutics, 2, Vivus, 5, Pfizer, 5, Selten, 5, Genentech, 1, 4, Morphogenic-IX, 8, FK-506 in PAH, 9; M. Flynn, Actelion Pharmaceuticals Ltd, 1, 3, 4; S. Leroy, Actelion Pharmaceuticals Ltd, 1, 3, 4; R. Ong, Actelion Pharmaceuticals Ltd, 3; G. Wetherill, Actelion Pharmaceuticals Ltd, 3; R. Channick, Actelion Pharmaceuticals Ltd,, 2, 5, 8, United Therapeutics, 2, Bayer Healthcare, 5, 8, Arena Pharmaceuticals, 5.

To cite this abstract in AMA style:

Lammi M, Chin K, Kim N, McLaughlin V, Zamanian R, Flynn M, Leroy S, Ong R, Wetherill G, Channick R. Real-World Mono-, Double and Triple Combination Treatment Patterns with Macitentan in Patients with Pulmonary Arterial Hypertension Associated with Connective Tissue Disease (PAH-CTD): Evidence from the Combined OPUS/OrPHeUS Dataset [abstract]. Arthritis Rheumatol. 2020; 72 (suppl 10). https://acrabstracts.org/abstract/real-world-mono-double-and-triple-combination-treatment-patterns-with-macitentan-in-patients-with-pulmonary-arterial-hypertension-associated-with-connective-tissue-disease-pah-ctd-evidence-from-t/. Accessed .

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