Date: Sunday, November 8, 2020
Session 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.
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 April 11, 2021.
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