Abstract
Purpose: This study examined the efficacy and safety of selexipag in treating pulmonary arterial hypertension (PAH) associated with congenital heart disease (CHD). Materials and Methods: We conducted a retrospective study of patients with CHD-associated PAH, treated with selexipag since December 2017. Thirteen adult patients (mean age, 45.4 years; women, 77%) were treated with selexipag as add-on therapy. Baseline characteristics, World Health Organization functional class, 6-minute walking distance (6MWD) test results, N-terminal pro-B-type natriuretic peptide levels, echocardiographic data, and incidence of side effects were assessed. Results: The majority of patients (12/13, 92.3%) experienced more than one treatment-associated complication; one patient dropped out of the study due to intolerable myalgia. The results of 6MWD test (from 299.2 ± 56.2 m to 363.8 ± 86.5 m, p = 0.039) and tricuspid regurgitation (TR) pressure gradient (from 84.7 ± 20.5 mmHg to 61.6 ± 24.0 mmHg, p = 0.018) improved and remained improved after selexipag treatment in 12 patients. Based on the results of a non-invasive risk assessment, 8 (66.7%) patients showed improvement, 3 (25.0%) showed no interval change, and the status of one patient (8.3%) deteriorated. Moreover, compared to patients treated with a low dosage, patients treated with a medium-to-high dosage showed a greater increase in 6MWD results (88.3 ± 26.4 m vs. 55.3 ± 27.6 m, p = 0.043) and a greater reduction in the TR pressure gradient (−33.7 ± 10.9 mmHg vs. −12.5 ± 12.0 mmHg, p = 0.015). Conclusion: Selexipag is an efficient pulmonary vasodilator as add-on therapy in treating CHD-associated PAH.
Highlights
Pulmonary arterial hypertension (PAH) associated with congenital heart disease (CHD) (PAH-CHD) is one of the most common types of PAH [1,2,3]
CHD, 2021, vol.16, no.3 stratified by shunt lesions consists of heterogeneous subgroups [6,7], as follows: (1) Eisenmenger syndrome, (2) PAH associated with predominant systemic-to-pulmonary shunts, (3) PAH with small/coincidental defects, and (4) PAH after repair
A phase III trial (GRIPHON) has shown that, compared to placebo, selexipag reduced the risk of mortality or major complications associated with PAH [11] and improved the results of a 6-minute walking distance test
Summary
Pulmonary arterial hypertension (PAH) associated with congenital heart disease (CHD) (PAH-CHD) is one of the most common types of PAH [1,2,3]. Selexipag (Uptravi, Actelion Pharmaceuticals) is a highly selective, oral, long-acting prostacyclin receptor (IP receptor) agonist [9,10] It was approved by the Food and Drug Administration in December 2015 and by the European Medicines Agency in May 2016, allowing for marketing of an oral combination therapy, targeting the prostacyclin pathway. Regarding treatment of PAH-CHD, post-hoc analysis of the GRIPHON study data has demonstrated that selexipag may delay disease progression and is welltolerated [17]; this trial only included corrected PAH-CHD patients. This study examined the efficacy and safety of selexipag in PAH-CHD patients, including patients with Eisenmenger syndrome and partially repaired CHD
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