Objective This study assessed the long-term effects of triple therapy with prostanoids on patients with pulmonary arterial hypertension associated with congenital heart disease (PAH-CHD), as there is limited information on the safety and efficacy of this treatment approach.
Methods A retrospective cohort study was conducted on patients with PAH-CHD who were actively followed up at our centre. All patients were already receiving dual combination therapy at maximum doses. Clinical characteristics, including functional class (FC), 6-minute walking test distance (6MWTD) and N-terminal pro-brain natriuretic peptide (NT-proBNP) levels, were documented before initiating triple therapy and annually for a 2-year follow-up period.
Results A total of 60 patients were included in the study, with a median age of 41 years and 68% being women. Of these, 32 had Eisenmenger syndrome, 9 had coincidental shunts, 18 had postoperative PAH and 1 had a significant left-to-right shunt. After 1 year of triple combination initiation, a significant improvement in 6MWTD was observed (406 vs 450; p=0.0027), which was maintained at the 2-year follow-up. FC improved in 79% of patients at 1 year and remained stable in 76% at 2 years. NT-proBNP levels decreased significantly by 2 years, with an average reduction of 199 ng/L. Side effects were experienced by 33.3% of patients but were mostly mild and manageable. Subgroup analysis showed greater benefits in patients without Eisenmenger syndrome and those with pre-tricuspid defects.
Conclusions Triple therapy with prostanoids is safe and effective for patients with PAH-CHD, improving FC, 6MWTD and NT-proBNP levels over 2 years. The treatment is particularly beneficial for patients with pre-tricuspid defects and non-Eisenmenger PAH-CHD.
- Heart Defects, Congenital
- Pulmonary Arterial Hypertension
- Hypertension, Pulmonary
- Pharmacology, Clinical
Data availability statement
All data relevant to the study are included in the article or uploaded as supplemental information.
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RL-L and TSdlC contributed equally.
Contributors Conceptualisation—RL-L, TSdlC, FSC and PE-S. Methodology—RL-L, TSdlC, FSC and PE-S. Formal analysis—RL-L. Investigation—RL-L, TSdlC, FSC, IM, WH, ACU, MTV, JFD, AM, FAY and PE-S. Writing (original draft preparation)—RL-L, TSdlC, FSC and PE-S. Writing (review and editing)—RL-L, TSdlC, FSC and PE-S. Supervision—PE-S and FSC. Guarantors—RL-L and TSdlC.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests RL-L, TSdlC, IM, WH, ACU, MTV, AM and PE-S have received study grants from Janssen and MSD.
Patient and public involvement Patients and/or the public were not involved in the design, or conduct, or reporting, or dissemination plans of this research.
Provenance and peer review Not commissioned; externally peer reviewed.
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