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Abstract
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
- Pharmacology
- 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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- Heart Defects, Congenital
- Pharmacology
- 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.
Footnotes
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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.
Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.