Real-world experiences and treatment patterns among congenital heart disease patients with associated pulmonary vascular disease: Results from a real-world survey in the United States
Background: Limited data exist on the use of drugs in patients with pulmonary arterial hypertension associated with congenital heart disease (PAH-CHD). Therefore, we evaluated their real-world patient journey, including symptomatology, diagnostic pathway, treatment patterns, and guideline adherence....
Saved in:
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2025-08-01
|
| Series: | JHLT Open |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2950133425001211 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1849405685437038592 |
|---|---|
| author | Jordan D. Awerbach, MD, MPH Carly J. Paoli, PharmD, MPH Megan Scott, BSc Gurinderpal Doad, PharmD Julia Harley, BSc Daniel Graham, MSc Mark Small, BSc Sumeet Panjabi, PhD Leigh C. Reardon, MD, FACC |
| author_facet | Jordan D. Awerbach, MD, MPH Carly J. Paoli, PharmD, MPH Megan Scott, BSc Gurinderpal Doad, PharmD Julia Harley, BSc Daniel Graham, MSc Mark Small, BSc Sumeet Panjabi, PhD Leigh C. Reardon, MD, FACC |
| author_sort | Jordan D. Awerbach, MD, MPH |
| collection | DOAJ |
| description | Background: Limited data exist on the use of drugs in patients with pulmonary arterial hypertension associated with congenital heart disease (PAH-CHD). Therefore, we evaluated their real-world patient journey, including symptomatology, diagnostic pathway, treatment patterns, and guideline adherence. Methods: Data were drawn from the Adelphi Real World pulmonary hypertension congenital heart disease Disease Specific Programme (DSP), a cross-sectional survey of clinicians and patients with PAH-CHD or Fontan circulation associated with elevated pulmonary vascular resistance (PVR). Data were collected in the United States from November 2021 to May 2022. Analyses were descriptive. Results: Overall, 51 clinicians reported data for 191 patients with PAH-CHD or Fontan circulation associated with elevated PVR. Fifty-eight patients voluntarily provided data. Overall, 10.5% of patients had a gap of ≥1 year in their disease management. Mean (standard deviation, SD) time from pulmonary hypertension symptom onset to diagnosis/confirmation was 1.7 (2.2) years. Overall, 75.0% of patients underwent a right heart catheterization (RHC) at diagnosis/confirmation. Clinicians reported that 75.9% of patients were prescribed treatment for their PAH-CHD or elevated PVR. Pulmonary hypertension specific therapy was prescribed as combination therapy for 47.6% of patients prescribed pulmonary hypertension specific treatment. Conclusions: Patients experienced delays to diagnosis and gaps in congenital heart disease management. We observed low utilization of RHC and combination therapy. Key unmet needs in this population include more frequent testing to shorten time-to-diagnosis and proactive management with initial combination therapy. |
| format | Article |
| id | doaj-art-3d864e41acf64a69befee513eeafe637 |
| institution | Kabale University |
| issn | 2950-1334 |
| language | English |
| publishDate | 2025-08-01 |
| publisher | Elsevier |
| record_format | Article |
| series | JHLT Open |
| spelling | doaj-art-3d864e41acf64a69befee513eeafe6372025-08-20T03:36:37ZengElsevierJHLT Open2950-13342025-08-01910032610.1016/j.jhlto.2025.100326Real-world experiences and treatment patterns among congenital heart disease patients with associated pulmonary vascular disease: Results from a real-world survey in the United StatesJordan D. Awerbach, MD, MPH0Carly J. Paoli, PharmD, MPH1Megan Scott, BSc2Gurinderpal Doad, PharmD3Julia Harley, BSc4Daniel Graham, MSc5Mark Small, BSc6Sumeet Panjabi, PhD7Leigh C. Reardon, MD, FACC8Center for Heart Care, Phoenix Children’s, Phoenix, AZ; Departments of Internal Medicine and Child Health, University of Arizona College of Medicine-Phoenix, Phoenix, AZ; Corresponding author: Jordan D. Awerbach, MD, MPH, Phoenix Children's Hospital, Thomas Campus 1919 E. Thomas Rd., Phoenix, AZ 85016.Actelion Pharmaceuticals US Inc, San Francisco, CAAdelphi Real World, Bollington, United KingdomActelion Pharmaceuticals US Inc, San Francisco, CAAdelphi Real World, Bollington, United KingdomAdelphi Real World, Bollington, United KingdomAdelphi Real World, Bollington, United KingdomActelion Pharmaceuticals US Inc, San Francisco, CAAhmanson/UCLA Adult Congenital Heart Disease Center and UCLA Mattel Children’s Hospital, University of California Los Angeles, Los Angeles, CABackground: Limited data exist on the use of drugs in patients with pulmonary arterial hypertension associated with congenital heart disease (PAH-CHD). Therefore, we evaluated their real-world patient journey, including symptomatology, diagnostic pathway, treatment patterns, and guideline adherence. Methods: Data were drawn from the Adelphi Real World pulmonary hypertension congenital heart disease Disease Specific Programme (DSP), a cross-sectional survey of clinicians and patients with PAH-CHD or Fontan circulation associated with elevated pulmonary vascular resistance (PVR). Data were collected in the United States from November 2021 to May 2022. Analyses were descriptive. Results: Overall, 51 clinicians reported data for 191 patients with PAH-CHD or Fontan circulation associated with elevated PVR. Fifty-eight patients voluntarily provided data. Overall, 10.5% of patients had a gap of ≥1 year in their disease management. Mean (standard deviation, SD) time from pulmonary hypertension symptom onset to diagnosis/confirmation was 1.7 (2.2) years. Overall, 75.0% of patients underwent a right heart catheterization (RHC) at diagnosis/confirmation. Clinicians reported that 75.9% of patients were prescribed treatment for their PAH-CHD or elevated PVR. Pulmonary hypertension specific therapy was prescribed as combination therapy for 47.6% of patients prescribed pulmonary hypertension specific treatment. Conclusions: Patients experienced delays to diagnosis and gaps in congenital heart disease management. We observed low utilization of RHC and combination therapy. Key unmet needs in this population include more frequent testing to shorten time-to-diagnosis and proactive management with initial combination therapy.http://www.sciencedirect.com/science/article/pii/S2950133425001211Pulmonary arterial hypertensionCongenital heart diseasePulmonary vascular diseaseFontan circulationTreatment patternsReal-world evidence |
| spellingShingle | Jordan D. Awerbach, MD, MPH Carly J. Paoli, PharmD, MPH Megan Scott, BSc Gurinderpal Doad, PharmD Julia Harley, BSc Daniel Graham, MSc Mark Small, BSc Sumeet Panjabi, PhD Leigh C. Reardon, MD, FACC Real-world experiences and treatment patterns among congenital heart disease patients with associated pulmonary vascular disease: Results from a real-world survey in the United States JHLT Open Pulmonary arterial hypertension Congenital heart disease Pulmonary vascular disease Fontan circulation Treatment patterns Real-world evidence |
| title | Real-world experiences and treatment patterns among congenital heart disease patients with associated pulmonary vascular disease: Results from a real-world survey in the United States |
| title_full | Real-world experiences and treatment patterns among congenital heart disease patients with associated pulmonary vascular disease: Results from a real-world survey in the United States |
| title_fullStr | Real-world experiences and treatment patterns among congenital heart disease patients with associated pulmonary vascular disease: Results from a real-world survey in the United States |
| title_full_unstemmed | Real-world experiences and treatment patterns among congenital heart disease patients with associated pulmonary vascular disease: Results from a real-world survey in the United States |
| title_short | Real-world experiences and treatment patterns among congenital heart disease patients with associated pulmonary vascular disease: Results from a real-world survey in the United States |
| title_sort | real world experiences and treatment patterns among congenital heart disease patients with associated pulmonary vascular disease results from a real world survey in the united states |
| topic | Pulmonary arterial hypertension Congenital heart disease Pulmonary vascular disease Fontan circulation Treatment patterns Real-world evidence |
| url | http://www.sciencedirect.com/science/article/pii/S2950133425001211 |
| work_keys_str_mv | AT jordandawerbachmdmph realworldexperiencesandtreatmentpatternsamongcongenitalheartdiseasepatientswithassociatedpulmonaryvasculardiseaseresultsfromarealworldsurveyintheunitedstates AT carlyjpaolipharmdmph realworldexperiencesandtreatmentpatternsamongcongenitalheartdiseasepatientswithassociatedpulmonaryvasculardiseaseresultsfromarealworldsurveyintheunitedstates AT meganscottbsc realworldexperiencesandtreatmentpatternsamongcongenitalheartdiseasepatientswithassociatedpulmonaryvasculardiseaseresultsfromarealworldsurveyintheunitedstates AT gurinderpaldoadpharmd realworldexperiencesandtreatmentpatternsamongcongenitalheartdiseasepatientswithassociatedpulmonaryvasculardiseaseresultsfromarealworldsurveyintheunitedstates AT juliaharleybsc realworldexperiencesandtreatmentpatternsamongcongenitalheartdiseasepatientswithassociatedpulmonaryvasculardiseaseresultsfromarealworldsurveyintheunitedstates AT danielgrahammsc realworldexperiencesandtreatmentpatternsamongcongenitalheartdiseasepatientswithassociatedpulmonaryvasculardiseaseresultsfromarealworldsurveyintheunitedstates AT marksmallbsc realworldexperiencesandtreatmentpatternsamongcongenitalheartdiseasepatientswithassociatedpulmonaryvasculardiseaseresultsfromarealworldsurveyintheunitedstates AT sumeetpanjabiphd realworldexperiencesandtreatmentpatternsamongcongenitalheartdiseasepatientswithassociatedpulmonaryvasculardiseaseresultsfromarealworldsurveyintheunitedstates AT leighcreardonmdfacc realworldexperiencesandtreatmentpatternsamongcongenitalheartdiseasepatientswithassociatedpulmonaryvasculardiseaseresultsfromarealworldsurveyintheunitedstates |