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. 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.