Association Between a Novel Adult Congenital Heart Disease-Specific Patient-Reported Health Status Metric and Objective Clinical Status

BACKGROUND: Although disease-specific patient-reported outcomes (PROs) are well accepted as direct and indirect clinical outcomes in various diseases, data on PRO performance in adult congenital heart disease (ACHD) are limited to nondisease-specific metrics. We, therefore, investigated the association between responses to a novel ACHD-specific PRO metric and both clinical variables and gold standard PROs. METHODS: We assessed the association between patient-perceived health status as assessed by the summary score of the Adult Congenital Heart Disease Patient-Reported Outcome Version 1 (ACHD PRO V.1) and hospital admission within the preceding 5 years in the English-speaking subgroup of an international cohort of patients with ACHD enrolled in APPROACH IS-II between February 10, 2019, and December 14, 2022. We also examined the relationship between ACHD PRO V.1 domain scores and domain-relevant clinical variables, the Linear Analog Scale Quality of Life, the Patient Health Questionnaire-8, and the Generalized Anxiety Disorder-7. RESULTS: The analysis includes 333 patients. Both unadjusted and adjusted models revealed that worse patient-perceived health status was associated with unplanned hospital admission (adjusted β, −5.7 [95% CI, −9.9 to −1.6]; P =0.007). Univariate analysis indicated a strong association between ACHD PRO V.1 domains and relevant clinical variables. A moderate correlation was found between the QOL and anxiety/depression domains and their corresponding gold standard ( r =0.467, P <0.001 with the Linear Analog Scale Quality of Life; r =−0.581, P <0.001 with the Patient Health Questionnaire-8; and r =−0.540, P <0.001 with the Generalized Anxiety Disorder-7). CONCLUSIONS: A lower ACHD PRO V.1 summary score is associated with a history of unplanned cardiac admission. In addition, the ACHD PRO V.1 domains show good content validity by comparison with relevant clinical conditions and gold standard PROs. These data support the use of the ACHD PRO V.1 as a direct and surrogate clinical outcome in ACHD.