Functional Class and Health-Related Quality of Life in Patients With Pulmonary Arterial Hypertension Associated With Congenital Heart Disease: Findings From a Real-World Study in the US

OBJECTIVES: Pulmonary arterial hypertension is a progressive, incurable complication of some forms of congenital heart disease (PAH-CHD), imposing a high patient burden and impact on daily life. This study aimed to understand the relationship between functional class (FC) and health-related quality of life (HRQoL) for PAH-CHD patients across US real-world settings.

METHODS: Data were drawn from the US Adelphi Real World PAH-CHD Disease Specific Programme 2022, a point-in-time survey of PAH-CHD patients. Enrolled physicians provided evaluation of New York Heart Association (NYHA) FC, a standard classification for PAH severity. The same patients completed the EQ-5D visual analogue scale (EQ-5D-VAS, range 0-100), EQ-5D utility score (range 0-1) [higher scores indicating a better health state] and emPHasis-10 (e10), a disease specific HRQoL measure [range 0-50, higher scores indicating poorer HRQoL]. Analyses were performed by overall population, and FC. Tukey tests compared significance across FC group I vs II/III (combined due to low FC III base).

RESULTS: 56 PAH-CHD patients provided HRQoL data. Mean age was 38.2 years (SD, 17.6), 70% were male, 25% were classified as FC I and 75% as FC II/III. Mean VAS and utility scores were 69.8 and 0.7, respectively. Lower scores were observed with higher FC, indicating poorer HRQoL (VAS [FC I, 80.9; FC II/III, 66.1] Utility [FC I, 0.9; FC II/III, 0.7]). Mean e10 score was 27.0. The e10 minimally important difference of 6 points was exceeded across FC groups (FC I, 15.9; FC II/III, 30.7). All three HRQoL scores differed significantly between FC groups I and II/III (p<0.05).

CONCLUSIONS: PAH-CHD patients reported high HRQoL burden, associated with worsening functional limitation. Compared to demographic matched general populations (VAS, 84.4; Utility, 0.9), patient scores revealed a high burden of PAH-CHD on HRQoL. Results demonstrated the importance of regular HRQoL assessment. Further research is needed to identify therapeutic targets to improve HRQoL.