OBJECTIVES: PAH is a rare, progressive cardiopulmonary disorder that requires a multifaceted management strategy and active involvement of patients. This study measured patient preferences and identified preference heterogeneity for PAH-specific treatment.

METHODS: Adult PAH patients with slight to marked limitations during physical activity were recruited through a patient organization in Germany between March and July 2022. Participants completed an online, best-worst scaling case 3 survey in which they chose between three treatment profiles (no opt-out) which were characterized by survival rate, unplanned PAH-related hospitalizations, and physical activity limitations, digestive symptoms, pain, and headache. Participants completed 12 tasks in random order (120 tasks in 10 blocks) plus a fixed task to test dominance. Preference heterogeneity was assessed using latent class analysis. Model fit tests were used to select number of classes.

RESULTS: 83 PAH patients (76% female) completed the survey and assigned the greatest importance to physical activity limitations and survival. The best-fit model revealed 4 classes of patients with similar response patterns. Class 1 (19% of participants) assigned high importance to all attributes, Class 2 (34%) to physical activity. Class 3 (30%) on survival and physical activity, and Class 4 (17%) assigned most importance to survival. No differences were found in sociodemographic characteristics across the classes. Compared to Class 1-3, Class 4 was most likely to report having marked physical activity limitations (79%) and needing daily help (100%), while reporting higher daily activity levels as normal [walking >1 kilometer (71%) or climbing several flights of stairs (50%)].

CONCLUSIONS: This first patient preference study in a PAH population suggests that physical activity limitations and survival matter most; however, heterogeneity across latent classes may be associated with differences in normal daily physical activities and activity limitations. Patient preferences should be considered in personalized treatment decisions.