OBJECTIVES: The primary objective of the BREEZE clinical trial was to evaluate the safety and tolerability of treprostinil inhalation powder via a dry-powder inhaler (DPI) in PAH patients treated with treprostinil inhalation solution via a nebulizer (NEB). This analysis focused on patient reported outcomes.

METHODS: Two patient questionnaires were included as secondary endpoints: 1) The Preference Questionnaire for Inhaled Treprostinil Devices (PQ-ITD) and 2) The PAH Symptoms and Impact Questionnaire (PAH-SYMPACT). The PQ-ITD was administered at week 0 and week 3 and provides twelve statements on device satisfaction allowing for five responses from “strongly disagree” to “strongly agree”. The PAH-SYMPACT is a validated PAH questionnaire focused on four domains: cardiopulmonary symptoms, cardiovascular symptoms, physical impacts, and cognitive/emotional impacts, and was administered at weeks 0, 3, and 11. For continuous outcomes the paired t-test was used and for categorical outcomes the Cochran-Mantel-Haenszel test was used.

RESULTS: 51 patients enrolled in the BREEZE study and switched from inhaled treprostinil via the NEB to DPI. Mean (SD) age was 55.9 years (13.4) and 84.3% of patients were female. Mean (SD) time since first PAH diagnosis was 8.7 (6.5) years. 95.7% of patients reported overall satisfaction with DPI compared to 31.4% for the NEB (p<0.001). Patients reported satisfaction with specific aspects of the DPI such as size of inhaler (95.7%), ease of use (97.8%) and number of breaths required (93.5%); all items statistically significant compared to NEB (p<0.001). For the PAH-SYMPACT, mean changes from baseline were improved for all domain scores. Significant improvements for physical impacts were observed at week 3 (mean change -0.14, p=0.044) and week 11 (mean change -0.21, p=0.043) and for cognitive/emotional impacts at week 3 (mean change -0.17, p=0.005).

CONCLUSIONS: PAH patients in the BREEZE study reported high satisfaction with DPI and showed improvement in quality of life.