AAT deficiency (AATD), a common genetic disorder which predisposes subjects to early onset emphysema, has been associated with significant decrease in HRQoL over time. Chronic AAT augmentation therapy slows the progression of emphysema, however, its effect on HRQoL has not been clearly delineated. The purpose of our study is to evaluate HRQoL in AAT deficient patients receiving augmentation therapy and to compare the HRQoL scores of AAT deficient patients to the general population.
To date, 68 patients with AATD receiving AAT augmentation therapy have been enrolled in this longitudinal observational patient outcomes study. Interviewer-administered questionnaires, which collected demographic, clinical outcomes, healthcare resource utilization, and HRQoL [Short Form-36 Health Survey version 2 (SF-36)] data were administered at baseline (start of augmentation therapy), and at 6-month intervals. Six-month data has been collected on 36 patients.
Analysis of 6-month HRQoL scores indicate no clinically important differences in the eight SF-36 scales and Physical and Mental Component Scores (Figure). Patients with AATD demonstrated clinically important decreases in HRQoL Physical Functioning scores compared to the general population, changes in HRQoL mental health scale scores were not clinically important.
SF-36 scale scores remain stable from baseline to 6-month follow-up indicating a lack of significant disease progression during that timeframe. Trends toward improved HRQoL mental health scores from baseline to 6 months may be indicative of satisfaction with AAT augmentation therapy, resilience in this group of patients with regard to coping strategies and their disease, and/or Hawthorne effect. Overall, AAT deficient patients are disproportionately affected with regard to HRQoL Physical Functioning compared to the general population.
Results suggest patients with AATD have significantly compromised physical health. Although HRQoL appeared stable from baseline to 6-month follow-up, additional longitudinal data are needed to assess the long-term stability of the HRQoL scores. HRQoL assessment can be used as an additional measure of disease severity and progression in AAT deficient patients.
Jennifer Short, Grant monies (from industry related sources) MEB and JS received a grant from Baxter BioScience to conduct data entry and analysis.; Employee KCC and DMG - Baxter BioScience.