Quality of Life and Mortality Outcomes for Augmentation Naive and Augmented Patients with Severe Alpha-1 Antitrypsin Deficiency.

ABSTRACT:

Background

Intravenous alpha-1 antitrypsin (AAT) augmentation therapy is the only specific treatment available for alpha-1 antitrypsin deficiency (AATD)-related lung disease. It is widely used worldwide but remains unavailable to patients with AATD in the United Kingdom. While randomized trials of augmentation therapy have demonstrated biochemical efficacy and lung tissue preservation using computed tomography (CT) densitometry, these studies were not adequately powered to demonstrate effectiveness in well-accepted clinical endpoints such as quality of life (QOL) or survival. We used large, prospectively followed AATD patient populations in the United States and United Kingdom to explore these important clinical endpoints.

Methods

Our inclusion criterion was adults with seve

SUBMITTER: Ellis PR 

PROVIDER: S-EPMC10392875 | biostudies-literature | 2023 Apr

REPOSITORIES: biostudies-literature