Monitoring early lung disease and decline in AATD
Research type
Research Study
Full title
Monitoring early lung disease and decline in alpha-1 antitrypsin deficiency
IRAS ID
311818
Contact name
Alice Turner
Contact email
Sponsor organisation
University of Birmingham
Duration of Study in the UK
1 years, 9 months, 31 days
Research summary
Patients with alpha-1 antitrypsin deficiency (AATD) experience symptoms due to progresive lung damage. Due to the expense associated with newer treatments for the disease, it is important to identify lung function decline early in these patients. Currently, lung function is monitored using tests such as spirometry. However, these tests are poor at picking up early disease, and only identify a decline once significant damage has already occured in the lungs.
A pilot study in 2015 identified that some AATD patients with initially normal lung function had abnormal measures of small airways disease. Hence, tests which measure decline of the small airways are likely the most sensitive way to detect early disease changes. Furthermore, tests such as spirometry are dependent on significant effort by the patient; effort independent tests are therefore more helpful in identifying decline as they show less variability.
The patients from the 2015 pilot had a decline in lung function, measured using standard lung function tools. As it has been almost ten years since generation of these data, this study will aim to restudy the same cohort to determine changes in their health, lung function tests and the measures of small airways dysfunction. This will identify whether the patients with abnormal parameters of small airways have continued to decline, and whether this is reflected in their general health. The aim of this study is to assess progression of early disease in this AATD cohort by comparing lung function tests to health status and a repeat CT scan to determine the sensitivity of these measurements in detecting decline. This will also be correlated with plasma samples taken from the patients both at baseline almost ten years ago, and repeated now.
REC name
West Midlands - Black Country Research Ethics Committee
REC reference
25/WM/0083
Date of REC Opinion
4 Jun 2025
REC opinion
Further Information Favourable Opinion