Patient Outcomes - AATD
Research type
Research Study
Full title
Outcomes that define disease severity in alpha-1 Antitrypsin Deficiency (AATD); the design of a patient-centred treatment pathway.
IRAS ID
233675
Contact name
Alice Turner
Contact email
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
Alpha-1 Antitrypsin Deficiency (AATD) is an inherited condition associated with lung and liver damage. It is unclear which medical investigations are essential to monitor their disease or what aspects of disease are most important to people with AATD. Clarity on these issues will assist in the design of future treatment trials and rationalise the number of investigations that patients undergo. Emphasis is consistently put on many physical tests that patients often report being difficult and uncomfortable to do yet these tests may not be the best for monitoring their disease. By interviewing patients from across the country with AATD and the people that work in specialist AATD services we hope to get a better understanding what measurements they feel are important.
Prospective data from the Birmingham AATD patient cohort will be collected with the most up to date measurements available including more patient centred outcomes. This will help identify which tests are the best at identifying early disease and use these to help predict when patients start to deteriorate and treatments should be started. We will also evaluate how these tests and treatments can be used in specialist AATD clinics, to ensure health care professionals and patients find them useful and practical within the NHS.
We will use the UHB NHS AATD service to identify the majority of patients. The Birmingham site has acted as the main coordinating site in the UK for the national NIHR rare diseases collaboration and our partner sites across the UK will notify potential patients and act as participant identification sites. All recruitment to the interviews are to be completed by May 2018 and we would plan to continue to recruit participants to the cohort for 24 months following ethical approval.
REC name
South Central - Oxford C Research Ethics Committee
REC reference
18/SC/0541
Date of REC Opinion
24 Oct 2018
REC opinion
Further Information Favourable Opinion