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National Centre
ADAPT stands for Antitrypsin Deficiency Assessment and Programme for Treatment. It was established in 1996 to resurrect the previous UK registry of ?1 antitrypsin patients which had ceased some 6-10 years earlier. The major purpose was initially to develop a comprehensive database for alpha-1-antitrypsin deficient patients within the UK and to provide information on the current level of knowledge of the condition. The centre is supported primarily by a non-commercial grant from Bayer Plc and also 2 European Union grants to standardize methodology for patient assessment and management and foster International collaboration throughout Europe, Australasia and South Africa.
It is a dedicated research facility for alpha-1-antitrypsin deficiency consisting of several clinic rooms, patient research areas and a seminar meeting room. It is adjacent to a comprehensive physiological laboratory and linked to basic research laboratories with expertise in lung biochemistry, microbiology, cell and molecular biology.
The ADAPT centre has close links with Professor E. J. Campbell who runs the alpha-1-antitrypsin deficiency detection centre at the University of Utah, Salt Lake City, Utah USA.
Principal Investigator
ADAPT is headed by Professor R. A. Stockley. M.D., D.Sc., F.R.C.P. He is a Professor
of Medicine at the Queen Elizabeth Hospital which is the main teaching hospital
associated with the University of Birmingham. Professor Stockley is a scientific
clinician with a longstanding interest in lung biochemistry and cell biology
with particular reference to alpha-1-antitrypsin deficiency since the mid 1970s.
He is currently Chairman of the British Thoracic Society Research Committee
on alpha-1-antitrypsin deficiency, Chairman of the Co-ordinating Committee and
Council of AIR, Chairman of the ERS task force on the standards of care of lung
disease in alpha-1-antitrypsin deficiency and a member of the Steering Committee
of the ERS/ATS alpha-1-antitrypsin deficiency task force.
Goals and Activities of the National
Registry
The national registry was established to develop in depth knowledge about alpha-1-antitrypsin
deficiency by seeing and assessing as many patients as possible in a single
centre. The long-term aims were (a) to develop standards of care for the management
of patients with alpha-1-antitrypsin deficiency, (b) to understand the implications
of the disease on patients health, (c) to provide appropriate counselling and
information for patients with deficiency and (d) specifically to develop a research
knowledge and the design of robust clinical trial.
To achieve these goals several steps
had to be undertaken:
| 1. | An extensive baseline had to be established to set the standard for the assessment, management and follow up of alpha-1-antitrypsin deficiency. |
| 2. | A key objective was to determine the relationship between the physiological and radiological features of alpha-1-antitrypsin deficiency and the clinical signs, symptoms and particularly the health of the patients. |
| 3. | Because alpha-1-antitrypsin is a protective protein it plays a key role not only on a day-to-day basis, but particularly during acute infective exacerbations. A further major aim of the programme therefore was to assess the nature of the exacerbations and their role and effects on patients health and progression of the lung disease. |
| 4. | The programme aimed to develop new ways of measuring the effect of alpha-1-antitrypsin deficiency on the lungs enabling its progression to be monitored much more accurately than using conventional methods. |
| 5. | With these initial goals as the background robust clinical trials based on patients symptoms and related to accurate clinical and scientific measurement could be designed and executed. |
Patients have been recruited from our own clinic, fellow clinicians, some patients have referred themselves and others have been detected through the testing of relatives.
The ADAPT programme currently knows of 697 number of patients who are on the registry. There are 434 on the assessment programme and patients are continually being recruited to the database which is increasing by 36 new patients per 3 months (see graph). The ADAPT database is currently the largest most comprehensive database available worldwide where all patients are seen and assessed within a single centre. This clinical experience provides a unique opportunity to understand how the deficiency affects patients and to see patterns emerge that indicate important clinical features of the condition that will determine the long-term management programme.
Participation in the Assessment Programme
All patients who are known to the centre or seen within the centre are added
to the ADAPT database. Those visiting Birmingham are counselled on the genetics
and informed of the current state of knowledge about the condition and its management.
Most of those visiting Birmingham are assessed in detail on at least 1 occasion,
which includes an extensive history of previous illnesses, symptoms, occupation,
smoking, infections and other factors that are related to lung disease; including
family history. The patients undergo full examination and then routine lung
function testing but the data is then assessed in far more detail than is routinely
carried out. In addition, all patients undergo high resolution CT scanning to
identify the presence, nature and extent of lung disease with particular reference
to the amount of emphysema. Patients who produce sputum have this collected
and some patients undergo routine exercise testing. At the end of this assessment
programme the overall treatment is reviewed and where necessary treatment is
optimised to improve symptoms. Patients are given specific advice on the management
of their lung condition as well as advice on its implications for their own
immediate and nearby family.
Patients on the assessment programme are usually followed up on an annual basis. The database that has been established is comprehensive and locked into a computer programme for protection of confidentiality. As the information collected is more extensive and in more detail than is routinely carried out in the clinical assessment of patients seen in ordinary clinics it remains a research programme and all patients participating are asked to give informed consent for the information to be collected.
Some patients take part in other studies that are also voluntary and subject to a separate consent form. These projects include the monitoring of acute exacerbations, a previous trial of intravenous treatment with transgenic alpha-1-antitrypsin produced by PPL and finally a short-term study on the biochemical effects of Prolastin on lung inflammation.
To Obtain More Information
To obtain more information you should contact Rebecca Lewis at the ADAPT Centre
on 0121 697 8257.
The mailing address
of the ADAPT Centre is:
ADAPT Project,
1st Floor Nuffield House,
Queen Elizabeth Hospital,
Edgbaston,
Birmingham. B15 2TH UK
Tel: 0121 697 8257
Fax: 0121 697 8256
E-mail: rebecca.lewis2@uhb.nhs.uk
National website: to be established
© 2003, Alpha One International Registry. All rights reserved.
