The primary objective of this registry will be to characterize the clinical and laboratory course of severe congential alpha1- antitrypsin deficiency whether or not the patient is undergoing long-term replacement therapy. Severe congenital alpha1-antitrypsin is associated with the early onset of emphysema, usually by the third decade of life. One approach to correct this deficiency is through replacement with alpha1-antitrypsin (referred to as alpha1-proteinase (A1 Pi) inhibitor in its purified form). There currently is no data base available for estimating the degree of clinical benefit if any of A1 Pi. The slow progression of emphysema and lack of an adequate control group have made it difficult to evaluate the proteinase inhibitor through a controlled clinical trial. A registry is an alternative to collect data on the effect of long- term replacement therapy with A1 Pi on rate of decline of lung function. The registry will also include individuals who are not receiving the replacement therapy in order to obtain a better knowledge of the rate of decline of lung function associated with the congenital deficiency for alpha1-antitrypsin.

Agency
National Institute of Health (NIH)
Institute
Division of Lung Diseases (NHLBI)
Type
Research and Development Contracts (N01)
Project #
N01HR086036-008
Application #
2317410
Study Section
Project Start
1988-09-30
Project End
1994-09-29
Budget Start
1992-04-24
Budget End
1993-10-30
Support Year
Fiscal Year
1992
Total Cost
Indirect Cost
Name
Cleveland Clinic Lerner
Department
Type
DUNS #
017730458
City
Cleveland
State
OH
Country
United States
Zip Code
44195
Stoller, James K; Tomashefski Jr, Joseph; Crystal, Ronald G et al. (2005) Mortality in individuals with severe deficiency of alpha1-antitrypsin: findings from the National Heart, Lung, and Blood Institute Registry. Chest 127:1196-204
Tomashefski Jr, Joseph F; Crystal, Ronald G; Wiedemann, Herbert P et al. (2004) The bronchopulmonary pathology of alpha-1 antitrypsin (AAT) deficiency: findings of the Death Review Committee of the national registry for individuals with Severe Deficiency of Alpha-1 Antitrypsin. Hum Pathol 35:1452-61
Eden, Edward; Hammel, Jeffrey; Rouhani, Farshid N et al. (2003) Asthma features in severe alpha1-antitrypsin deficiency: experience of the National Heart, Lung, and Blood Institute Registry. Chest 123:765-71
Schluchter, M D; Stoller, J K; Barker, A F et al. (2000) Feasibility of a clinical trial of augmentation therapy for alpha(1)-antitrypsin deficiency. The Alpha 1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med 161:796-801
(1998) Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. The Alpha-1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med 158:49-59
McElvaney, N G; Stoller, J K; Buist, A S et al. (1997) Baseline characteristics of enrollees in the National Heart, Lung and Blood Institute Registry of alpha 1-antitrypsin deficiency. Alpha 1-Antitrypsin Deficiency Registry Study Group. Chest 111:394-403
Turino, G M; Barker, A F; Brantly, M L et al. (1996) Clinical features of individuals with PI*SZ phenotype of alpha 1-antitrypsin deficiency. alpha 1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med 154:1718-25
Schluchter, M D (1992) Methods for the analysis of informatively censored longitudinal data. Stat Med 11:1861-70