Alpha1-Antitrypsin Deficiency Registry (AADR)
Open BioLINCC Study See bottom of this webpage for request information
October 13, 2008
June 23, 2005
Clinical Trials URL
To characterize the clinical and laboratory course of patients with severe alpha 1-antitrypsin deficiency whether or not the patient is undergoing long-term augmentation therapy.
A hereditary disorder, patients with low serum levels of alpha-1-antitrypsin are at an increased risk for the early onset of emphysema. The only approved treatment for alpha-1-antitrypsin deficiency is augmentation therapy using a purified preparation of human alpha-1-antitryspin. Sample sizes for a randomized controlled clinical trial of augmentation therapy were determined to be infeasible; therefore, a multi-center registry was initiated in 1988 to explore the natural history of the disease and the relative efficacy of augmentation therapy in patients with a severe deficiency of alpha-1-antitrypsin.
Eligible subjects included individuals 18 years of age or greater for whom the Central Laboratory confirmed that the serum alpha 1-antitrypsin level is < 11 rnicromolar, or a ZZ genotype confirmed by DNA gene-probe analysis. Individuals with alpha 1-antitrypsin deficiency were accepted into the Registry independent of status of augmentation therapy. However, if the individual was receiving therapy, the serum alpha 1-antitrypsin phenotype and level in the absence of therapy were confirmed by the Central Laboratory. A total of 1,129 subjects were enrolled from 37 clinical centers between March 1989 and October 1992. Follow-up continued through April 1996.
Subjects receiving augmentation therapy had decreased mortality risk during follow-up. FEV1 decline among all subjects did not differ by augmentation therapy; however, among subjects with FEV1 35-49% predicted, FEV1 decline was significantly slower for subjects on augmentation therapy than for those not receiving therapy. (Am J Respir Crit Care Med, 1998; 158:49-59)
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