Alpha1 - Antitrypsin Deficiency Registry (AADR)
Objectives:
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.
Background:
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.
Subjects:
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.
Conclusions:
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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Study Website |
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Study Documentation |
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Data Distribution Agreement |
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