Biologic Specimen and Data Repository Information Coordinating Center
Home > Studies > AADR

Resources Available

Study Datasets Only

Study Documents

PDF Data Dictionary (PDF - 680.7 KB)
Forms (HTML)
PDF Using Data (PDF - 734.0 KB)

Persons using assistive technology may not be able to fully access information in the study documents. For assistance, Contact BioLINCC and include the web address and/or publication title in your message.

If you need help accessing information in different file formats such as PDF, XLS, DOC, see Instructions for Downloading Viewers and Players.

Alpha1-Antitrypsin Deficiency Registry (AADR)

Clinical Trials URL: http://www.clinicaltrials.gov/ct...
Study Type: Epidemiology Study
Prepared on October 13, 2008
Last Updated on June 23, 2005
Study Dates: 1988-1999
Consent: Unrestricted Consent
Commercial Use Restrictions: No
NHLBI Division: DCVS
Collection Type: Open BioLINCC Study - See bottom of this webpage for request information

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)