A REGISTRY OF PATIENTS WITH SEVERE DEFICIENCY OF ALPHA(1)-ANTITRYPSIN- DESIGN AND METHODS

The Registry of Patients with Severe Deficiency of Alpha(1)-Antitrypsi n (AlAT) is a multicenter natural history study, with 37 participating clinical centers in the United States (36 centers) and Canada (1 cent er). The study has enrolled 1,129 individuals aged greater than or equ al to 18 years with severe deficiency of AlAT (serum level less than o r equal to 11 mu M), and will follow them longitudinally for up to 7 y ears, characterizing the clinical course of the disease, regardless of whether they are receiving augmentation therapy. Primary outcomes of interest are the yearly decline in FEV(1) and mortality. This article describes the design and structure of the Registry.