A registry of patients with severe deficiency of alpha 1-antitrypsin. Design and methods. The Alpha 1-Antitrypsin Deficiency Registry Study Group

Abstract

The Registry of Patients with Severe Deficiency of Alpha 1-Antitrypsin (A1AT) is a multicenter natural history study, with 37 participating clinical centers in the United States (36 centers) and Canada (1 center). The study has enrolled 1,129 individuals aged > or = 18 years with severe deficiency of A1AT (serum level < or = 11 microM), and will follow them longitudinally for up to 7 years, characterizing the clinical course of the disease, regardless of whether they are receiving augmentation therapy. Primary outcomes of interest are the yearly decline in FEV1 and mortality. This article describes the design and structure of the Registry.