The Registry of Patients with Severe Deficiency of Alpha1-Antitrypsin (AIAT) 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 ≥18 years with severe deficiency of AIAT (serum level ≤11 μM), 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.
All Science Journal Classification (ASJC) codes
- Pulmonary and Respiratory Medicine
- Critical Care and Intensive Care Medicine
- Cardiology and Cardiovascular Medicine