Epidemiologic and Biostatistical Support to AlphaNet

Abstract: Epidemiologic and Biostatistical Support to AlphaNet Alpha-1 antitrypsin deficiency (A1AD) is the most common known genetic cause of chronic obstructive pulmonary disease (COPD) (1) . Since the discovery of this deficiency by Laurell and Eriksson in 1962 (2) and its link to emphysema, it has been the focus of a great deal of research, including potential therapies. A major advancement in the treatment of A1AD was the development of augmentation therapy that was developed in 1980 and became commercially available in the late 1980s (3). In the early days of augmentation therapy access to the replacement product was limited and patients with A1AD faced a number of obstacles with regard to therapy and disease management. These gaps were addressed by the formation of the nonprofit company Alphanet and the Alpha-1 Foundation in 1995. While Alphanet had its origins as a Home Health Care Company it changed into a virtual “health management organization” over time and currently manages over 3,500 patients with A1AD. As part of this case management, data are collected on patients being cared for. These data have already resulted in several key publications looking at A1AD (4, 5). In addition, Alphanet has started a trial that compares standard therapy to a rehabilitation program in patients on augmentation therapy. The availability of both baseline, follow-up, and intervention-related data provides a wonderful opportunity to increase our understanding of the natural history and treatment response of patients with A1AD. We are proposing a multiyear partnership between Dr. Mannino’s research group at the University of Kentucky College of Public Health and AlphaNet to provide biostatistical and epidemiologic support to AlphaNet. The proposed work will be done collaboratively with key researchers in AlphaNet and the Alpha-1 Foundation, including Dr. Robert Sandhaus, Dr. Charlie Strange, and others as appropriate. The Aims for the initial year of this agreement are noted below.