General Introduction & Background

Alpha-1-antitrypsin (A1 AT) is produced in the liver and is secreted into the circulation. It is the major serum and critical lung inhibitor of serine proteases, particularly of the enzyme neutrophil elastase. Inadequately controlled neutrophil elastase has been implicated as a cause of tissue destruction in the lung, resulting in emphysema and bronchial disease, which are the two major features of chronic obstructive pulmonary disease (COPD). Inherited deficiency of alpha1-AT (AATD) is predominantly a European disorder originating in Scandinavia, and emigrants have carried the disorder to all other parts in the world. It is as common as cystic fibrosis and affects between 1 in 1700 and 1 in 5000 individuals, depending upon the local prevalence of individuals from Scandinavian origin. Although affected individuals have a predisposition to the development of cirrhosis, liver cancer or vasculitis, they most commonly present with early onset of rapidly progressive emphysema.

A clinical trial to prove the efficacy of a1 AT infusion has succeeded in 2015 and the results were published in The Lancet on July 25, 2015. However, it remains currently impossible to prospectively select individuals in this population to identify who will develop disabling lung disease, and who have a critical need for early preventive therapy with alpha-1-antitrypsin weekly intravenous treatment.


In 1996 the World Health Organization in Geneva held an international meeting to clarify the current state of the art in a1 ATD and to develop recommendations for future work. The meeting was summarized in the 1997 memorandum (1) indicating that specific research was required to identify the risk factors and clarify the prognosis of the lung disease. In order to bring these recommendations to fruition it was recognized that an international registry was required. In 1998 a provisional database was agreed and established in Malmo under the direction of Professor S. Eriksson who first described a1 ATD in 1963. Since then it has been shown that electronic transfer of the basic database is feasible from individual countries and as of 1999 more than 300 new patients have been added to this registry. In the year 2000 increasing at a pace of 20 newly identified individuals each month to over 5000 in June 2016.

In 1999 the Alpha a1 International Registry (AIR) was formally founded and the statutes and rules regarding intellectual property rights were established and legalized in The Netherlands as a foundation. An administrative structure was developed to include a Chairman, Treasurer, Secretary and two other members of a Coordinating Committee. A council of management was established to include 17 representatives of 12 international a1 ATD registries.
Thus we have brought the central feature of the WHO recommendations to fruition and have established the base upon which it will be possible to address the other main recommendations.

One of the missions of AIR is to share and disseminate results of original research in the field of alpha-1-antitrypsin (AAT). For this purpose, a biannual scientific meeting are organized in collaboration with the Alpha One Foundation from the USA.

In this meeting, researchers working in the different aspects of this genetic disorder will be invited to participate. Aspects to be covered will include clinical manifestations, e.g., lung and liver disease, genetics, biochemistry, epidemiology, ethics, pharmacoeconomics and new drug development.

The structure of the meeting include invited lectures with up-to-date information by recognized researchers, and presentation of original work in the form of posters, discussion or oral presentations. In addition, patients are able to speak about general issues in the format of a symposium.

The duration of the meeting is two days, and approximately 60% of the time will be dedicated to update lectures and 40% to free communications.

The organization of the meeting will be responsibility of a team composed of the chairman of AIR, one member of the council, the organizer of the last meeting and the local representative. The scientific program will be proposed by an ad hoc scientific committee composed by 6-7 scientific leaders in the field of alpha-1-antitrypsin, at least 3 of them selected from the AIR council (chairman and the local representative should be among them). The election of members of the scientific committee is the exclusive responsibility of the AIR council.

The selection of the organizing agency will be the responsibility of the local organizer under the approval of the organizing team.

The relationship between the organizing team (representing AIR), the sponsors and the organizing agency will be regulated by contract, as depicted in the figure below.

All funding will be accepted only as unrestricted grants.