Author
Prof. Dr. Rainer Ganschow, Director of the Clinic and Polyclinic for General Pediatrics Bonn
Since the registry was established, only 170 children from Germany have been recorded. Reports from the relevant pediatric hepatology centers have therefore been sporadic.
Given that liver involvement in adult-onset AATD has rightly received much more attention, the pediatric Alpha-1 centers expressed a desire to restructure the registry to learn from the data both clinically and scientifically. The pediatric centers pledged close collaboration and welcomed the creation of a shared database to better advise patients and families in the future. It is quite possible that pediatric studies (similar to those in adults, such as siRNA studies) will be offered in the near future, and we in Germany should be optimally prepared for such studies. Our team in Bonn is already collaborating clinically and scientifically with Prof. Strnad's team in Aachen, and it would be desirable to extend this cooperation to other pediatric centers. In my view, the Aachen team is currently the world's leading authority on liver involvement in AATD, and we can certainly learn a great deal from internists for the care of our patients, and vice versa.
For our 46 homozygous patients in Bonn, we have already established a very detailed database, which we intend to use as a model for the future direction of the registry. Following approval from our local ethics committee, we are permitted to transfer the data of the 170 patients currently entered into the registry to Bonn. There is a consensus among the pediatric centers that our team in Bonn will take the lead in maintaining the registry going forward, in close collaboration with the other centers. Reorganizing the pediatric registry will certainly require considerable effort. Data entry should be as simple and decentralized as possible, ideally with some data being entered by the parents themselves. We have already secured the services of a computer scientist who will develop the necessary technical infrastructure and create a corresponding website for us in the coming months.
We hope to be able to report on initial successful steps soon.
