On behalf of the working group COPD, the BERS is pleased to announce that new initiatives are taken in the field of alpha-1 antitrypsin deficiency (AATD).
What is the EARCO ?
First of all, we would like to introduce the European Alpha-1-Research Collaboration (EARCO). EARCO is a clinical research collaboration establishing a worldwide network to promote clinical research and education in alpha-1 antitrypsin deficiency (AATD). The core project is the AATD Registry, an open collaboration for all investigators caring for patients with AATD. This registry has the ambition to collect prospective follow-up data of thousands of patients with AATD. These data will lead to a better understanding of the natural history of the disease, risk factors and the role of therapy in patients with AATD. The databank may become the missing piece of evidence for augmentation therapy in a subgroup of subjects in Belgium with an inherited progressive lung disease.
Registration of patients
Patients can be registered through participating hospitals, after approval of your local ethics committee. An alternative is to refer these rare patients to an expert center for inclusion and follow-up. At this stage, UZ Leuven, UZ Gent and Hospital Erasmus Brussels are happy to include your subject, but if you are interested in becoming a registering center, please contact the national coordination in UZ Leuven for initiation.
MORE INFORMATION CAN BE FOUND ON THE EARCO WEBSITE
Two new studies
In the context of the EARCO project, we would also like to announce that there are currently 2 new studies recruiting AATD patients in Belgium. In theses studies AATD patients have a chance to receive disease specific medication (and if proven beneficial, a potential continuation through open label).
1. Astraeus study (Mereo BioPharma): oral administration of alvelestat (elastase inhibitor) A Phase 2, proof-of-concept, multicentre, double-blind, randomised, dose-ascending, sequential group, placebo-controlled study to evaluate the mechanistic effect, safety, and tolerability of 12 weeks twice daily oral administration of alvelestat (MPH966) in participants with alpha-1 (PiZZ or null genotype/phenotype) antitrypsin deficiency.
2. InnovAATe study (Kamada): 2% human alpha 1 antitrypsin by inhalation A prospective phase 3 multi-center, placebo controlled, double blind study to evaluate the efficacy and safety of “Kamada-AAT for Inhalation” 80 mg per day during 104 weeks in adult patients with congenital Alpha-1 Antitrypsin Deficiency with moderate airflow limitation (40% ≤ FEV1 ≤ 80% of predicted; FEV1/FVC ≤ 70%).
We hope that these initiatives will encourage you to think about the disease with every new COPD diagnosis. If AATD is diagnosed in your patient, you may want to discuss the potential of follow-up in a dedicated center. As we hope that new drug interventions studies may finally offer a therapeutic solution in patients with AATD, we would be very pleased with your referral.
In case you need more information or documentation please do not hesitate to contact us (firstname.lastname@example.org).
On behalf of the working group COPD of the BeRS & the patient organization Alpha-1 Plus Belgium
Prof Wim Janssens, UZ Leuven - Prof Eric Derom, UZ Gent - Dr. Silvia Perez-Bogerd, Hospital Erasme Brussels - Prof Didier Cataldo, University of Liège - Dr Frank Willersin, Alpha-1 Plus Belgium