Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences.

The findings underscore a call to make augmentation therapy accessible to patients with AATD in Ireland and more widely across Europe. 
 
People with severe AATD are born with very low levels of a protective protein called alpha-1 antitrypsin, and they can develop serious lung and liver disease. Previous studies of augmentation therapy in AATD showed it can slow the rate of lung damage that leads to emphysema and COPD, but they did not show a long-term benefit on survival. A weakness of these studies was that they included patients with AATD who already had serious lung disease. 
 
The new study gathered a real-word perspective from patients with severe AATD who were not as seriously ill to start with. The researchers examined health data from 615 patients with severe AATD and the lung condition emphysema, tracking their progress over a period of about 10 years by looking at patient registries in Ireland, Switzerland and Austria.
 
The results, published recently in the American Journal of Respiratory and Critical Care Medicine, showed that boosting levels of alpha-1 antitrypsin in these patients improved survival rates.