A recent study conducted by RCSI University of Medicine and Health Sciences has revealed that augmentation therapy can have significant survival benefits for individuals with alpha-1 antitrypsin deficiency (AATD). AATD is a genetic condition wherein individuals are born with low levels of a protective protein called alpha-1 antitrypsin, which can lead to severe lung and liver diseases. Previous studies on augmentation therapy showed it could slow down lung damage, but the long-term survival benefits were unclear. However, the new study focused on less seriously ill patients and found that boosting levels of alpha-1 antitrypsin improved survival rates.
The research involved 615 patients with severe AATD and emphysema from Ireland, Switzerland, and Austria. The results, published in the American Journal of Respiratory and Critical Care Medicine, showed that augmentation therapy had a clear survival advantage, especially in Switzerland and Austria, where it is reimbursed. On the other hand, in Ireland, where the therapy is not reimbursed, patients did not experience the same benefits. These findings highlight the urgent need to make augmentation therapy accessible to all AATD patients in Ireland and across Europe.
This study provides real-world evidence that augmentation therapy can significantly benefit individuals with emphysema caused by severe AATD. The evidence supports the reimbursement of this therapy in Ireland and other European countries. Currently, augmentation therapy is not reimbursed, and this study emphasizes the importance of making it available as it is the only specific treatment for the lung disease associated with AATD. Additionally, the study underscores the need for early detection of AATD and the inclusion of younger and healthier patients in future studies on augmentation therapy. The ultimate goal is to intervene before severe lung disease occurs, potentially through newborn screening for AATD.
The international collaboration for this study was made possible by funding from the US Alpha-1 Foundation. These findings have significant implications for the healthcare industry and the treatment options available for individuals with AATD.
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1. Source: Coherent Market Insights, Public sources, Desk research
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