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What are the goals of augmentation therapy?

The basic goal of augmentation therapy is to increase the level of alpha-1 protein in the lungs. Alpha-1 antitrypsin protects the lungs from the destructive effects of neutrophil elastase, an enzyme released by our body’s white blood cells as they respond to inflammation or infection.
The ultimate goal is to slow or stop the progression of lung destruction by replacing the deficient protein. The therapy cannot restore lost lung function — nor is it considered a cure. There is also some evidence that augmentation therapy can reduce the frequency and severity of pulmonary exacerbations (flare-ups of lung disease) and it appears to be an effective treatment for the Alpha-1 related skin disease, Necrotizing Panniculitis.

Alpha-1 Foundation

Augmentation therapy (also called replacement therapy) infusions are intended to augment (add to) the amount of alpha-1 antitrypsin protein (AAT) floating in the blood and bathing the tissues of the body in people with lung disease related to Alpha-1 Antitrypsin Deficiency. Although some people report that they notice improvements in their health when on augmentation therapy, and there is some evidence for a decrease in the number of lung infections in individuals receiving augmentation therapy, the primary aim of this therapy is to reduce the rate of decline of lung function towards normal and, therefore, improve the long-term quality of life and even the lifespan of those with lung disease due to Alpha-1.

Everyone loses lung function during their adult life, whether they have Alpha-1 or not. Alphas with lung disease lose their lung function at a more rapid rate than normal. If augmentation therapy is effective, it will be expected to slow this increased rate of decline, regardless of the severity of the underlying lung disease.

Alpha-1 Lung Disease Questions & Answers – Alpha-1 Foundation