Treatment of lung disease in patients with AATD

Lung disease in AATD patients typically presents earlier than in those with usual COPD and is initially managed in the same way, namely smoking cessation and use of inhaled therapies. AAT augmentation therapy is the only specific treatment available to modify the progression of emphysema and involves intravenous administration of AAT from pooled human plasma. Randomised control trials have consistently demonstrated its efficacy in reducing the decline in computed tomography lung density. Once emphysema has developed, surgical intervention may be indicated. Lung volume reduction can be achieved via direct surgical removal of the hyperinflated area of lung or endobronchial deflation with one-way valves, and can be used a standalone therapy or as a bridge to lung transplant. The use of gene therapy is an exciting prospect in the treatment of AATD-related lung disease, with demonstration of its potential feasibility in multiple early-phase trials in humans. Cite as: Ellis P, Dirksen A, Turner AM. Treatment of lung disease. In: Strnad P, Brantly M, Bals R, eds.