Alpha 1-antitrypsin deficiency medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Cafer Zorkun, M.D., Ph.D. [2]
Overview
Alpha 1-antitrypsin deficiency (A1AD) may be treated through intravenous infusions derived from donated human plasma.
Medical Therapy
In the United States, Canada, and several European countries, lung-affected A1AD patients may receive intravenous infusions of alpha-1 antitrypsin, derived from donated human plasma. This augmentation therapy is thought to arrest the course of the disease and halt any further damage to the lungs. Long-term studies of the effectiveness of A1AT replacement therapy are not available. It is currently recommended that patients begin augmentation therapy only after the onset of emphysema symptoms.
Augmentation therapy is not appropriate for liver-affected patients; treatment of A1AD-related liver damage focuses on alleviating the symptoms of the disease. In severe cases, liver transplantation may be necessary.
As α1-antitrypsin is an acute phase reactant, its transcription is markedly increased during inflammation elsewhere in response to increased interleukin-1 and 6 and TNFα production. Any treatment that blunts this response, specifically paracetamol (acetaminophen), can delay the accumulation of A1AT polymers in the liver and (hence) cirrhosis. A1AD patients are therefore encouraged to use paracetamol when slightly to moderately ill, even if they would otherwise not have used antipyretics.
- Treatment guidelines of AATD that are similar to COPD include:
- Smoking cessation
- Long-acting bronchodilators
- Preventive vaccinations
- Pulmonary rehabilitation
- Supplemental oxygen if needed
- Lung transplantation
- Augmentation therapy is the specific therapy for Alpha 1-antitrypsin deficiency (A1AD) associated lung disease.
- Augmentation therapy includes intravenous infusion of purified pooled human plasma alpha 1-antitrypsin deficiency to raise and maintain serum Alpha 1-antitrypsin levels above the threshold and to slow emphysema progression and enhance the duration and quality of life.
- Food and Drug Administration has approved four preparations of purified AAT.
- Following infusion AAT levels remain above the protective threshold for most of the dosing interval.
- The infused AAT has the ability to neutralize neutrophil elastase activity.
- Augmentation therapy recipients demonstrate a slower rate of FEV1 decline than nonrecipients
- Wencker and colleagues conducted a before–after study and found that the greatest effect of augmentation therapy in changing FEV1 slope was observed in individuals with a rapid FEV1 decline before augmentation therapy was initiated (ie, FEV1 decline 256 mL/y before therapy vs 53 mL/y during therapy).