Related Experiment Videos
Alpha-1-antitrypsin augmentation therapy for alpha-1-antitrypsin deficiency
1Pulmonary Branch, National Heart, Lung and Blood Institute, Bethesda, Maryland 20892.
The American Journal of Medicine
|June 24, 1988
Summary
Alpha-1-antitrypsin (A1AT) deficiency treatment involves weekly infusions of A1AT to boost lung protection against emphysema. This safe and effective therapy normalizes anti-neutrophil elastase capacity, preventing further lung damage.
Area of Science:
- Pulmonology
- Genetics
- Pharmacology
Background:
- Alpha-1-antitrypsin (A1AT) deficiency is a genetic disorder linked to emphysema risk due to insufficient A1AT.
- A1AT inhibits neutrophil elastase, protecting the lungs from degradation.
Purpose of the Study:
- To evaluate the efficacy and safety of A1AT augmentation therapy for A1AT deficiency.
- To determine if intravenous A1AT infusions can restore protective A1AT levels in the lungs.
Main Methods:
- Intravenous administration of human A1AT (60 mg/kg weekly).
- Monitoring of serum and epithelial lining fluid (ELF) A1AT levels.
- Assessing ELF anti-neutrophil elastase capacity.
Main Results:
- Serum A1AT levels increased from 33 mg/dl to a steady-state trough of 117 mg/dl.
- ELF A1AT levels significantly increased from 0.44 microM to 2.62 microM.
- A1AT infusions augmented ELF anti-neutrophil elastase capacity, normalizing lung protection.
Conclusions:
- Weekly A1AT infusions are a safe and biochemically effective therapy for A1AT deficiency.
- Augmentation therapy restores adequate anti-elastase protection in the lungs.
- Long-term treatment demonstrates no significant adverse reactions over 800 infusions.