Abstract
Alpha-1 antitrypsin deficiency is a genetic disease that first highlighted the importance of protease balance in normal lung homeostasis. Proteases such as neutrophil elastase are important in many pulmonary diseases. However, the first commercially licensed anti-protease therapy was used for emphysema in alpha-1 antitrypsin deficiency. The 4 commercial alpha-1 protease inhibitor preparations in the United States are currently given intravenously to augment the serum and alveolar epithelial lining fluid concentrations of alpha-1 antitrypsin. Since the discovery of alpha-1 antitrypsin deficiency in 1963, many genetic polymorphisms of the SERPINA1 gene have been discovered. Because neutrophilic inflammation is the hallmark of usual COPD, anti-protease therapy also has been explored in this disease. Further trials with different dosing schema, inhaled therapy, and in different disease states are all ongoing. These studies would be facilitated by having COPD outcomes that are independent of measurement of air flow and achievable in smaller populations over a short period of time.
- antitrypsin
- alpha-1
- emphysema
- protease
- anti-protease
- alpha-1 protease inhibitor
- A1PI
- alpha-1 antitrypsin deficiency
Footnotes
- Correspondence: Charlie Strange MD, Division of Pulmonary, Critical Care, Allergy, and Sleep Medicine, Department of Medicine, Medical University of South Carolina, MSC 630, 816 CSB, Charleston, SC 29425. E-mail: strangec{at}musc.edu.
Dr Strange has disclosed relationships with Adverum, the Alpha-1 Foundation, Baxalta/Shire, CSL Behring, Grifols, MatRx, the National Institutes of Health, Abeona, AstraZeneca, Bronchus, BTG, CSA Medical, Pulmonx, and Uptake Medical.
Dr Strange presented a version of this paper at the 56th Respiratory Care Journal Conference, Respiratory Medications for COPD and Adult Asthma: Pharmacologic Actions to Clinical Applications, held June 22-23, 2017 in St Petersburg, Florida.
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