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Alpha 1-antitrypsin. Hope on the horizon for emphysema sufferers?
1Department of Internal Medicine, Klinikum Grosshadern, University of Munich, Germany.
Insights
Alpha 1-Antitrypsin (alpha 1AT) deficiency is a genetic disorder causing liver disease and emphysema. Augmentation therapy using alpha 1AT can slow emphysema progression with few adverse events.
Area of Science:
- Pulmonology
- Genetics
- Hepatology
Background:
- Alpha 1-Antitrypsin (alpha 1AT) deficiency is the leading genetic cause of liver disease in children and emphysema in adults.
- Current therapy for pulmonary disease involves alpha 1AT augmentation and supportive care.
Purpose of the Study:
- To review the efficacy and safety of alpha 1AT augmentation therapy for emphysema.
- To identify patient populations who may benefit from augmentation therapy.
- To explore novel therapeutic approaches for alpha 1AT deficiency.
Main Methods:
- Review of clinical trial data on alpha 1AT augmentation therapy.
- Analysis of patient criteria for initiating therapy.
- Summary of emerging treatment strategies.
Main Results:
- Clinical trials indicate that alpha 1AT augmentation therapy slows emphysema progression.
- The therapy is associated with a low incidence of adverse events.
- Patients with plasma alpha 1AT levels < 11 mumol/L and airway obstruction are candidates for therapy.
Conclusions:
- Alpha 1AT augmentation therapy is a viable treatment for emphysema in patients with alpha 1AT deficiency.
- Novel therapies including aerosolized alpha 1AT, recombinant alpha 1AT, gene therapy, and synthetic elastase inhibitors are under investigation.
Abstract:
Alpha 1-Antitrypsin (alpha 1AT) deficiency is the most common genetic cause of liver disease in children and emphysema in adults. Therapy for pulmonary disease attributable to alpha 1AT deficiency includes alpha 1AT augmentation therapy along with supportive measures. The alpha 1AT preparation that is currently used for therapy is derived from fractionated plasma. The results of clinical trials suggest that augmentation therapy with alpha 1AT slows the progression of emphysema and causes few adverse events. Patients with plasma levels of alpha 1AT that are < 11 mumol/L and who have airway obstruction should be considered for augmentation therapy. Novel approaches include the administration of aerosolised alpha 1AT, recombinant alpha 1AT, gene therapy and synthetic elastase inhibitors.