Alpha 1-antitrypsin. Hope on the horizon for emphysema sufferers?

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.