Augmentation therapy of alpha-1 antitrypsin deficiency associated emphysema

Introduction. - Alpha-1 antitrypsin, secreted by the liver, inhibits neutrophil elastase. Its deficiency favours the development of emphysema. Restoring a "protective" serum level in deficient patients should make it possible to inhibit the development of emphysema. State of the art. - Human plasma-derived alpha-1 antitrypsin is a blood-derived drug sold in France under the name Alfalastin (R). The recommended posology is an I.V. administration of 60 mg/kg once a week. Human plasma-derived alpha-1 antitrypsin restores anti-elastase protection in the lower lung and prevents experimental emphysema induced by the elastasis of human neutrophils in hamster. The low number of patients with alpha-1 antitrypsin deficiency is one of the difficulties to perform sufficiently powerful randomised studies. However, randomised studies have reported the efficacy of human plasma-derived alpha-1 antitrypsin perfusions on mortality, FEV1 decline and the frequency of exacerbations. Randomised control trials have demonstrated the efficacy of human plasma-derived alpha-1 antitrypsin perfusions on the loss of lung density assessed by CT scan. Conclusion. - Augmentation therapy is simple in its conception and implementation, but it is expensive. However, there are currently no other solutions. (C) 2014 SPLF. Published by Elsevier Masson SAS. All rights reserved.