Augmentation therapy of alpha-1 antitrypsin deficiency associated emphysema

Abstract

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(®). 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.

Keywords: Administration and posology; Administration et posologie; Alpha-1 antitrypsin; Alpha-1 antitrypsin deficiency; Alpha-1 antitrypsine; Blood proteins; Déficit en alpha-1 antitrypsine; Emphysema; Emphysème; Protéines du sang.