title: Augmentation therapy of alpha-1 antitrypsin deficiency associated emphysema.
abstract: BACKGROUND
  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.
  
  
  BACKGROUND
  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.
  
  
  CONCLUSIONS
  Augmentation therapy is simple in its conception and implementation, but it is expensive. However, there are currently no other solutions.
authors.affiliation: Hospices civils de Lyon, 69000 Lyon, France; Centre de référence des maladies rares pulmonaires, 69000 Lyon, France.
authors.forename: J
authors.initials: J
authors.lastname: Traclet
authors.affiliation: IRSET UMR Inserm U1085, université de Rennes 1, 35000 Rennes, France; Centre hospitalier universitaire de Rennes, 35000 Rennes, France.
authors.forename: P
authors.initials: P
authors.lastname: Delaval
authors.affiliation: Cabinet de pneumologie, 77100 Meaux, France.
authors.forename: P
authors.initials: P
authors.lastname: Terrioux
authors.affiliation: Hospices civils de Lyon, 69000 Lyon, France; Centre de référence des maladies rares pulmonaires, 69000 Lyon, France; Université Lyon 1, 69000 Lyon, France; INRA, UMR754, 69000 Lyon, France. Electronic address: mornex@univ-lyon1.fr.
authors.forename: J-F
authors.initials: JF
authors.lastname: Mornex
mesh_terms: D000818:Animals; D004357:Drug Synergism; D006801:Humans; D008168:Lung / Q000187:drug effects / Q000473:pathology; D011656:Pulmonary Emphysema / Q000188:drug therapy*; D000515:alpha 1-Antitrypsin / Q000627:therapeutic use*; D019896:alpha 1-Antitrypsin Deficiency / Q000188:drug therapy*
chemical_list: D000515:alpha 1-Antitrypsin
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