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- α1-antitrypsin deficiency
- augmentation therapy
- plasma neutrophil derived fibrinogen fragments (PMN-FDP)
A recent review by Stoller and Aboussouan presented the current understanding of intravenous augmentation therapy for α1-antitrypsin (AAT) deficiency.1 Their criteria for demonstrating efficacy of this therapy did not include evidence of protection against lung tissue destruction. Such studies would show that sufficient levels of AAT are reached in the lungs to allow inhibition of neutrophil derived enzymes before they degrade elastin fibres to cause alveolar destruction—the hallmark of emphysema. To date, no such evidence has been presented. A study with the currently recommended augmentation regimen using assays of elastin degradation products failed to show any efficacy.2 The authors argued that the duration of treatment was probably too short, but one may also argue that the dose was too low.
Ever since the introduction of AAT augmentation therapy, no clinical benefit has been demonstrated in a randomised clinical trial. However, the direct health care costs associated with AAT deficiency are high.1 As no effect of this treatment on lung function has …