To review the epidemiology, pathogenesis, and management of patients with α-antitrypsin (AAT) deficiency syndrome and compare Aralast with Prolastin, 2 of the 3 available human plasma–derived AAT agents.
DATA SOURCES
Articles were identified using a MEDLINE (1966–September 2005) search with MESH headings that included α-antitrypsin and emphysema.
STUDY SELECTION AND DATA EXTRACTION
All papers from peer-reviewed journals on the laboratory or clinical efficacy of plasma-derived AAT (eg, Prolastin, Aralast) for patients with this autosomal recessive disorder were reviewed.
DATA SYNTHESIS
Clinical trials found that AAT augmentation prevents progression of AAT-deficient emphysema and thus its associated morbidity and mortality. Treatment with Aralast has been shown to be safe and well tolerated, with a low incidence of mild to moderate adverse events. Pharmacoeconomics studies of AAT augmentation demonstrated that the use of Aralast was cost-effective as lifelong augmentation therapy for AAT-deficient emphysema.
CONCLUSIONS
Because of its effectiveness and extra safety measure compared with Prolastin, Aralast should be recommended for formulary inclusion.
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