Objective: To review the epidemiology, pathogenesis, and management of patients with alpha-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 alpha-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.