The Direct Medical Costs of α[sub1]-Antitrypsin Deficiency.

Background: For individuals with emphysema because of severe α[sub 1]-antitrypsin deficiency, specific therapy called IV augmentation therapy has been available since 1989. Such therapy consists of IV infusion of pooled human plasma α[sub 1]-antiprotease. Methods: To assess the direct medical costs of having α[sub 1]-antitrypsin deficiency, the current study surveyed members of the Alpha One Foundation Registry for Individuals With α[sub 1]-Antitrypsin Deficiency regarding their annual expenditures for treatment of this disease. Data regarding demographic features, α[sub 1]-antitrypsin status, and health-resource utilization were collected from a self-administered questionnaire. Respondents were asked to provide total health-care expenditures, but costs by specific items of care (eg, drugs, physician visits, etc) were not available. Results: Mean annual cost estimates were higher for PI[sup *]ZZ-phenotype individuals ($30,948, n = 292) than for non-PI[sup *]ZZ-phenotype individuals ($20,673, n = 53; p = 0.049). Among PI[sup *]ZZphenotype individuals, self-reported costs of health-care services were further analyzed for those 288 individuals whose α[sub 1]-antiprotease use status was reported. For the 185 current α[sub 1]antiprotease users, the mean annual cost was $40,123 (median, $36,000). Conclusions: Annual health-care expenditures by individuals with α[sub 1]-antitrypsin deficiency are very high, whether or not they are currently receiving augmentation therapy. Augmentation therapy adds substantial costs, especially for heavier individuals who are receiving weekly infusions. [ABSTRACT FROM AUTHOR]