The direct medical costs of alpha-antitrypsin deficiency

Background: For individuals with emphysema because of severe alpha (1)-anti trypsin deficiency, specific therapy called IV augmentation therapy has bee n available since 1989. Such therapy consists of IV infusion of pooled huma n plasma alpha (1)-antiprotease. Methods: To assess the direct medical costs of having alpha (1)-antitrypsin deficiency, the current study surveyed members of the Alpha One Foundation Registry for Individuals With alpha (1)-Antitrypsin Deficiency regarding t heir annual expenditures for treatment of this disease. Data regarding demo graphic Features, alpha (1)-antitrypsin status, and health-resource utiliza tion were collected from a self-administered questionnaire. Respondents wer e asked to provide total health-care expenditures, but costs by specific it ems of care (eg, drugs, physician visits, etc) were not available. Results: Mean annual cost estimates were higher for PI*ZZ-phenotype individ uals ($30,948, n = 292) than for non-PI*ZZ-phenotype individuals ($20,673, n = 53; p = 0.049). Among PI*ZZ-phenotype individuals, self-reported costs of health-care services were further analyzed for those 288 individuals who se alpha (1)-antiprotease use status was reported. For the 185 current alph a (1)-antiprotease users, the mean annual cost was $40,123 (median, $36,000 ). Conclusions: Annual health-care expenditures by individuals with alpha (1)- antitrypsin deficiency are very high, whether or not they are currently rec eiving augmentation therapy. Augmentation therapy adds substantial costs, e specially For heavier individuals who are receiving weekly infusions.