Background: For individuals with emphysema because of
severe α1-antitrypsin deficiency, specific therapy called
IV augmentation therapy has been available since 1989. Such therapy
consists of IV infusion of pooled human plasmaα
Methods: To assess the
direct medical costs of having α1-antitrypsin deficiency,
the current study surveyed members of the Alpha One Foundation Registry
for Individuals With α1-Antitrypsin Deficiency regarding
their annual expenditures for treatment of this disease. Data regarding
demographic features, α1-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*ZZ-phenotype individuals ($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
whose α1-antiprotease use status was reported. For the
185 current α1-antiprotease users, the mean annual
cost was $40,123 (median, $36,000).
Annual health-care expenditures by individuals withα
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.