The direct medical costs of alpha(1)-antitrypsin deficiency.
BACKGROUND
For individuals with emphysema because of severe alpha(1)-antitrypsin deficiency, specific therapy called IV augmentation therapy has been available since 1989. Such therapy consists of IV infusion of pooled human 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 their annual expenditures for treatment of this disease. Data regarding demographic features, alpha(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*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 alpha(1)-antiprotease use status was reported. For the 185 current alpha(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 receiving augmentation therapy. Augmentation therapy adds substantial costs, especially for heavier individuals who are receiving weekly infusions.
University of Maryland School of Pharmacy, Baltimore, MD 21201, USA. dmullins@rx.umaryland.edu
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Mullins
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Huang
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Merchant
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Stoller
D004180:Direct Service Costs / Q000706:statistics & numerical data*; D005260:Female; D017048:Health Care Costs / Q000706:statistics & numerical data; D005102:Health Expenditures / Q000706:statistics & numerical data*; D006801:Humans; D007262:Infusions, Intravenous; D008297:Male; D008875:Middle Aged; D010641:Phenotype; D011656:Pulmonary Emphysema / Q000188:drug therapy* / Q000191:economics; D012042:Registries / Q000706:statistics & numerical data; D011795:Surveys and Questionnaires; D000515:alpha 1-Antitrypsin / Q000191:economics* / Q000627:therapeutic use*; D019896:alpha 1-Antitrypsin Deficiency / Q000188:drug therapy* / Q000191:economics*
D000515:alpha 1-Antitrypsin