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.
Mullins
C D
CD
University of Maryland School of Pharmacy, Baltimore, MD 21201, USA. dmullins@rx.umaryland.edu
Huang
X
X
Merchant
S
S
Stoller
J K
JK
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