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1-Antitrypsin Deficiency*
*
From the University of Maryland School of Pharmacy (Dr. Mullins, Mr. Huang, and Mr. Merchant), Baltimore, MD; and Department of Pulmonary and Critical Care Medicine (Dr. Stoller), The Cleveland Clinic Foundation, Cleveland, OH.
A complete list of investigators is presented in the Appendix.
Correspondence to: C. Daniel Mullins, PhD, University of Maryland School of Pharmacy, 6th Floor, 100 N. Greene St, Baltimore, MD 21201; e-mail: dmullins{at}rx.umaryland.edu
Background: For individuals with emphysema because of
severe
1-antitrypsin deficiency, specific therapy called
IV augmentation therapy has been available since 1989. Such therapy
consists of IV infusion of pooled human plasma
1-antiprotease.
Methods: To assess the
direct medical costs of having
1-antitrypsin deficiency,
the current study surveyed members of the Alpha One Foundation Registry
for Individuals With
1-Antitrypsin Deficiency regarding
their annual expenditures for treatment of this disease. Data regarding
demographic features,
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*ZZphenotype 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
1-antiprotease use status was reported. For the
185 current
1-antiprotease users, the mean annual
cost was $40,123 (median, $36,000).
Conclusions:
Annual health-care expenditures by individuals with
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.
Key Words:
1-antitrypsin deficiency cost registry
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