Mullins C Daniel, Wang Junling, Stoller James K
University of Maryland School of Pharmacy, Center on Drugs and Public Policy, 2nd Floor, 515 W. Lombard Street, Baltimore, MD 21201, USA.
Chest. 2003 Sep;124(3):826-31. doi: 10.1378/chest.124.3.826.
To examine the sources of the direct medical costs of alpha(1)-antitrypsin (AAT) deficiency based on survey data from affected individuals.
Prior research has reported the total cost of AAT deficiency but has not examined the specific components of the direct medical costs of affected individuals.
In order to detail the sources of the direct medical costs, we sent surveys to 688 respondents of a previous survey. We estimated the costs in three ways, which differed in the method of managing missing values. With method 1, the group mean value of cost per unit of utilization, multiplied by the occurrences of utilization, was used to replace the missing value. Two sensitivity analyses (ie, methods 2 and 3) were conducted to test the robustness of our estimate. With method 2, values of zero were entered for all missing values. With method 3, the missing values were replaced by the group mean value. The Wilcoxon test was used to test the cost differences between patients of different phenotypes. All cost data were expressed in 1998 US dollars.
Two hundred ninety-two individuals responded to the survey. The annual total health-care costs were high (mean range, $36,471 to $46,114 US dollars; median range, $12,485 to $37,100 US dollars [according to the method for managing missing data]) for AAT deficiency. The total costs for individuals with the PIZZ phenotype exceeded those for individuals with a non-PIZZ phenotype. The use of IV augmentation therapy accounted for more than half of all direct medical costs for the respondents. Besides the costs for therapy with alpha(1)-proteinase inhibitor (Prolastin; Bayer; West Haven, CT), other major cost sources were prescription drugs other than alpha(1)-proteinase inhibitor, hospitalization, health insurance, and physician visits.
基于对受影响个体的调查数据，研究α1抗胰蛋白酶（AAT）缺乏症直接医疗费用的来源。
先前的研究报告了AAT缺乏症的总成本，但未研究受影响个体直接医疗费用的具体构成部分。
为详细说明直接医疗费用的来源，我们向先前一项调查的688名受访者发送了调查问卷。我们用三种方法估算费用，这三种方法在处理缺失值的方式上有所不同。方法1中，每单位使用量的成本组均值乘以使用次数，用于替代缺失值。进行了两项敏感性分析（即方法2和方法3）以检验我们估算的稳健性。方法2中，所有缺失值均输入零值。方法3中，缺失值用组均值替代。采用Wilcoxon检验来检验不同表型患者之间的费用差异。所有费用数据均以1998年美元表示。
292人回复了调查问卷。AAT缺乏症患者的年度总医疗费用较高（均值范围为36,471美元至46,114美元；中位数范围为12,485美元至37,100美元[根据处理缺失数据的方法]）。PIZZ表型个体的总成本超过非PIZZ表型个体。静脉补充疗法的使用占受访者所有直接医疗费用的一半以上。除了α1蛋白酶抑制剂（普洛斯汀；拜耳公司；康涅狄格州韦斯特黑文）治疗费用外，其他主要费用来源是α1蛋白酶抑制剂以外的处方药、住院、医疗保险和医生诊疗。
