Lieu T A, Ray G T, Farmer G, Shay G F
Department of Ambulatory Care and Prevention, Harvard Pilgrim Health Care and Harvard Medical School, 126 Brookline Ave, Suite 200, Boston, MA 02215, USA.
Pediatrics. 1999 Jun;103(6):e72. doi: 10.1542/peds.103.6.e72.
Cystic fibrosis (CF) is the most common life-shortening genetic disorder among white individuals worldwide. Previous estimates of the costs of medical care have been based on expert opinion rather than observed costs. Accurate cost estimates are needed to enable evaluation of the cost-effectiveness of new interventions and prenatal genetic screening recommendations.
To evaluate the cost of medical care for patients (N = 136) served by a health maintenance organization with a CF center.
Retrospective analysis of data from computerized cost databases and the Cystic Fibrosis Foundation annual survey. Severity of disease was classified based on the percent predicted forced expiratory volume at 1 second.
The annual cost of medical care in 1996 averaged $13 300 and ranged from $6200 among patients with mild disease to $43 300 among patients with severe disease. Of total costs, 47% were from hospitalization, 18% were from DNase (Pulmozyme), 12% were from clinic visits, and 10% were from outpatient antibiotics. When the observed costs were used to estimate the costs of medical care for the entire population of CF patients in the United States, these costs were estimated to be $314 million per year in 1996 dollars.
We conclude that the cost of medical care for CF varies greatly with severity but is substantial even among patients with mild disease. These findings underscore the need for strategies to ensure good health insurance coverage and high quality care for all individuals with this condition.
囊性纤维化（CF）是全球白人中最常见的缩短寿命的遗传性疾病。先前对医疗费用的估计是基于专家意见而非实际观察到的费用。需要准确的费用估计来评估新干预措施和产前基因筛查建议的成本效益。
评估由一家设有CF中心的健康维护组织服务的患者（N = 136）的医疗费用。
对来自计算机化成本数据库的数据和囊性纤维化基金会年度调查进行回顾性分析。根据预测的1秒用力呼气量百分比对疾病严重程度进行分类。
1996年的年度医疗费用平均为13300美元，轻度疾病患者的费用为6200美元，重度疾病患者的费用为43300美元。在总费用中，47%来自住院治疗，18%来自脱氧核糖核酸酶（普米克令舒），12%来自门诊就诊，10%来自门诊抗生素。当使用观察到的费用来估计美国所有CF患者的医疗费用时，这些费用在1996年估计为每年3.14亿美元（按1996年美元计算）。
我们得出结论，CF的医疗费用因严重程度而异，即使在轻度疾病患者中也相当可观。这些发现强调了需要采取策略，以确保所有患有这种疾病的人都能获得良好的医疗保险覆盖和高质量的护理。
