Tsevat J, Wong J B, Pauker S G, Steinberg M H
Department of Medicine, New England Medical Center, Boston, MA 02111.
J Pediatr. 1991 Apr;118(4 Pt 1):546-54. doi: 10.1016/s0022-3476(05)83375-x.
To determine the cost-effectiveness of screening newborn infants for sickle cell disease.
We developed a decision model that examined two strategies: (1) screening neonates and administering penicillin to infants found to have sickle cell disease in the hope of preventing pneumococcal sepsis, and (2) not screening but administering penicillin to infants after symptoms of sickle cell disease develop. The model calculates the cost-effectiveness of these strategies during the first 3 years of life. We applied the model to three prototypic populations of neonates--black, nonblack with a relatively high prevalence of hemoglobin S genes, and nonblack with a low prevalence of hemoglobin S genes.
We obtained from the published literature the effectiveness and risk of penicillin prophylaxis, the risk of pneumococcal sepsis, and the probability that in infants not screened the development of symptoms would lead to the discovery of sickle cell disease within the first 3 years of life; we used the published literature and the Hardy-Weinberg law to determine the prevalence of sickle cell disease. We used actual variable costs of screening, antibiotic prophylaxis, and hospitalization for pneumococcal sepsis or anaphylaxis.
Screening and then treating affected black infants costs only $3100 more per life saved than not screening. Screening nonblack populations with a high prevalence of hemoglobin S genes would cost $1.4 million per life saved, and screening low prevalence populations would cost $450 billion per life saved.
Screening black infants is very worthwhile, but screening populations in which the hemoglobin S gene is rare is unjustified.
确定对新生儿进行镰状细胞病筛查的成本效益。
我们开发了一个决策模型，该模型考察了两种策略：（1）对新生儿进行筛查，并对被发现患有镰状细胞病的婴儿给予青霉素治疗，以期预防肺炎球菌败血症；（2）不进行筛查，而是在镰状细胞病症状出现后对婴儿给予青霉素治疗。该模型计算了这些策略在生命最初3年期间的成本效益。我们将该模型应用于三个典型的新生儿群体——黑人、血红蛋白S基因患病率相对较高的非黑人以及血红蛋白S基因患病率较低的非黑人。
我们从已发表的文献中获取了青霉素预防的有效性和风险、肺炎球菌败血症的风险，以及未筛查婴儿中症状出现导致在生命最初3年内发现镰状细胞病的概率；我们使用已发表的文献和哈迪 - 温伯格定律来确定镰状细胞病的患病率。我们使用了筛查、抗生素预防以及肺炎球菌败血症或过敏反应住院治疗的实际可变成本。
对受影响的黑人婴儿进行筛查然后治疗，每挽救一条生命的成本仅比不筛查高3100美元。对血红蛋白S基因患病率高的非黑人人群进行筛查，每挽救一条生命的成本为140万美元，而对患病率低的人群进行筛查，每挽救一条生命的成本为4500亿美元。
对黑人婴儿进行筛查非常值得，但对血红蛋白S基因罕见的人群进行筛查是不合理的。
