Cost-effectiveness of 4 neonatal screening strategies for cystic fibrosis
Cost-effectiveness of 4 neonatal screening strategies for cystic fibrosis
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DOI：
10.1542/peds.2005-2782
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发表时间：
2006-09-01
期刊：
影响因子：
8
通讯作者：
Dankert-Roelse, Jeannette E.
中科院分区：
文献类型：
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作者：
Marle, M. Elske van den Akker-van;Dankert, Hinke M.;Dankert-Roelse, Jeannette E.
目标.这项工作的目的是评估4新生儿囊性纤维化筛查策略的成本与健康影响的关系。在每种策略中，第一项试验是测量免疫反应性胰蛋白酶的血清浓度。第二步包括第二次免疫反应性胰蛋白酶试验（策略1）或多重突变分析（策略2）。在策略3和4中，在策略2的基础上增加了第三步：第二次免疫反应性胰蛋白酶试验（策略3）或囊性纤维化基因的扩展突变分析，即变性梯度凝胶电泳分析（策略4）。我们根据公布的数据和专家意见在荷兰进行了一次经济建模。受试者是一个假设的20万新生儿队列，大约每年在荷兰出生的儿童人数，我们评估了成本和获得的生命年数作为新生儿囊性纤维化筛查的结果。由于新生儿筛查而改变生育决定的成本和影响也进行了评估。免疫反应性胰蛋白酶+免疫反应性胰蛋白酶具有最有利的成本-效果比，每生命年获得24800欧元。免疫反应性胰蛋白酶+ DNA +变性梯度凝胶电泳比免疫反应性胰蛋白酶+ DNA +免疫反应性胰蛋白酶具有更好的保健效果，且成本较低。免疫反应性胰蛋白酶+ DNA +变性梯度凝胶电泳策略与免疫反应性胰蛋白酶+免疫反应性胰蛋白酶策略相比，每生命年获得的增量成本为130 700欧元，而免疫反应性胰蛋白酶+ DNA策略与免疫反应性胰蛋白酶+ DNA +变性梯度凝胶电泳策略相比，每生命年获得的增量成本为2 154 300欧元。当考虑到新生儿筛查导致的生育决定的变化时，新生儿囊性纤维化筛查每年可节省约180万欧元，具体取决于所使用的筛查策略。新生儿囊性纤维化筛查是一个很好的经济选择，也可以预期积极的健康影响。免疫反应性胰蛋白酶+免疫反应性胰蛋白酶和免疫反应性胰蛋白酶+ DNA +变性梯度凝胶电泳是最具成本效益的策略。
OBJECTIVES. The purpose of this work was to assess the costs of 4 neonatal screening strategies for cystic fibrosis in relation to health effects. In each strategy, the first test was the measurement of serum concentration of immunoreactive trypsin. The second step consisted of either a second immunoreactive trypsin test (strategy 1) or a multiple mutation analysis (strategy 2). In strategies 3 and 4, a third step was added to strategy 2: a second immunoreactive trypsin test (strategy 3) or an extended mutation analysis of the cystic fibrosis gene, that is, a denaturing gradient gel electrophoresis analysis (strategy 4).METHODS. We conducted an economic-modeling exercise in the Netherlands based on published data and expert opinions. Subjects were a hypothetical cohort of 200 000 neonates, the approximate number of children born annually in the Netherlands, and we assessed the costs and number of life-years gained as a result of neonatal screening for cystic fibrosis. The costs and effects of changes in reproductive decisions because of neonatal screening were also assessed.RESULTS. Immunoreactive trypsin + immunoreactive trypsin had the most favorable cost-effectiveness ratio of 24 800 euro per life-year gained. Immunoreactive trypsin + DNA + denaturing gradient gel electrophoresis achieved more health effects than immunoreactive trypsin + DNA + immunoreactive trypsin at lower cost. The incremental costs per life-year gained of the immunoreactive trypsin + DNA + denaturing gradient gel electrophoresis strategy compared with the immunoreactive trypsin + immunoreactive trypsin strategy were 130 700, whereas the incremental costs of the immunoreactive trypsin + DNA strategy compared with the immunoreactive trypsin + DNA + denaturing gradient gel electrophoresis strategy were 2 154 300 euro. When changes in reproductive decisions as a result of neonatal screening are also taken into account, neonatal screening for cystic fibrosis may lead to financial savings of approximately 1.8 million euro annually, depending on the screening strategy used.CONCLUSIONS. Cystic fibrosis screening for neonates is a good economic option, and positive health effects can also be expected. Immunoreactive trypsin + immunoreactive trypsin and immunoreactive trypsin + DNA + denaturing gradient gel electrophoresis are the most cost-effective strategies.