Comprehensive cost-utility analysis of newborn screening strategies
Comprehensive cost-utility analysis of newborn screening strategies
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DOI：
10.1542/peds.2005-2633h
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发表时间：
2006-05-01
期刊：
影响因子：
8
通讯作者：
Downs, SM
中科院分区：
文献类型：
--
作者：
Carroll, AE;Downs, SM
背景先天性代谢缺陷是儿童发病和死亡的一个重要原因。然而，各州如何制定筛查策略的不确定性导致了各州之间的测试差异。确定多重检测新生儿筛查项目中每种成分检测的成本-效果，包括苯丙酮尿症、先天性肾上腺皮质增生、先天性甲状腺功能减退、生物素酶缺乏、枫糖浆尿病、半乳糖血症、同型胱氨酸尿症和中链酰基辅酶A脱氢酶缺乏。使用决策模型，包括队列研究、政府报告、二次分析和其他来源。贴现成本，质量调整生命年（QHMS），和增量成本效益比进行了测量。除2项筛选试验外，其余均以“不试验”策略为主。2个例外是先天性肾上腺增生筛查，每获得一个QALY的费用略高于20 000美元，半乳糖血症筛查，每获得一个QALY的费用为94 000美元。预期成本最低的筛选试验是串联质谱法。在我们的基础案例分析中发现的结果在几乎所有变量的变化中都是稳定的。在风险、后遗症、成本或效用的变化确实影响我们的结果的情况下，与基本情况估计值的差异相当大。新生儿筛查似乎是对患者有益的罕见医疗保健干预措施之一，在许多情况下，可以节省成本。从长远来看，资助全面的新生儿筛查项目可能会为社会节省资金。
BACKGROUND. Inborn errors of metabolism are a significant cause of morbidity and death among children. Inconsistencies in how individual states arrive at screening strategies, however, lead to marked variations in testing between states.OBJECTIVE. To determine the cost-effectiveness of each component test of a multitest newborn screening program, including screening for phenylketonuria, congenital adrenal hyperplasia, congenital hypothyroidism, biotinidase deficiency, maple syrup urine disease, galactosemia, homocystinuria, and medium-chain acyl-CoA dehydrogenase deficiency.METHODS. A decision model was used, with cohort studies, government reports, secondary analyses, and other sources. Discounted costs, quality-adjusted life-years ( QALYs), and incremental cost-effectiveness ratios were measured.RESULTS. All except 2 screening tests dominated the "no-test" strategy. The 2 exceptions were screening for congenital adrenal hyperplasia, which cost slightly more than $20 000 per QALY gained, and screening for galactosemia, which cost $94 000 per QALY gained. The screening test with the lowest expected cost was tandem mass spectrometry. The results found in our base-case analysis were stable across variations in nearly all variables. In instances in which changes in risks, sequelae, costs, or utilities did affect our results, the variation from base-case estimates was quite large.CONCLUSIONS. Newborn screening seems to be one of the rare health care interventions that is beneficial to patients and, in many cases, cost saving. Over the long term, funding comprehensive newborn screening programs is likely to save money for society.