Cost-effectiveness analysis of universal screening for biliary atresia in Japan

Cost-effectiveness analysis of universal screening for biliary atresia in Japan
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日本胆道闭锁普遍筛查的成本效益分析

DOI:
10.1016/j.jpeds.2022.09.028
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发表时间:
2023
期刊:
J Pediatr
影响因子:
--
通讯作者:
Shimozuma K
Shimozuma K
中科院分区:
--
文献类型:
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作者:
Hoshino E; Moriwaki K; Morimoto K; Sakai K; Shimohata N; Konomura K;Urayama KY; Suzuki M; Shimozuma K

文献摘要

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目的比较日本采用大便颜色卡或直接胆红素(DB)筛查新生儿与不筛查新生儿胆道闭锁的成本-效果。研究设计:建立决策分析马尔可夫微观模拟模型,评估胆道闭锁的普遍筛查。我们的筛查策略包括大便颜色卡、DB或不筛查。模拟所有接受3种策略的新生儿的结局,分析无事件生命年(无肝移植生存)、成本和25年期间的增量成本-效果比(ICER),成本和结果的年贴现率均为2%。采用单向敏感性分析来评估不确定性。结果本组新生儿94.1万例,胆道闭锁114例。基本案例分析显示,大便颜色卡策略比不筛查高出14 927 337美元,增加了44个无事件生命年,导致每获得一个无事件生命年的ICER为339 258美元。与粪便颜色卡相比,DB筛查策略增加了138 994 060美元,增加了271个无事件生命年,每个无事件生命年的ICER为512 893美元。与没有筛查相比,DB筛查策略导致每个无事件生命年的ICER为488 639美元。DB筛查比大便颜色卡少16例肝移植,大便颜色卡比未筛查少2例肝移植。结论:胆道闭锁的普遍筛查可能具有成本效益,这取决于支付健康福利阈值的意愿。
Objective To evaluate the cost-effectiveness of universal newborn screening using stool color card or direct bilirubin (DB) testing when comparing with no screening for biliary atresia in Japanese setting. Study design A decision analytic Markov microsimulation model was developed to evaluate the universal screening for biliary atresia. Our screening strategies included stool color card, DB, or no screening. The outcomes of all newborns undergoing 3 strategies were simulated to analyze event-free life-years defined as liver transplant-free survival, costs, and incremental cost-effectiveness ratio (ICER) over a 25-year period with an annual discount rate of 2% applied for both costs and outcomes. A 1-way sensitivity analysis was performed to assess the uncertainty. Results There were 941 000 newborn infants in our cohort and 114 cases of biliary atresia. The base case analysis showed that the stool color card strategy was $14 927 337 higher than no screening with an increase in 44 more event-free life-years gained, resulting in an ICER of $339 258 per event-free life-year gained. The DB screening strategy compared with stool color card was $138 994 060 higher with an increase in 271 more event-free life-years gained and an ICER of $512 893 per event-free life-year gained. The DB screening strategy compared with no screening resulted in an ICER of $488 639 per event-free life-year gained. The DB screening resulted in 16 fewer liver transplants than stool color card and stool color card had 2 fewer liver transplants than no screening. Conclusions Universal screening for biliary atresia could be cost-effective depending on the willingness to pay thresholds for health benefits.