de Vos Thijs W, Tersteeg Ilonka, Lopriore Enrico, Oepkes Dick, Porcelijn Leendert, van der Schoot C Ellen, Verweij E Joanne T, Winkelhorst Dian, de Haas Masja, van den Akker-van Marle M Elske
Willem-Alexander Children's Hospital, Department of Pediatrics, Division of Neonatology, Leiden University Medical Center, Leiden, the Netherlands.
Department of Experimental Immunohematology, Sanquin Research, Amsterdam, the Netherlands.
Vox Sang. 2025 Feb;120(2):178-187. doi: 10.1111/vox.13779. Epub 2024 Dec 5.
Foetal and neonatal alloimmune thrombocytopenia (FNAIT) results from maternal platelet-directed antibodies and can result in severe intracranial haemorrhage (ICH) in foetuses and newborns. Screening for human platelet antigen-1a (HPA-1a)-directed antibodies during pregnancy could allow timely intervention with antenatal treatment and prevent ICH. We assessed the cost effectiveness of HPA-1a typing and anti-HPA-1a-screening as part of the prenatal screening programme.
Different HPA-1a screening scenarios were tested in a decision analysis model and assessed for diagnostic, treatment, intervention and lifetime costs and prevention effects compared to the current situation without screening in the Netherlands. Model parameters were based on available data, literature and expert opinions. One-way sensitivity analysis and probabilistic sensitivity analysis were performed.
Adding screening for anti-HPA-1a antibodies to the current antenatal screening programme of the Netherlands will lead to an additional cost of €4.7 million per year and a gain of 226 quality-adjusted life years (QALYs) per year, indicating an incremental cost-effectiveness ratio (ICER) of €20,782 per QALY gained. One-way sensitivity analysis showed that the uncertainty around the incidence of ICH, lifetime costs of disabled children and the probability of having antibody quantitation >3.0 IU/mL at 20 weeks had the highest effect on the ICER.
Antenatal anti-HPA-1a screening might be cost effective. To obtain more knowledge and thereby to improve risk stratification, a pilot screening programme is warranted.
胎儿及新生儿同种免疫性血小板减少症（FNAIT）由母体血小板定向抗体引起，可导致胎儿和新生儿严重颅内出血（ICH）。孕期筛查人类血小板抗原-1a（HPA-1a）定向抗体可实现产前治疗的及时干预并预防ICH。我们评估了HPA-1a分型及抗HPA-1a筛查作为产前筛查项目一部分的成本效益。
在决策分析模型中测试了不同的HPA-1a筛查方案，并与荷兰目前不进行筛查的情况相比，评估其诊断、治疗、干预和终生成本以及预防效果。模型参数基于现有数据、文献和专家意见。进行了单向敏感性分析和概率敏感性分析。
在荷兰目前的产前筛查项目中增加抗HPA-1a抗体筛查，每年将导致额外成本470万欧元，每年可增加226个质量调整生命年（QALY），表明每获得一个QALY的增量成本效益比（ICER）为20,782欧元。单向敏感性分析表明，ICH发病率、残疾儿童终生成本以及孕20周时抗体定量>3.0 IU/mL概率的不确定性对ICER影响最大。
产前抗HPA-1a筛查可能具有成本效益。为获取更多知识从而改善风险分层，有必要开展一项试点筛查项目。
