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原发性免疫缺陷病:比较澳大利亚静脉注射与皮下免疫球蛋白替代疗法的成本-效用分析。

Primary immunodeficiency disease: a cost-utility analysis comparing intravenous vs subcutaneous immunoglobulin replacement therapy in Australia.

机构信息

School of Health and Sport Sciences, University of the Sunshine Coast, Sippy Down, QLD, Australia.

Menzies Health Institute Queensland, Griffith University, Brisbane, QLD, Australia.

出版信息

Blood Transfus. 2020 Mar;18(2):96-105. doi: 10.2450/2019.0083-19. Epub 2019 Aug 5.

Abstract

BACKGROUND

Hospital-based intravenous immunoglobulin (IVIg) treatment has been the standard treatment mode for patients with primary immunodeficiency disease (PID). With the newer home-based subcutaneous immunoglobulin (SCIg) becoming approved for use in most countries, the question arises as to whether SCIg is a cost-effective treatment mode compared to IVIg in Australia.

MATERIALS AND METHODS

We developed a Markov cohort simulation model with six health states: PID without infection, PID with infection treated at home or hospital, bronchiectasis without infection, bronchiectasis with infection treated at home or hospital, bronchiectasis with chronic Pseudomonas aeruginosa infection, and death, from an Australian healthcare system perspective. A 10-year time horizon with weekly cycles was chosen, and the expected costs and quality-adjusted life-years (QALYs) of the two treatment options estimated.

RESULTS

The cumulative 10-year cost per patient was 297,547 Australian dollars (A$) with IVIg and A$ 251,713 for SCIg. IVIg resulted in 5.55 QALYs and SCIg 5.57 QALYs. Thus, SCIg appears to be a cost-saving option and possibly improves QALY from the Australian healthcare system perspective (i.e., the dominant treatment option). A probabilistic sensitivity analysis showed that the SCIg option is preferred in 93.2% of simulations given willingness to pay of A$ 50,000 per QALY gained.

DISCUSSION

The results suggest that home-based SCIg is a cost-effective treatment option for patients with PID in Queensland, Australia.

摘要

背景

基于医院的静脉注射免疫球蛋白(IVIg)治疗一直是原发性免疫缺陷病(PID)患者的标准治疗模式。随着新型的皮下免疫球蛋白(SCIg)在大多数国家获得批准使用,人们开始质疑 SCIg 是否比澳大利亚的 IVIg 更具成本效益。

材料和方法

我们从澳大利亚医疗保健系统的角度出发,开发了一个具有六个健康状态的马尔可夫队列模拟模型:无感染的 PID、在家或医院治疗的感染性 PID、无感染的支气管扩张症、在家或医院治疗的感染性支气管扩张症、患有慢性铜绿假单胞菌感染的支气管扩张症和死亡。选择了 10 年的时间范围和每周循环,估计了两种治疗方案的预期成本和质量调整生命年(QALYs)。

结果

使用 IVIg 的患者 10 年内的累计成本为 297,547 澳元(A$),使用 SCIg 的成本为 251,713 A$。IVIg 产生 5.55 个 QALY,SCIg 产生 5.57 个 QALY。因此,从澳大利亚医疗保健系统的角度来看,SCIg 似乎是一种节省成本的选择,并且可能提高了 QALY(即,作为主要治疗方案)。概率敏感性分析表明,在支付意愿为 50,000 澳元/QALY 的情况下,93.2%的模拟中,SCIg 方案更受青睐。

讨论

结果表明,对于澳大利亚昆士兰州的 PID 患者,家庭为基础的 SCIg 是一种具有成本效益的治疗选择。

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