Beijing Neurosurgical Institute, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Department of Neurosurgery, The Third Xiangya Hospital, Central South University, Changsha, China.
BMJ Open. 2022 Nov 14;12(11):e065133. doi: 10.1136/bmjopen-2022-065133.
The effectiveness of MRI-guided intravenous recombinant tissue-type plasminogen activator (r-tPA) for acute ischaemic stroke (AIS) with an unknown time of onset has been demonstrated by the WAKE-UP Trial. We aim to evaluate its long-term cost-effectiveness from the perspective of Chinese and US healthcare payers.
A combination of decision tree and Markov model was built to project lifetime costs and quality-adjusted life-years (QALYs) associated with intravenous r-tPA or placebo treatment. Model inputs including the transition probabilities, costs and utilities were derived from the WAKE-UP Trial, similar cost-effectiveness studies and other published sources. To compare intravenous r-tPA to placebo, we calculated incremental costs, incremental QALYs and incremental cost-effectiveness ratio (ICER). One-way sensitivity, probabilistic sensitivity and subgroup analyses were performed to evaluate uncertainty in the results.
In China, intravenous r-tPA gained an additional lifetime QALY of 0.293 with an additional cost of the Chinese Yuan (¥) of 7871 when compared with placebo, resulting in an ICER of ¥26 870 (US$3894)/QALY. In the USA, intravenous r-tPA yielded a higher QALY (difference: 0.430) and lower cost (difference: ¥-4563) when compared with placebo. In probabilistic sensitivity analyses, intravenous r-tPA had a 97.8% and 99.8% probability of being cost-effective or cost-saving in China and the USA, respectively. These findings remained robust under one-way sensitivity and subgroup analysis except for patients with a National Institute of Health Stroke Scale Score of less than 4, between 11 and 16, and over 16.
MRI-guided intravenous r-tPA for patients with AIS with an unknown time of onset is cost-effective in China and cost-saving in the USA.
WAKE-UP 试验已经证明,对于起病时间不明的急性缺血性脑卒中(AIS)患者,MRI 引导下静脉注射重组组织型纤溶酶原激活剂(r-tPA)具有疗效。本研究旨在从中国和美国医疗保健支付者的角度评估其长期成本效益。
采用决策树和 Markov 模型相结合的方法,预测静脉注射 r-tPA 或安慰剂治疗相关的终生成本和质量调整生命年(QALYs)。模型输入包括转移概率、成本和效用,来源于 WAKE-UP 试验、类似的成本效益研究和其他已发表的来源。为了比较静脉注射 r-tPA 与安慰剂,我们计算了增量成本、增量 QALYs 和增量成本效益比(ICER)。进行了单因素敏感性分析、概率敏感性分析和亚组分析,以评估结果的不确定性。
在中国,与安慰剂相比,静脉注射 r-tPA 可额外获得 0.293 个终生 QALY,额外成本为 7871 元人民币(¥),ICER 为 ¥26870($3894)/QALY。在美国,与安慰剂相比,静脉注射 r-tPA 可获得更高的 QALY(差异:0.430)和更低的成本(差异:¥-4563)。在概率敏感性分析中,静脉注射 r-tPA 在我国有 97.8%和 99.8%的概率具有成本效益或成本节约,在美国有 99.8%的概率具有成本效益或成本节约。除 NIHSS 评分低于 4、11-16 和大于 16 的患者外,这些发现经单因素敏感性分析和亚组分析后仍然稳健。
对于起病时间不明的 AIS 患者,MRI 引导下静脉注射 r-tPA 在我国具有成本效益,在美国具有成本节约效果。