The George Institute for Global Health, University of New South Wales, Kensington, New South Wales, Australia.
School of Health Policy & Management, Nanjing Medical University, Nanjing, China.
Cerebrovasc Dis. 2023;52(2):145-152. doi: 10.1159/000525869. Epub 2022 Aug 31.
The Enhanced Control of Hypertension and Thrombolysis Stroke Study (ENCHANTED) showed that a low-dose alteplase was safe but not clearly non-inferior to standard-dose alteplase in acute ischemic stroke (AIS). Given the significant cost of this medicine, we undertook a cost-effectiveness analysis to determine the probability that low-dose is cost-effective relative to standard-dose alteplase in China.
For ENCHANTED participants in China with available health cost data, cost-effectiveness and cost-utility analyses were undertaken in which death or disability (modified Rankin scale scores 2-6) at 90 days and quality-adjusted life-years (QALYs) were used as outcome measures, respectively. There was adherence to standard guidelines for health economic evaluations alongside non-inferiority trials and according to a health-care payer's perspective. The equivalence margin for cost and effectiveness was set at USD 691 and -0.025 QALYs, respectively, for the base-case analysis. Probabilistic sensitivity analyses were used to evaluate the probability of low-dose alteplase being non-inferior.
While the mean cost of alteplase was lower in the low-dose group (USD 1,569 vs. USD 2,154 in the standard-dose group), the total cost was USD 56 (95% confidence interval [CI]: -1,000-1,113) higher compared to the standard-dose group due to higher hospitalization costs in the low-dose group. There were 462 (95% CI: 415-509) and 410 (95% CI: 363-457) patients with death or disability per 1,000 patients in the low-dose and standard-dose groups, respectively. The low-dose group had marginally lower (0.008, 95% CI: -0.016-0.001) QALYs compared to their standard-dose counterparts. The low-dose group was found to have an 88% probability of being non-inferior based on cost-effectiveness versus the standard-dose group.
This health economic evaluation alongside the ENCHANTED indicates that the use of low-dose alteplase does not save overall healthcare costs nor lead to a gain in QALYs in the management of Chinese patients with AIS compared to the use of standard dose. There is little justification on economic grounds to shift from standard-of-care thrombolysis in AIS.
强化降压溶栓治疗急性缺血性卒中研究（ENCHANTED）表明，小剂量阿替普酶溶栓是安全的，但与标准剂量阿替普酶相比，在急性缺血性卒中（AIS）患者中并不具有明显的非劣效性。鉴于该药的显著成本，我们进行了一项成本效益分析，以确定在中国，小剂量相对于标准剂量阿替普酶的成本效益的概率。
对于在中国参加 ENCHANTED 研究且有可用健康成本数据的患者，我们进行了成本效益和成本效用分析，分别使用 90 天时的死亡或残疾（改良 Rankin 量表评分 2-6）和健康调整生命年（QALY）作为结局指标。我们遵循了非劣效性试验和基于医保支付方视角的标准健康经济学评价指南。成本和效果的等效边界分别设定为 691 美元和 -0.025 QALY，用于基础情况分析。概率敏感性分析用于评估小剂量阿替普酶非劣效的概率。
尽管小剂量组阿替普酶的平均成本较低（1569 美元比标准剂量组的 2154 美元），但由于小剂量组住院费用较高，总费用仍高出 56 美元（95%置信区间[CI]：-1000 至 1113 美元）。小剂量组和标准剂量组每 1000 例患者中有 462（95%CI：415-509）和 410（95%CI：363-457）例患者发生死亡或残疾。小剂量组的 QALY 略低（0.008，95%CI：-0.016-0.001）。基于成本效益，小剂量组被发现有 88%的可能性优于标准剂量组。
这项与 ENCHANTED 研究相关的健康经济学评价表明，与标准剂量相比，在中国 AIS 患者的治疗中，使用小剂量阿替普酶不会节省总体医疗保健成本，也不会带来 QALY 的增加。在经济上，没有理由将 AIS 的标准溶栓治疗转变为小剂量治疗。
