Sruamsiri Rosarin, Dilokthornsakul Piyameth, Pratoomsoot Chayanin, Chaiyakunapruk Nathorn
Department of Pharmacy Practice, Faculty of Pharmaceutical Sciences, Center of Pharmaceutical Outcomes Research, Naresuan University, Phitsanulok, Thailand.
Pharmacoeconomics. 2014 Aug;32(8):801-13. doi: 10.1007/s40273-014-0171-9.
Although the international guideline recommends intravenous immunoglobulin (IVIG) as the first-line treatment for childhood idiopathic thrombocytopenia purpura (ITP) with life-threatening bleeding, ITP patients may not be able to access IVIG because of the limitation in health benefit packages especially in developing countries. There remains an important policy question as to whether IVIG used as a first-line treatment is worth the money spent. Thus, the objective of this study was to perform a cost-effectiveness analysis of adding IVIG to the standard treatment of platelet transfusion and corticosteroids, for the treatment of childhood ITP with life-threatening bleeding in the context of Thailand.
A cost-effectiveness analysis using a hybrid model consisting of a decision tree and Markov models was conducted with a societal perspective. The effectiveness and utility parameters were determined by systematic reviews, while costs and mortality parameters were determined using a retrospective electronic hospital database analysis. All costs were presented in 2012 US$. The discount rate of 3 % was applied for both costs and outcomes. One-way and probabilistic sensitivity analyses were also performed.
The incremental cost-effectiveness ratio (ICER) was $3,172 per quality-adjusted life-year gained ($/QALY) for the addition of IVIG versus standard treatment alone. The probability of response to corticosteroids was the most influential parameter on ICER. According to the willingness-to-pay of Thailand, of approximately $3,861/QALY, the probability of IVIG being cost effective was 33 %.
The addition of IVIG to standard treatment in the treatment of childhood ITP with life-threatening bleeding is possibly a cost-effective intervention in Thailand. However, our findings were highly sensitive. Policy makers may consider our findings as part of the information for their decision making.
尽管国际指南推荐静脉注射免疫球蛋白(IVIG)作为治疗伴有危及生命出血的儿童特发性血小板减少性紫癜(ITP)的一线治疗方法,但由于医保覆盖范围的限制,尤其是在发展中国家,ITP患者可能无法获得IVIG。关于将IVIG用作一线治疗是否物有所值,仍然存在一个重要的政策问题。因此,本研究的目的是在泰国的背景下,对在血小板输注和皮质类固醇标准治疗基础上加用IVIG治疗伴有危及生命出血的儿童ITP进行成本效益分析。
采用由决策树和马尔可夫模型组成的混合模型,从社会角度进行成本效益分析。有效性和效用参数通过系统评价确定,而成本和死亡率参数通过回顾性电子医院数据库分析确定。所有成本均以2012年美元表示。成本和结果均采用3%的贴现率。还进行了单向和概率敏感性分析。
与单独的标准治疗相比,加用IVIG的增量成本效益比(ICER)为每获得一个质量调整生命年(QALY)3172美元($/QALY)。对皮质类固醇的反应概率是对ICER影响最大的参数。根据泰国约3861美元/QALY的支付意愿,IVIG具有成本效益的概率为33%。
在泰国,对于治疗伴有危及生命出血的儿童ITP,在标准治疗基础上加用IVIG可能是一种具有成本效益的干预措施。然而,我们的研究结果具有高度敏感性。政策制定者可将我们的研究结果作为其决策信息的一部分加以考虑。