地塞米松联合利妥昔单抗一线治疗成人慢性原发免疫性血小板减少症的经济性评价

王樱澄 芮明军 商叶 马爱霞

中圖分类号 R956;R593.31 文献标志码 A 文章编号 1001-0408（2021）24-3013-06

DOI 10.6039/j.issn.1001-0408.2021.24.12

摘 要 目的：评价地塞米松（DXM）联用利妥昔单抗（RTX）一线治疗成人慢性原发免疫性血小板减少症（ITP）的经济性。方法：从我国医疗卫生体系角度出发，以单用DXM方案为对照，构建八状态Markov模型，模型周期为4周，时限为20年，评价DXM联用RTX方案治疗成人慢性ITP的成本-效用，临床疗效、效用值参数来自已发表的文献，成本参数来自米内网、各地卫生健康委以及医保局官网;通过单因素敏感性分析、概率敏感性分析和情境分析来考察模型及参数来源的不确定性。结果：DXM+RTX方案的平均治疗费用为51 064美元，DXM方案的平均治疗费用为50 455美元;与单用DXM相比，DXM+RTX方案可以让每位患者平均多获得0.14个质量调整生命年（QALYs），增量成本-效果比（ICER）为4 356美元/QALY，低于2020年我国人均国内生产总值（GDP）这一意愿支付阈值。在单因素敏感性分析中，药品费用是对结果影响最大的参数;在概率敏感性分析中，当支付阈值为1～3倍2020年我国人均GDP时，DXM+RTX方案具有经济性的概率为57.5%～61.0%;情境分析结果表明，DXM+RTX方案可能具有明显的长期获益，效用值对结论的影响较小。结论：DXM+RTX相比单用DXM一线治疗成人慢性ITP具有经济性，但是结果具有一定的不确定性。

关键词 药物经济学评价;原发免疫性血小板减少症;利妥昔单抗;地塞米松;一线治疗

Economic Evaluation of Dexamethasone Combined with Rituximab for the First-line Treatment of Chronic Primary Immune Thrombocytopenia in Adults

WANG Yingcheng1，2，RUI Mingjun1，2，SHANG Ye1，2，MA Aixia1，2（1. School of International Pharmaceutical Business， China Pharmaceutical University， Nanjing 211198，China; 2. Center for Pharmacoeconomic Evaluation Research， China Pharmaceutical University， Nanjing 211198，China）

ABSTRACT   OBJECTIVE： To evaluate the economy performance of dexamethasone （DXM） combined with rituximab （RTX） for the first-line treatment of chronic primary immune thrombocytopenia（ITP） in adults. METHODS： From the perspective of China’s medical and health system，Markov model for eight states was constructed with a period of 4 weeks and a time limit of 20 years， using DXM regimen as control. The cost-utility of DXM+RTX regimen for the treatment of chronic ITP in adults were evaluated. The parameters of clinical efficacy and utility value were derived from own published literature; cost parameters were from the MENET website and the official websites of local health committees and medical insurance bureaus;one-way sensitivity analysis， probability sensitivity analysis and scenario analysis were performed to observe the uncertainty of model and data source. RESULTS： The average cost of DXM+RTX regimen was 51 064 dollars and that of DXM regimen was 50 455 dollars. Compared with DXM regimen， DXM+RTX regimen yielded an additional 0.14 QALYs for each patient; the incremental cost-effectiveness ratio （ICER） was 4 356 dollars/QALY， and was lower than the willingness-to-pay threshold of China’s per capita gross domestic product （GDP） in 2020. In the one-way sensitivity analysis， the cost of drugs was the main driver in the model. Probability sensitivity analysis demonstrated that DXM+RTX regimen had 57.5%-61.0% probability of being cost-effective at a willingness- to-pay threshold of 1-3 times per capita GDP in 2020. The results of scenario analysis showed that DXM+RTX regimen would have obvious long-term benefits， and the utility value had little impact on the conclusion. CONCLUSIONS：DXM+RTX is more economical than DXM in the treatment of chronic ITP in adults， but the results have the uncertainty.

KEYWORDS   Pharmacoeconomic evaluation; Primary immune thrombocytopenia; Rituximab; Dexamethasone; First-line treatment

基金项目：江苏省研究生科研与实践创新计划项目（No. SJCX20_0219）

硕士研究生。研究方向：药物经济学、卫生经济与政策。E-mail：suisui1909@163.com

通信作者：教授，博士生导师，博士。研究方向：药物经济学、卫生经济与政策。……