Rui Mingjun, Wang Yingcheng, Fei Zhengyang, Shang Ye, Ma Aixia, Li Hongchao
School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, China.
Center for Pharmacoeconomics and Outcomes Research, China Pharmaceutical University, Nanjing, China.
Front Med (Lausanne). 2021 Mar 18;8:657539. doi: 10.3389/fmed.2021.657539. eCollection 2021.
This study aimed to compare the economic evaluation of recombinant human thrombopoietin+rituximab (rhTPO + RTX) vs. RTX as second-line treatment for adult patients with immunologic thrombocytopenic purpura in China. The Markov model was used in our research. The response rate and relapse rate data were derived from two clinical trials and one retrospective study. Cost and utility values were derived from published literature, a third-party database, and healthcare documents. In addition, one-way sensitivity analysis and probabilistic sensitivity analysis were performed to observe the stability of the model and data source. In the Markov model, compared with RTX, rhTPO+RTX yielded an additional 0.04 QALYs, with an incremental cost of 2,802 USD. The ICER was 69,097 USD/QALY. According to the results from the one-way sensitivity analysis, complete response of rhTPO+RTX, utility of complete response and response of RTX were the main drivers in the model. The results from the probabilistic sensitivity analysis demonstrated that there was a 100% probability that rhTPO+RTX was not cost-effective vs. RTX alone at a threshold of $10,805/QALY and an 84% probability at a threshold of $32,415/QALY. RTX+rhTPO was not more cost-effective than RTX alone as second-line treatment for adult patients with immunologic thrombocytopenic purpura in China.
本研究旨在比较重组人血小板生成素联合利妥昔单抗（rhTPO + RTX）与利妥昔单抗（RTX）作为中国成人免疫性血小板减少症二线治疗方案的经济学评估。我们的研究采用了马尔可夫模型。缓解率和复发率数据来自两项临床试验和一项回顾性研究。成本和效用值来自已发表的文献、第三方数据库和医疗保健文件。此外，还进行了单向敏感性分析和概率敏感性分析，以观察模型和数据来源的稳定性。在马尔可夫模型中，与RTX相比，rhTPO+RTX可额外产生0.04个质量调整生命年（QALY），增量成本为2802美元。增量成本效果比（ICER）为69,097美元/QALY。根据单向敏感性分析结果，rhTPO+RTX的完全缓解率、完全缓解的效用值和RTX的缓解率是模型中的主要驱动因素。概率敏感性分析结果表明，在阈值为10,805美元/QALY时，rhTPO+RTX相对于单独使用RTX不具有成本效益的概率为100%；在阈值为32,415美元/QALY时，该概率为84%。在中国，对于成人免疫性血小板减少症患者，RTX联合rhTPO作为二线治疗方案并不比单独使用RTX更具成本效益。
