Department of Pharmacy/Evidence-Based Pharmacy Center, West China Second University Hospital, Sichuan University, Chengdu, China.
Key Laboratory of Birth Defects and Related Diseases of Women and Children, Sichuan University, Ministry of Education, Chengdu, China.
BMC Health Serv Res. 2023 Jun 19;23(1):652. doi: 10.1186/s12913-023-09600-7.
Tyrosine kinase inhibitors combined with conventional chemotherapy (CC) in treating Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph-positive ALL) has achieved promising efficacy and safety outcomes. The study was conducted to compare the cost-effectiveness between imatinib (HANSOH Pharma, Jiangsu, China) and dasatinib (CHIATAI TIANQING Pharma, Jiangsu, China) in treating pediatric Ph-positive ALL when combined with CC from the perspective of the health system in China.
A Markov model was established to simulate a hypothetical cohort of pediatric Ph-positive ALL patients receiving imatinib or dasatinib, combined with CC. The model was designed using a 10-year horizon, a 3- month cycle, and a 5% discount rate. Three health states were included: alive with progression-free survival, progressed disease, and death. Patient characteristics and transition probabilities were estimated based on clinical trials. Other relevant data, such as direct treatment costs and health utility data were extracted from published literature and Sichuan Province's centralized procurement and supervision platform. One-way sensitivity analysis and probabilistic sensitivity analysis were performed to assess the robustness of the results. The willingness-to-pay (WTP) was set as three times China's GDP per capita in 2021.
In the base-case analysis, the total medical costs were $89,701 and $101,182, and the quality-adjusted life years (QALYs) gained were 1.99 and 2.70, for imatinib and dasatinib regimens, respectively. The incremental cost-effectiveness ratio for dasatinib versus imatinib was $16,170/QALY. The probabilistic sensitivity analysis indicated that treatment with dasatinib combined with CC achieved a 96.4% probability of cost-effectiveness at a WTP threshold of $37,765/QALY.
Dasatinib combined with CC is likely to be a cost-effective strategy compared to imatinib combination therapy for pediatric Ph-positive ALL in China at a WTP threshold of $37,765/QALY.
酪氨酸激酶抑制剂联合常规化疗（CC）治疗费城染色体阳性急性淋巴细胞白血病（Ph 阳性 ALL）已取得令人满意的疗效和安全性结果。本研究旨在比较伊马替尼（江苏豪森药业集团有限公司）和达沙替尼（正大天晴药业集团股份有限公司）联合 CC 治疗儿童 Ph 阳性 ALL 的成本效果，从中国卫生系统的角度出发。
采用 Markov 模型模拟接受伊马替尼或达沙替尼联合 CC 治疗的儿童 Ph 阳性 ALL 患者的假设队列。该模型的设计采用 10 年的时间范围、3 个月的周期和 5%的贴现率。包括 3 种健康状态：无进展生存的存活状态、疾病进展和死亡。基于临床试验，对患者特征和转移概率进行了估计。其他相关数据，如直接治疗成本和健康效用数据，均从已发表的文献和四川省集中采购和监管平台中提取。进行了单因素敏感性分析和概率敏感性分析，以评估结果的稳健性。支付意愿（WTP）设定为 2021 年中国人均 GDP 的 3 倍。
在基线分析中，伊马替尼和达沙替尼方案的总医疗费用分别为 89701 美元和 101182 美元，获得的质量调整生命年（QALY）分别为 1.99 和 2.70。达沙替尼与伊马替尼相比，增量成本效果比为 16170 美元/QALY。概率敏感性分析表明，达沙替尼联合 CC 治疗方案在 WTP 阈值为 37765 美元/QALY 时，有 96.4%的可能性达到成本效果。
在 WTP 阈值为 37765 美元/QALY 时，达沙替尼联合 CC 可能是治疗中国儿童 Ph 阳性 ALL 的一种具有成本效果的策略。
