中国慢性淋巴细胞白血病患者的年均经济负担分析

Annual Economic Burden of Chronic Lymphocytic Leukemia in China

目的 :通过对我国慢性淋巴细胞白血病(Chronic Lymphocytic Leukemia, CLL)患者的直接医疗成本、直接非医疗成本和间接成本进行定量研究,评估该疾病(包括初治以及复发病人)目前以化疗为基础的临床常规治疗方案(包括常规化疗和免疫化疗)的年均经济负担、为进一步促进临床合理优选治疗方案和卫生资源的合理配置提供科学依据和政策建议。方法 :通过系统性文献综述以确定公开发表的CLL疾病的流行病学数据、CLL患者的经济负担数据以及生命质量。并通过在中国五个城市中的专家咨询会(Delphi panel)收集真实世界中CLL患者年均的直接医疗成本、直接非医疗成本和间接成本数据并进行统计分析。最后通过Microsoft Excel 2013构建年均经济负担模型对CLL患者的经济负担进行测算,并对关键参数进行单因素敏感性分析。结果 :对我国5个城市22家三甲医院的血液科和肿瘤科(副主任以上)的临床专家进行调研,共收集到36份初治CLL的有效问卷,34份复发CLL的有效问卷。采用目前临床上的常规治疗方案进行治疗的初治CLL患者的年均总成本为258,818元,其中年均直接医疗成本为199,091元。复发CLL患者的年均总成本为387,450元,其中年均直接医疗成本为309,378元。单因素敏感性分析显示,影响初治CLL患者和复发CLL患者的年均经济负担最大的两个因素均为化疗相关的支持性用药花费和化疗相关的年均住院次数。结论 :慢性淋巴细胞白血病给社会及患者造成沉重的经济负担,政府和医疗机构应引起重视并采取合理的政策和措施降低慢性淋巴细胞白血病患者的经济负担,减轻化疗相关的花费极为重要。鼓励采用给药途径简单、疗效更高、安全性更好的创新靶向药物的开发和使用。

Objectives： Chronic Lymphocytic Leukemia （CLL） is a rare, incurable, relapse disease. Chemo-based therapy, although documented with severe adverse events and extremely negative impact on patients’ quality of life （QoL）, were still the most common treatment in CLL patients in present clinical practice, due to the fact that lack of better treatment choices. This study aims to evaluate the annual economic costs （including direct and indirect cost） of chemo-based therapy in CLL patients （including frontline CLL patients and relapse/refractory CLL（rrCLL） patients） from societal perspective. Methods： A comprehensive literature review was performed to collect the published disease burden data on CLL patients. Questionnaires were designed to collect relevant clinical and costs information （including direct medical cost, direct non-medical cost and indirect cost） via 5 Delphi panels. The panel were conducted with hematologists and oncologists to document the treatment burden. CLL disease burden model is built via Microsoft Excel 2013 to assess the economic burden of CLL. One-way sensitive analysis is was performed.Results： Total 36 hematologists and oncologists from Hangzhou, Wuhan, Xi’an, Guangzhou and Chengdu participated in Delphi panels. The average total annual cost per frontline CLL patient and perr rCLL patients with chemo-based therapy were 258,818CNY and 387,450CNY respectively, with the annual direct medical cost of 199,091CNY and 309,378CNY respectivelyy. Sensitivity analysis shows that chemotherapy-related supportive drug cost and annual frequency of inpatient care are the factor which impact the result most.Conclusion： Chemo-based therapy, although clinically eff cacious, imposing signiff cant economic burden in frontline CLL patients and rrCLL patients in China. This study highlights the critical need to introduce innovative therapies with better clinical eff cacies with improved patient QoL, reduced total cost and a simple admission for CLL patients in China.