注射用贝林妥欧单抗治疗中国儿童复发或难治性急性淋巴细胞白血病的成本-效果分析  

Cost effectiveness analysis of blinatumomab in pediatric patients with relapsed or refractory precursor B-Cell acute lymphoblastic leukemia in China

作　　者：陈伟琪 朱奇云 孙沁怡 杨照 江滨[1,2] CHEN Weiqi;ZHU Qiyun;SUN Qinyi;YANG Zhao;JIANG Bin(School of Pharmacy,Peking University,Beijing 100191,China;Public Policy Research Center,Peking University,Beijing 100871,China;Scientific Research Department,Peking University First Hospital,Beijing 100034,China)

机构地区：[1]北京大学药学院,北京100191 [2]北京大学公共政策研究中心,北京100871 [3]北京大学第一医院科研处,北京100034

出　　处：《世界临床药物》2024年第1期45-52,共8页World Clinical Drug

摘　　要：目的 从全社会角度,评估注射用贝林妥欧单抗对比化疗治疗儿童复发或难治性前体B细胞急性淋巴细胞白血病的经济性。方法 构建包含无事件生存、复发及死亡三种健康状态的分区生存模型,基线模拟时长为20年。临床参数来源于Ⅲ期随机对照临床研究20120215,效用参数来源于上海儿童医学中心白血病患者的生命质量研究,成本参数来源于易联华招数据库、米内网数据库及国家统计局等。支付意愿阈值设定为128 547元(1.50倍2022年中国人均GDP)。采用单因素敏感性分析和概率敏感性分析检验基础分析结果的稳健性。结果 干预组患者可获得8.74个质量调整生命年(quality adjusted life years,QALYs)、10.53个生命年(life years,LYs),相比对照组增加3.01个QALYs、3.46个LYs。同时,干预组的总成本为1 060 929元,相比于对照组增加245 719元。干预组相比于对照组的增量成本-效果比为81 623元/QALY。敏感性分析结果证实基础分析结果的稳健性。结论 贝林妥欧单抗相比化疗治疗中国儿童复发或难治性前体B细胞急性淋巴细胞白血病,更具有成本-效果。Objective To compare the cost effectiveness of blinatumomab and chemotherapy in the treatment of relapsed or refractory precursor B-Cell acute lymphoblastic leukemia(ALL)from Chinese societal perspective.Methods A partition survival model with three health states(event-free,recurrence and death)was conducted over a 20 years horizon.Clinical data accessed from the phaseⅢrandomized controlled clinical study 20120215,utility values were obtained from the quality of life study of leukemia patients in Shanghai Children's Medical Centre,and cost inputs were identified from the Eliancloud database,Menet database,National Bureau of Statistics etc.The threshold of willingness to pay(WTP)was CNY 128547(1.50 times of 2022 Chinese per capita GDP).Deterministic sensitivity analysis and probabilistic sensitivity analysis were conducted to access the robustness of model and findings.Results The patients of intervention group achieved 8.74 quality-adjusted life years(QALYs)and 10.53 life years(LYs),compared with control group,an increase of 3.01 QALYs and 3.46 LYs.The total cost for intervention group was CNY 1060929,an increase of CNY 245719 compared with control group.The incremental cost effectiveness ratio(ICER)in intervention group compared with control group was CNY 81623 per QALY.Results were robust to sensitivity analyses.Conclusion Blinatumomab is more cost effective in pediatric patients with relapsed or refractory precursor B-Cell ALL compared with chemotherapy from Chinese societal perspective.

关 键 词：急性淋巴细胞白血病 贝林妥欧单抗 化疗 分区生存模型 成本-效果分析 

分 类 号：R473.73[医药卫生—护理学]