单中心PAD、PCD与RVD方案治疗新诊断多发性骨髓瘤的疗效及成本-效果分析  

Efficacy and cost-effectiveness analysis of PAD,PCD and RVD regimens in the treatment of newly diagnosed multiple myeloma in a single center

作　　者：杨宁 刘佳[1] 肖菲[1] 李昕[1] 徐岚[1] 侯健 钟华[1] YANG Ning;LIU Jia;XIAO Fei;LI Xin;XU Lan;HOU Jian;ZHONG Hua(Department of Hematology,Ren Ji Hospital,School of Medicine,Shanghai Jiao Tong University,Shanghai 201112,China)

机构地区：[1]上海交通大学医学院附属仁济医院血液科,上海200127

出　　处：《肿瘤》2021年第3期186-196,共11页Tumor

摘　　要：目的:通过单中心数据回顾性分析比较PAD方案[硼替佐米(bortezomib)+多柔比星(doxorubicin)+地塞米松(dexamethasone)]、PCD方案[硼替佐米+环磷酰胺(cyclophosphamide)+地塞米松]与RVD方案[硼替佐米+来那多胺(lenalidomide)+地塞米松]治疗新诊断多发性骨髓瘤(newly diagnosed multiple myeloma,NDMM)的疗效及药物经济学效果。方法:回顾性分析2014年7月—2019年12月由上海交通大学医学院附属仁济医院血液科收治的93例NDMM患者的临床资料,其中PAD方案组31例,PCD方案组30例,RVD方案组32例,比较3组患者的疗效以及不良反应,并进行成本-效果分析。结果:PAD、PCD和RVD组患者在治疗4个疗程后的总有效率分别为80.64%、80.00%和90.63%(P=0.441),高质量缓解率分别为41.94%、36.67%和62.50%(P=0.096)。PAD、PCD和RVD组预估中位无进展生存期(median progression-free survival,mPFS)分别为28.0、29.0和24.0个月,差异无统计学意义(P=0.35)。3组患者与治疗相关的不良反应中,PAD组患者肝损害发生率、中性粒细胞减少发生率和血小板减少发生率分别为12.90%、38.71%和6.25%,均高于其他2组(P=0.020,P=0.003和P=0.001)。RVD组血栓发生率(6.25%)较其他2组更高(P=0.326)。3组患者中,未预防性使用阿昔洛韦者带状疱疹的发生率为13.04%,预防性使用阿昔洛韦者带状疱疹的发生率为1.43%(P=0.045)。PAD、PCD和RVD组的成本-效果比分别为1430.69、1415.71和1668.95。PAD组和RVD组相对PCD组的增量成本-效果比分别为3303.59和3574.83,敏感度分析结果与该结果相符。结论:PAD、PCD和RVD组患者治疗4个疗程后的总有效率相当,但PCD组方案成本-效果优于其他2组。3组间临床不良反应虽有一定差异,但是从安全、有效和经济的角度考虑,PCD组方案可作为国内NDMM患者的首选治疗方案之一。Objective:To compare the efficacy and the pharmacoeconomic effect of PAD regimen(bortezomib+doxorubicin+dexamethasone),PCD regimen(bortezomib+cyclophosphamide+dexamethasone)and VRD regimen(bortezomib+lenalidomide+dexamethasone)in the treatment of patients with newly diagnosed multiple myeloma(NDMM).Methods:The clinical data of 93 NDMM patients admitted to Department of Hematology,Ren Ji Hospital from July 2014 to December 2019 were analyzed retrospectively.NDMM patients were divided into three groups according to different treatment regimens.PAD group(31 patients)was treated with PAD regimen(bortezomib+doxorubicin+dexamethasone),PCD group(30 patients)was treated with PCD regimen(bortezomib+cyclophosphamide+dexamethasone),RVD group(32 patients)was treated with RVD regimen(bortezomib+lenalidomide+dexamethasone).The efficacy and adverse reactions of the three groups of patients were compared,and cost-effectiveness analysis was performed.Results:The overall response rates of PAD,PCD and RVD groups after 4 courses of treatment were 80.64%,80.00%and 90.63%(P=0.441).The high quality response rates were 41.94%,36.67%and 62.50%respectively(P=0.096).The estimated median progression-free survival(mPFS)of PAD,PCD and RVD groups were 28.0 months,29.0 months and 24.0 months respectively,and the difference was not statistically significant(P=0.35).Among the treatment-related adverse reactions in the three groups of patients,the incidences of hepatic dysfunction,neutropenia and thrombocytopenia were 12.90%,38.71%and 41.94%in PAD group,which were higher than those of the other two groups(P=0.020,P=0.003 and P=0.001).The incidence of thrombosis in the RVD group was 6.25%,which was higher than that of the other two groups(P=0.326).The incidence of herpes zoster in patients who did not use acyclovir preventively was significantly higher than those patients who used acyclovir preventively(13.04%vs 1.43%,P=0.045).The cost-effectiveness ratios of the PAD,PCD and RVD groups were 1430.69,1415.71 and 1668.95,respectively.The incremental c

关 键 词：多发性骨髓瘤 抗肿瘤联合化疗方案 疗效比较研究 药物相关性副作用和不良反应 经济学 药物 

分 类 号：R733.3[医药卫生—肿瘤]