自体造血干细胞移植治疗髓外多发性骨髓瘤疗效分析  

Efficacy of autologous hematopoietic stem cell transplantation in the treatment of extramedullary multiple myeloma

作　　者：何静松[1] 张恩帆 赵毅[1] 杨杨[1] 何冬花[1] 韩晓雁[1] 郑高锋[1] 陈晶晶[2] 罗依[1] 施继敏[1] 黄河[1] 蔡真[1] He Jingsong;Zhang Enfan;Zhao Yi;Yang Yang;He Donghua;Han Xiaoyan;Zheng Gaofeng;Chen Jingjing;Luo Yi;Shi Jimin;Huang He;Cai Zhen(Department of Hematology and Bone Marrow Transplantation,the First Affiliated Hospital,School of Medicine,Zhejiang University,Hangzhou 310003,China;Intravenous Drug Dispensing Center,the First Affiliated Hospital,School of Medicine,Zhejiang University,Hangzhou 310003,China)

机构地区：[1]浙江大学医学院附属第一医院血液科&骨髓移植中心,杭州310003 [2]浙江大学医学院附属第一医院静脉用药调配中心,杭州310003

出　　处：《中华移植杂志（电子版）》2021年第2期93-100,共8页Chinese Journal of Transplantation(Electronic Edition)

基　　金：国家自然科学基金(81800201,81800202,81872322);国家科技重大专项“重大新药开发”(2018ZX09733-003)。

摘　　要：目的探究自体造血干细胞移植(ASCT)治疗初发髓外多发性骨髓瘤(EMM)的疗效及其对生存的影响。方法回顾性分析2013年5月至2019年9月在浙江大学医学院附属第一医院确诊为EMM且适合接受ASCT的55例患者临床资料。55例EMM患者均接受以硼替佐米为基础的诱导治疗方案。根据患者是否接受ASCT为巩固治疗将其分为ASCT组(14例)和非ASCT组(41例)。采用国际骨髓瘤工作组制订的疗效评定标准评估疗效,包括完全缓解、非常好的部分缓解、部分缓解、疾病稳定和疾病进展。ASCT组和非ASCT组间非正态分布计量资料比较采用Mann-Whitney U检验;两组间计数资料比较采用卡方检验或Fisher确切概率法。采用Kaplan-Meier法绘制两组患者生存曲线,并采用log-rank检验进行比较。采用Cox比例风险模型进行影响EMM患者无进展生存(PFS)和总生存(OS)的单因素和多因素分析。P<0.05为差异有统计学意义。结果55例EMM患者中位年龄57岁(31~65岁),其中骨相关髓外病变49例,骨外髓外病变6例。巩固治疗前ASCT组和非ASCT组患者疗效差异无统计学意义(χ^(2)=2.808,P>0.05);巩固治疗后两组患者疗效差异有统计学意义(χ^(2)=6.946,P<0.05)。截至2020年6月30日,ASCT组患者中位PFS为46.0个月(95%CI:29.0~63.1),1、3和5年PFS率分别为92.3%、60.2%和30.1%;非ASCT组患者中位PFS为18.8个月(95%CI:11.3~26.3),1、3和5年PFS率分别为72.5%、36.3%和24.2%,两组患者PFS差异无统计学意义(P均>0.05)。ASCT组患者中位OS为未达到,1、3和5年OS率均为100%;非ASCT组患者中位OS为43.6个月(95%CI:38.9~57.5),1、3和5年OS率分别为87.8%、60.3%和48.4%,两组患者OS差异有统计学意义(χ^(2)=5.838,P<0.05)。Cox比例风险模型多因素分析结果表明:EMM类型和ASCT是影响EMM患者PFS的独立危险因素(HR=4.11和2.81,P均<0.05);EMM类型是影响EMM患者OS的独立危险因素(HR=5.23,P<0.05)。结论ASCT可明显改善以硼替佐米为诱导治疗的初Objective To study the effect of autologous hematopoietic stem cell transplantation(ASCT)as a consolidation therapy for newly diagnosed extramedullary multiple myeloma(EMM)and its effect on survival.Methods The clinical features,treatment,efficacy and survival of 55 patients with newly diagnosed EMM who were eligible for ASCT from May 2013 to September 2019 in the First Affiliated Hospital,School of Medicine,Zhejiang University,were retrospectively analyzed.All the EMM patients received bortezomib-based induction therapy regimen and were divided into ASCT group(n=14)and no ASCT group(n=41)according to whether or not the ASCT was performed as a consolidation therapy.The evaluative criterias set down by International Myeloma Working Group were used to evaluate the curative effect including complete remission,very good partial response,partial response,stable disease and progressive disease.The abnormal distribution measurement data between ASCT group and no ASCT group were compared by Mann-Whitney U test,and the enumeration data between the 2 groups were compared with chi-square test and Fisher exact probality test.The Kaplan-Meier method was used to draw the survival curve of the 2 groups and the log-rank test was used to compare.The Cox proportional hazard model was used to analyse the influence factors of progress free survive(PFS)and overall survival(OS).P<0.05 was considered statistically significant.Results The median age of the 55 EMM patients were 57 years old(31-65 years old).There were 49 cases of extramedullary-bone related and 6 cases of extramedullary-extraosseous.The difference of the curative effect after consolidation therapy between ASCT group and no ASCT group after consolidation therapy had no statistical significance(χ^(2)=2.808,P>0.05),but the difference of the curative effect between the 2 groups was statistically significant(χ^(2)=6.946,P<0.05).The EMM patients were followed until June 30,2020,and the median PFS of ASCT group were 46.0 months(95%CI:29.0-63.1),and the PFS rates of 1,3,5 year

关 键 词：髓外多发性骨髓瘤 自体造血干细胞移植 硼替佐米 

分 类 号：R733.3[医药卫生—肿瘤]