泊马度胺治疗多发性骨髓瘤伴髓外病变患者的疗效观察  

Efficacy of Pomalidomide in Multiple Myeloma with Extramedul-lary Disease

作　　者：张平平 王蒙 伍燕平 李佳佳 ZHANG Ping-Ping;WANG Meng;WU Yan-Ping;LI Jia-Jia(Department of Hematology,The First Affiliated Hospital of Bengbu Medical College,Bengbu 233000,Anhui Province,China)

机构地区：[1]蚌埠医科大学第一附属医院血液科,安徽蚌埠233000

出　　处：《中国实验血液学杂志》2025年第2期463-468,共6页Journal of Experimental Hematology

基　　金：蚌埠医学院自然科学重点项目(2021byzd166);安徽省高等学校科学研究项目(2023AH051909)。

摘　　要：目的:探讨泊马度胺治疗多发性骨髓瘤(MM)伴髓外病变(EMD)患者的疗效与安全性。方法:回顾性分析2019年2月至2023年8月蚌埠医学院第一附属医院血液科收治的40例MM患者的临床资料,这些患者均接受以泊马度胺治疗为基础的化疗。结果:40例患者中初诊MM伴EMD患者8例,难治/复发MM伴EMD患者32例;伴骨相关髓外病变(EMD-B)21例,软组织相关髓外病变(EM-S)19例。与EM-B组相比,EM-S组LDH更低(P<0.05)、乳酸脱氢酶(LDH)水平升高(P<0.05),且无进展生存期(PFS)更短(11对21.5个月,P=0.0363)。32例患者完成3个疗程,进行短期疗效评估,初诊组与难治复发组3药及以上联合方案的使用率及ASCT治疗无差异(87.50%对93.75%,P>0.05;25.00%对15.63%,P>0.05)。相较于难治复发组,初诊患者总体缓解率(ORR)明显升高(83.33%对57.70%,P<0.05)。初诊组的深度缓解率(VGPR+CR)优于难治复发组(50.00%对29.62%,P<0.05),初诊组的无效率(SD+PD)明显低于难治复发组(33.33%对65.38%,P<0.05),而初诊组的部分缓解率(PR)、死亡率与难治复发组无显著性差异(P>0.05)。中位随访26个月,患者中位PFS 19个月。单因素分析结果显示,EM-S、高危遗传学异常、诱导治疗未达部分缓解(PR)或以上、2线及以上治疗失败与PFS短相关;进一步多因素分析结果显示,诱导治疗最佳疗效未达PR或以上、EM-S类型为影响患者PFS的独立不良预后因素。安全性分析结果显示,血液学不良反应16例,3/4级为3例,1/2级13例;最常见非血液学不良反应为恶心、呕吐、乏力和腹胀,反应轻微,可耐受。结论:泊马度胺为基础的化疗方案在MM伴髓外病变的患者中早期有效率高、可耐受。Objective:To investigate the efficacy and safety of pomalidomide in the treatment of multiple myeloma(MM)with extramedullary disease(EMD).Methods:The clinical data of 40 pomalidomide-based multiple myeloma patients with extramedullary disease admitted to the Department of Hematology,the First Affiliated Hospital of Bengbu Medical College from February 2019 to August 2023 were retrospectively analyzed.Results:Among the 40 patients,8 were newly diagnosed with EMD and 32 were refractory/relapsed EMD.There were 21 cases with bone-related extramedullary disease(EM-B)and 19 cases with soft tissue-related extramedullary disease(EM-S).Compared with the EM-B group,the EM-S group exhibited lower LDH levels,an elevation in LDH and a shorter progression-free survival(PFS)(11 months vs 21.5 months,P=0.0363).32 patients completed 3 courses of treatment,and the short-term efficacy was evaluated.There was no significant difference in the rate of use of 3 or more drugs and ASCT treatment between NDMM group and RRMM group(87.50%vs 93.75%,P>0.05;25.00%vs 15.63%,P>0.05).Compared with the RRMM group,the overall response rate(ORR)of the NDMM patients was significantly higher(83.33%vs 57.70%,P<0.05).The deep remission rate(VGPR+CR)of the NDMM group was better than that of the RRMM group(50.00%vs 29.62%,P<0.05),and the non-response rate(SD+PD)of the NDMM group was significantly lower than that of the R/RMM group(33.33%vs 65.38%,P<0.05),while the partial remission rate(PR)and mortality rate of the NDMM were not significantly different from those of the RRMM group(P>0.05).With a median follow-up of 26 months,the median PFS was 19 months.Univariate analysis showed that EM-S,high-risk genetic abnormalities,induction therapy did not achieve partial response(PR)or better,and more than 2 lines of treatment failure were associated with shorter PFS.Multivariate analysis showed that the best response to induction therapy did not achieve PR or better,EM-S were an independent adverses prognostic factor for PFS.The results of safety analysis showed

关 键 词：多发性骨髓瘤 髓外病变 泊马度胺 预后 

分 类 号：R733.3[医药卫生—肿瘤]