髓外病变对初诊多发性骨髓瘤患者预后的影响

Effects of extramedullary disease on patients with newly diagnosed multiple myeloma

目的比较初诊多发性骨髓瘤(NDMM)非髓外病变(non-EMD)、骨旁髓外(EM-B)和软组织髓外(EM-E)患者的临床特征及预后,并探讨自体造血干细胞移植(ASCT)对EMD的影响。方法回顾性分析上海交通大学医学院附属瑞金医院2015年1月至2022年1月收治的515例NDMM患者,114例(22%)伴EMD患者,其中EM-B组91例(18%),EM-E组23例(4%)。通过卡方检验比较3组患者临床特征的差异;通过Kaplan-Meier法比较3组患者无进展生存(PFS)和总生存(OS)差异;通过Cox比例风险模型的多因素分析确定独立预后因素。结果三组患者在年龄、性别、国际分期系统(ISS)分期、轻链、肌酐清除率、细胞遗传学危险度、17p缺失、是否接受ASCT和诱导方案类型等方面差异均无统计学意义。13%EM-E患者M蛋白为IgD型,显著增高(P=0.021)。non-EMD、EM-B和EM-E 3组患者中位PFS时间分别为27.4、23.1和14.0个月,中位OS期分别为未达到、76.8个月和25.6个月,EM-E的PFS(对non-EMD,P=0.004;对EM-B,P=0.036)和OS(对non-EMD,P<0.001;对EM-B,P=0.002)最差,而EM-B与non-EMD组之间PFS和OS差异无统计学意义。多因素分析中,EM-E是NDMM患者OS的独立预后危险因素(HR=8.779,P<0.001),对PFS也产生不良影响(HR=1.874,P=0.050)。在未接受ASCT患者中,EM-B患者OS显著差于non-EMD组(中位76.8个月对未达到,P=0.029),但在ASCT患者中,EM-B和non-EMD组PFS和OS差异无统计学意义。结论与初诊non-EMD、EM-B比较,EM-E患者预后最差,而且EM-E是NDMM患者OS的独立危险因素。ASCT能克服EM-B的不良预后。

Objective To summarize the characteristics of patients with newly diagnosed multiple myeloma(NDMM)admitted at Ruijin Hospital affiliated to Shanghai Jiaotong University School of Medicine.We compared the clinical characteristics and prognoses among patients with non-extramedullary disease(EMD),bone-related extramedullary(EM-B)disease,and extraosseous extramedullary(EM-E)disease and further explored the effects of autologous hematopoietic stem cell transplantation(ASCT)for EMD.Methods From January 2015 to January 2022,data of 114 patients(22%)with EMD out of 515 patients with NDMM were retrospectively analyzed;91(18%)and 23(4%)patients comprised the EM-B and EM-E groups,respectively.The clinical characteristics of patients in all groups were compared with the Chi-square test.Progression-free survival(PFS)and overall survival(OS)of patients were analyzed by the Kaplan-Meier method.Independent prognostic factors were determined using multivariate Cox proportional hazard model.Results There were no significant differences in age,gender,ISS stage,light chain,creatinine clearance,cytogenetic risk,17p deletion,ASCT,and induction regimens among the three groups.Overall,13%of EM-E patients had IgD-type M protein,which was significantly higher than that in EM-B patients(P=0.021).The median PFS of patients in the non-EMD,EM-B,and EM-E groups was 27.4,23.1,and 14.0 months;the median OS was not reached,76.8 months,and 25.6 months,respectively.The PFS(vs non-EMD,P=0.004;vs EM-B,P=0.036)and OS(vs non-EMD,P<0.001;vs EM-B,P=0.002)were significantly worse in patients with EM-E,while those were not significantly different between patients with EM-B and those with non-EMD.In the multivariate analysis,EM-E was an independent prognostic factor for OS in patients with NDMM(HR=8.779,P<0.001)and negatively impacted PFS(HR=1.874,P=0.050).In those who did not undergo ASCT,patients with EM-B had significantly worse OS than those with non-EMD(median 76.8 months vs.not reached,P=0.029).However,no significant difference was observed in the PFS and OS of patients with EM-B and those with non-EMD who underwent ASCT.Conclusions Compared to patients with either non-EMD or EM-B,those with EM-E had the worst prognosis.EM-E was an independent risk factor for OS in patients with NDMM.ASCT can overcome the poor prognosis of EM-B.